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Gene Therapy – Sickle Cell Anemia News

Gene therapy is an experimental technique that aims to treat genetic diseases by altering a disease-causing gene or introducing a healthy copy of a mutated gene to the body. The U.S. Food and Drug Administrationapprovedthe first gene therapy for an inherited disease a genetic form of blindness in December 2017.

Sickle cell anemia is caused by a mutation in the HBB gene which provides the instructions to make part of hemoglobin, the protein in red blood cells that carries oxygen.

Researchers are working on two different strategies to treat sickle cell anemia with gene therapy. Both of these strategies involve genetically altering the patients own hematopoietic stem cells. These are cells in the bone marrow that divide and specialize to produce different types of blood cells, including the red blood cells.

One strategy is to remove some of the patients hematopoietic stem cells, replace the mutated HBB gene in these cells with a healthy copy of the gene, and then transplant those cells back into the patient. The healthy copy of the gene is delivered to the cells using a modified, harmless virus. These genetically corrected cells will then hopefully repopulate the bone marrow and produce healthy, rather than sickled, red blood cells.

The other strategy is to genetically alter another gene in the patients hematopoietic stem cells so they boost production of fetal hemoglobin a form of hemoglobin produced by babies from about seven months before birth to about six months after birth. This type of hemoglobin represses sickling of cells in patients with sickle cell anemia, but most people only produce a tiny amount of it after infancy. Researchers aim to increase production of fetal hemoglobin in stem cells by using a highly specific enzyme to cut the cells DNA in the section containing one of the genes that suppress production of fetal hemoglobin. When the cell repairs its DNA, the gene no longer works and more fetal hemoglobin is produced.

Gene therapy offers an advantage over bone marrow transplant, in that complications associated with a bone marrow donation now the only cure for the disease such as finding the right match are not a concern.

Twelve clinical trials studying gene therapy to treat sickle cell anemia are now ongoing. Nine of the 12 are currently recruiting participants.

Four trials (NCT02186418, NCT03282656, NCT02247843, NCT02140554) are testing the efficacy and safety of gene therapy to replace the mutated HBB gene with a healthy HBB gene. These Phase 2 trials are recruiting both children and adults in the United States and Jamaica.

Three trials (NCT02193191, NCT02989701, NCT03226691) are investigating the use ofMozobil (plerixafor) in patients with sickle cell anemia to increase the production of stem cells to be used for gene therapy. This medication is already approved to treat certain types of cancer. All three are recruiting U.S. participants.

One trial (NCT00669305) is recruiting sickle cell anemia patients in Tennessee to donate bone marrow to be used in laboratory research to develop gene therapy techniques.

The final study(NCT00012545) is examining the best way to collect, process and store umbilical cord blood from babies with and without sickle cell anemia. Cord blood contains abundant stem cells that could be used in developing gene therapy for sickle cell anemia. This trial is open to pregnant women in Maryland both those who risk having an infant with sickle cell anemia, and those who do not.

One clinical trial (NCT02151526) conducted in France is still active but no longer recruiting participants. It is investigating the efficacy of gene therapy in seven patients. For the trial, a gene producing a therapeutic hemoglobin that functions similarly to fetal hemoglobin is introduced into the patients stem cells. A case studyfrom one of the seven was published in March 2017; it showed that the approach was safe and could be an effective treatment option for sickle cell anemia.

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Sickle Cell Anemia News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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Gene Therapy – Sickle Cell Anemia News

Gene therapy | medicine | Britannica.com

Gene therapy, also called gene transfer therapy, introduction of a normal gene into an individuals genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into the nucleus of a mutant cell, the gene most likely will integrate into a chromosomal site different from the defective allele; although that may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype.

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cancer: Gene therapy

Knowledge about the genetic defects that lead to cancer suggests that cancer can be treated by fixing those altered genes. One strategy is to replace a defective gene with its normal counterpart, using methods of recombinant DNA technology. Methods to insert genes into

Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis, adenosine deaminase deficiency, familial hypercholesterolemia, cancer, and severe combined immunodeficiency (SCID) syndrome. Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germline gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If that is achieved, those cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germline gene therapy has been achieved experimentally in animals but not in humans.

Scientists have also explored the possibility of combining gene therapy with stem cell therapy. In a preliminary test of that approach, scientists collected skin cells from a patient with alpha-1 antitrypsin deficiency (an inherited disorder associated with certain types of lung and liver disease), reprogrammed the cells into stem cells, corrected the causative gene mutation, and then stimulated the cells to mature into liver cells. The reprogrammed, genetically corrected cells functioned normally.

Prerequisites for gene therapy include finding the best delivery system (often a virus, typically referred to as a viral vector) for the gene, demonstrating that the transferred gene can express itself in the host cell, and establishing that the procedure is safe. Few clinical trials of gene therapy in humans have satisfied all those conditions, often because the delivery system fails to reach cells or the genes are not expressed by cells. Improved gene therapy systems are being developed by using nanotechnology. A promising application of that research involves packaging genes into nanoparticles that are targeted to cancer cells, thereby killing cancer cells specifically and leaving healthy cells unharmed.

Some aspects of gene therapy, including genetic manipulation and selection, research on embryonic tissue, and experimentation on human subjects, have aroused ethical controversy and safety concerns. Some objections to gene therapy are based on the view that humans should not play God and interfere in the natural order. On the other hand, others have argued that genetic engineering may be justified where it is consistent with the purposes of God as creator. Some critics are particularly concerned about the safety of germline gene therapy, because any harm caused by such treatment could be passed to successive generations. Benefits, however, would also be passed on indefinitely. There also has been concern that the use of somatic gene therapy may affect germ cells.

Although the successful use of somatic gene therapy has been reported, clinical trials have revealed risks. In 1999 American teenager Jesse Gelsinger died after having taken part in a gene therapy trial. In 2000 researchers in France announced that they had successfully used gene therapy to treat infants who suffered from X-linked SCID (XSCID; an inherited disorder that affects males). The researchers treated 11 patients, two of whom later developed a leukemia-like illness. Those outcomes highlight the difficulties foreseen in the use of viral vectors in somatic gene therapy. Although the viruses that are used as vectors are disabled so that they cannot replicate, patients may suffer an immune response.

Another concern associated with gene therapy is that it represents a form of eugenics, which aims to improve future generations through the selection of desired traits. Some have argued that gene therapy is eugenic but that it is a treatment that can be adopted to avoid disability. To others, such a view of gene therapy legitimates the so-called medical model of disability (in which disability is seen as an individual problem to be fixed with medicine) and raises peoples hopes for new treatments that may never materialize.

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Gene therapy | medicine | Britannica.com

Gene therapy – Mayo Clinic

Overview

Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body’s form and function, from making you grow taller to regulating your body systems. Genes that don’t work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body’s ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can’t easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells’ genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren’t the only vectors that can be used to carry altered genes into your body’s cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Dec. 29, 2017

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Gene therapy – Mayo Clinic

Gene therapy – Wikipedia

In the medicine field, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient’s cells as a drug to treat disease.[1][2] The first attempt at modifying human DNA was performed in 1980 by Martin Cline, but the first successful nuclear gene transfer in humans, approved by the National Institutes of Health, was performed in May 1989.[3] The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by French Anderson in a trial starting in September 1990.

Between 1989 and February 2016, over 2,300 clinical trials had been conducted, more than half of them in phase I.[4]

Not all medical procedures that introduce alterations to a patient’s genetic makeup can be considered gene therapy. Bone marrow transplantation and organ transplants in general have been found to introduce foreign DNA into patients.[5] Gene therapy is defined by the precision of the procedure and the intention of direct therapeutic effect.

Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies.

The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting organ transplantation) was performed by Martin Cline on 10 July 1980.[6][7] Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified[8] and even if he is correct, it’s unlikely it produced any significant beneficial effects treating beta-thalassemia.

After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990, when Ashi DeSilva was treated for ADA-SCID.[9]

The first somatic treatment that produced a permanent genetic change was performed in 1993.[citation needed]

Gene therapy is a way to fix a genetic problem at its source. The polymers are either translated into proteins, interfere with target gene expression, or possibly correct genetic mutations.

The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a “vector”, which carries the molecule inside cells.

Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers’ attention, although as of 2014[update], it was still largely an experimental technique.[10] These include treatment of retinal diseases Leber’s congenital amaurosis[11][12][13][14] and choroideremia,[15] X-linked SCID,[16] ADA-SCID,[17][18] adrenoleukodystrophy,[19] chronic lymphocytic leukemia (CLL),[20] acute lymphocytic leukemia (ALL),[21] multiple myeloma,[22] haemophilia,[18] and Parkinson’s disease.[23] Between 2013 and April 2014, US companies invested over $600 million in the field.[24]

The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers.[25] In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia.[26]In 2012 Glybera, a treatment for a rare inherited disorder, became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.[10][27]

Following early advances in genetic engineering of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered replacing or disrupting defective genes.[28] Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia, and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase.[27]

DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.[29] Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome.[30][31] Naked DNA approaches have also been explored, especially in the context of vaccine development.[32]

Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of nuclease function has led to more direct DNA editing, using techniques such as zinc finger nucleases and CRISPR. The vector incorporates genes into chromosomes. The expressed nucleases then knock out and replace genes in the chromosome. As of 2014[update] these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.[33]

Gene editing is a potential approach to alter the human genome to treat genetic diseases,[34] viral diseases,[35] and cancer.[36] As of 2016[update] these approaches were still years from being medicine.[37][38]

Gene therapy may be classified into two types:

In somatic cell gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a gamete, germ cell, gametocyte, or undifferentiated stem cell. Any such modifications affect the individual patient only, and are not inherited by offspring. Somatic gene therapy represents mainstream basic and clinical research, in which therapeutic DNA (either integrated in the genome or as an external episome or plasmid) is used to treat disease.

Over 600 clinical trials utilizing SCGT are underway[when?] in the US. Most focus on severe genetic disorders, including immunodeficiencies, haemophilia, thalassaemia, and cystic fibrosis. Such single gene disorders are good candidates for somatic cell therapy. The complete correction of a genetic disorder or the replacement of multiple genes is not yet possible. Only a few of the trials are in the advanced stages.[39]

In germline gene therapy (GGT), germ cells (sperm or egg cells) are modified by the introduction of functional genes into their genomes. Modifying a germ cell causes all the organism’s cells to contain the modified gene. The change is therefore heritable and passed on to later generations. Australia, Canada, Germany, Israel, Switzerland, and the Netherlands[40] prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations[40] and higher risks versus SCGT.[41] The US has no federal controls specifically addressing human genetic modification (beyond FDA regulations for therapies in general).[40][42][43][44]

The delivery of DNA into cells can be accomplished by multiple methods. The two major classes are recombinant viruses (sometimes called biological nanoparticles or viral vectors) and naked DNA or DNA complexes (non-viral methods).

In order to replicate, viruses introduce their genetic material into the host cell, tricking the host’s cellular machinery into using it as blueprints for viral proteins. Retroviruses go a stage further by having their genetic material copied into the genome of the host cell. Scientists exploit this by substituting a virus’s genetic material with therapeutic DNA. (The term ‘DNA’ may be an oversimplification, as some viruses contain RNA, and gene therapy could take this form as well.) A number of viruses have been used for human gene therapy, including retroviruses, adenoviruses, herpes simplex, vaccinia, and adeno-associated virus.[4] Like the genetic material (DNA or RNA) in viruses, therapeutic DNA can be designed to simply serve as a temporary blueprint that is degraded naturally or (at least theoretically) to enter the host’s genome, becoming a permanent part of the host’s DNA in infected cells.

Non-viral methods present certain advantages over viral methods, such as large scale production and low host immunogenicity. However, non-viral methods initially produced lower levels of transfection and gene expression, and thus lower therapeutic efficacy. Later technology remedied this deficiency.[citation needed]

Methods for non-viral gene therapy include the injection of naked DNA, electroporation, the gene gun, sonoporation, magnetofection, the use of oligonucleotides, lipoplexes, dendrimers, and inorganic nanoparticles.

Some of the unsolved problems include:

Three patients’ deaths have been reported in gene therapy trials, putting the field under close scrutiny. The first was that of Jesse Gelsinger, who died in 1999 because of immune rejection response.[51] One X-SCID patient died of leukemia in 2003.[9] In 2007, a rheumatoid arthritis patient died from an infection; the subsequent investigation concluded that the death was not related to gene therapy.[52]

In 1972 Friedmann and Roblin authored a paper in Science titled “Gene therapy for human genetic disease?”[53] Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those who suffer from genetic defects.[54]

In 1984 a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes.[55]

The first approved gene therapy clinical research in the US took place on 14 September 1990, at the National Institutes of Health (NIH), under the direction of William French Anderson.[56] Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The defective gene of the patient’s blood cells was replaced by the functional variant. Ashantis immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary.[57]

Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993).[58] The treatment of glioblastoma multiforme, the malignant brain tumor whose outcome is always fatal, was done using a vector expressing antisense IGF-I RNA (clinical trial approved by NIH protocolno.1602 November 24, 1993,[59] and by the FDA in 1994). This therapy also represents the beginning of cancer immunogene therapy, a treatment which proves to be effective due to the anti-tumor mechanism of IGF-I antisense, which is related to strong immune and apoptotic phenomena.

In 1992 Claudio Bordignon, working at the Vita-Salute San Raffaele University, performed the first gene therapy procedure using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases.[60] In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (ADA-SCID). The success of a multi-center trial for treating children with SCID (severe combined immune deficiency or “bubble boy” disease) from 2000 and 2002, was questioned when two of the ten children treated at the trial’s Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany.[61]

In 1993 Andrew Gobea was born with SCID following prenatal genetic screening. Blood was removed from his mother’s placenta and umbilical cord immediately after birth, to acquire stem cells. The allele that codes for adenosine deaminase (ADA) was obtained and inserted into a retrovirus. Retroviruses and stem cells were mixed, after which the viruses inserted the gene into the stem cell chromosomes. Stem cells containing the working ADA gene were injected into Andrew’s blood. Injections of the ADA enzyme were also given weekly. For four years T cells (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed.[62]

Jesse Gelsinger’s death in 1999 impeded gene therapy research in the US.[63][64] As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices.[65]

The modified cancer gene therapy strategy of antisense IGF-I RNA (NIH n 1602)[59] using antisense / triple helix anti-IGF-I approach was registered in 2002 by Wiley gene therapy clinical trial – n 635 and 636. The approach has shown promising results in the treatment of six different malignant tumors: glioblastoma, cancers of liver, colon, prostate, uterus, and ovary (Collaborative NATO Science Programme on Gene Therapy USA, France, Poland n LST 980517 conducted by J. Trojan) (Trojan et al., 2012). This anti-gene antisense/triple helix therapy has proven to be efficient, due to the mechanism stopping simultaneously IGF-I expression on translation and transcription levels, strengthening anti-tumor immune and apoptotic phenomena.

Sickle-cell disease can be treated in mice.[66] The mice which have essentially the same defect that causes human cases used a viral vector to induce production of fetal hemoglobin (HbF), which normally ceases to be produced shortly after birth. In humans, the use of hydroxyurea to stimulate the production of HbF temporarily alleviates sickle cell symptoms. The researchers demonstrated this treatment to be a more permanent means to increase therapeutic HbF production.[67]

A new gene therapy approach repaired errors in messenger RNA derived from defective genes. This technique has the potential to treat thalassaemia, cystic fibrosis and some cancers.[68]

Researchers created liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane.[69]

In 2003 a research team inserted genes into the brain for the first time. They used liposomes coated in a polymer called polyethylene glycol, which unlike viral vectors, are small enough to cross the bloodbrain barrier.[70]

Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced.[71]

Gendicine is a cancer gene therapy that delivers the tumor suppressor gene p53 using an engineered adenovirus. In 2003, it was approved in China for the treatment of head and neck squamous cell carcinoma.[25]

In March researchers announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease, a disease which affects myeloid cells and damages the immune system. The study is the first to show that gene therapy can treat the myeloid system.[72]

In May a team reported a way to prevent the immune system from rejecting a newly delivered gene.[73] Similar to organ transplantation, gene therapy has been plagued by this problem. The immune system normally recognizes the new gene as foreign and rejects the cells carrying it. The research utilized a newly uncovered network of genes regulated by molecules known as microRNAs. This natural function selectively obscured their therapeutic gene in immune system cells and protected it from discovery. Mice infected with the gene containing an immune-cell microRNA target sequence did not reject the gene.

In August scientists successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells.[74]

In November researchers reported on the use of VRX496, a gene-based immunotherapy for the treatment of HIV that uses a lentiviral vector to deliver an antisense gene against the HIV envelope. In a phase I clinical trial, five subjects with chronic HIV infection who had failed to respond to at least two antiretroviral regimens were treated. A single intravenous infusion of autologous CD4 T cells genetically modified with VRX496 was well tolerated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts. All five patients had stable or increased immune response to HIV antigens and other pathogens. This was the first evaluation of a lentiviral vector administered in a US human clinical trial.[75][76]

In May researchers announced the first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23-year-old British male, Robert Johnson, in early 2007.[77]

Leber’s congenital amaurosis is an inherited blinding disease caused by mutations in the RPE65 gene. The results of a small clinical trial in children were published in April.[11] Delivery of recombinant adeno-associated virus (AAV) carrying RPE65 yielded positive results. In May two more groups reported positive results in independent clinical trials using gene therapy to treat the condition. In all three clinical trials, patients recovered functional vision without apparent side-effects.[11][12][13][14]

In September researchers were able to give trichromatic vision to squirrel monkeys.[78] In November 2009, researchers halted a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder.[79]

An April paper reported that gene therapy addressed achromatopsia (color blindness) in dogs by targeting cone photoreceptors. Cone function and day vision were restored for at least 33 months in two young specimens. The therapy was less efficient for older dogs.[80]

In September it was announced that an 18-year-old male patient in France with beta-thalassemia major had been successfully treated.[81] Beta-thalassemia major is an inherited blood disease in which beta haemoglobin is missing and patients are dependent on regular lifelong blood transfusions.[82] The technique used a lentiviral vector to transduce the human -globin gene into purified blood and marrow cells obtained from the patient in June 2007.[83] The patient’s haemoglobin levels were stable at 9 to 10 g/dL. About a third of the hemoglobin contained the form introduced by the viral vector and blood transfusions were not needed.[83][84] Further clinical trials were planned.[85] Bone marrow transplants are the only cure for thalassemia, but 75% of patients do not find a matching donor.[84]

Cancer immunogene therapy using modified antigene, antisense/triple helix approach was introduced in South America in 2010/11 in La Sabana University, Bogota (Ethical Committee 14 December 2010, no P-004-10). Considering the ethical aspect of gene diagnostic and gene therapy targeting IGF-I, the IGF-I expressing tumors i.e. lung and epidermis cancers were treated (Trojan et al. 2016).[86][87]

In 2007 and 2008, a man (Timothy Ray Brown) was cured of HIV by repeated hematopoietic stem cell transplantation (see also allogeneic stem cell transplantation, allogeneic bone marrow transplantation, allotransplantation) with double-delta-32 mutation which disables the CCR5 receptor. This cure was accepted by the medical community in 2011.[88] It required complete ablation of existing bone marrow, which is very debilitating.

In August two of three subjects of a pilot study were confirmed to have been cured from chronic lymphocytic leukemia (CLL). The therapy used genetically modified T cells to attack cells that expressed the CD19 protein to fight the disease.[20] In 2013, the researchers announced that 26 of 59 patients had achieved complete remission and the original patient had remained tumor-free.[89]

Human HGF plasmid DNA therapy of cardiomyocytes is being examined as a potential treatment for coronary artery disease as well as treatment for the damage that occurs to the heart after myocardial infarction.[90][91]

In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia; it delivers the gene encoding for VEGF.[92][26] Neovasculogen is a plasmid encoding the CMV promoter and the 165 amino acid form of VEGF.[93][94]

The FDA approved Phase 1 clinical trials on thalassemia major patients in the US for 10 participants in July.[95] The study was expected to continue until 2015.[85]

In July 2012, the European Medicines Agency recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment used Alipogene tiparvovec (Glybera) to compensate for lipoprotein lipase deficiency, which can cause severe pancreatitis.[96] The recommendation was endorsed by the European Commission in November 2012[10][27][97][98] and commercial rollout began in late 2014.[99] Alipogene tiparvovec was expected to cost around $1.6 million per treatment in 2012,[100] revised to $1 million in 2015,[101] making it the most expensive medicine in the world at the time.[102] As of 2016[update], only one person had been treated with drug.[103]

In December 2012, it was reported that 10 of 13 patients with multiple myeloma were in remission “or very close to it” three months after being injected with a treatment involving genetically engineered T cells to target proteins NY-ESO-1 and LAGE-1, which exist only on cancerous myeloma cells.[22]

In March researchers reported that three of five adult subjects who had acute lymphocytic leukemia (ALL) had been in remission for five months to two years after being treated with genetically modified T cells which attacked cells with CD19 genes on their surface, i.e. all B-cells, cancerous or not. The researchers believed that the patients’ immune systems would make normal T-cells and B-cells after a couple of months. They were also given bone marrow. One patient relapsed and died and one died of a blood clot unrelated to the disease.[21]

Following encouraging Phase 1 trials, in April, researchers announced they were starting Phase 2 clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients[104] at several hospitals to combat heart disease. The therapy was designed to increase the levels of SERCA2, a protein in heart muscles, improving muscle function.[105] The FDA granted this a Breakthrough Therapy Designation to accelerate the trial and approval process.[106] In 2016 it was reported that no improvement was found from the CUPID 2 trial.[107]

In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 732 months. Three of the children had metachromatic leukodystrophy, which causes children to lose cognitive and motor skills.[108] The other children had Wiskott-Aldrich syndrome, which leaves them to open to infection, autoimmune diseases, and cancer.[109] Follow up trials with gene therapy on another six children with Wiskott-Aldrich syndrome were also reported as promising.[110][111]

In October researchers reported that two children born with adenosine deaminase severe combined immunodeficiency disease (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and that their immune systems were showing signs of full recovery. Another three children were making progress.[18] In 2014 a further 18 children with ADA-SCID were cured by gene therapy.[112] ADA-SCID children have no functioning immune system and are sometimes known as “bubble children.”[18]

Also in October researchers reported that they had treated six hemophilia sufferers in early 2011 using an adeno-associated virus. Over two years later all six were producing clotting factor.[18][113]

In January researchers reported that six choroideremia patients had been treated with adeno-associated virus with a copy of REP1. Over a six-month to two-year period all had improved their sight.[114][115] By 2016, 32 patients had been treated with positive results and researchers were hopeful the treatment would be long-lasting.[15] Choroideremia is an inherited genetic eye disease with no approved treatment, leading to loss of sight.

In March researchers reported that 12 HIV patients had been treated since 2009 in a trial with a genetically engineered virus with a rare mutation (CCR5 deficiency) known to protect against HIV with promising results.[116][117]

Clinical trials of gene therapy for sickle cell disease were started in 2014.[118][119] There is a need for high quality randomised controlled trials assessing the risks and benefits involved with gene therapy for people with sickle cell disease.[120]

In February LentiGlobin BB305, a gene therapy treatment undergoing clinical trials for treatment of beta thalassemia gained FDA “breakthrough” status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease.[121]

In March researchers delivered a recombinant gene encoding a broadly neutralizing antibody into monkeys infected with simian HIV; the monkeys’ cells produced the antibody, which cleared them of HIV. The technique is named immunoprophylaxis by gene transfer (IGT). Animal tests for antibodies to ebola, malaria, influenza, and hepatitis were underway.[122][123]

In March, scientists, including an inventor of CRISPR, Jennifer Doudna, urged a worldwide moratorium on germline gene therapy, writing “scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans” until the full implications “are discussed among scientific and governmental organizations”.[124][125][126][127]

In October, researchers announced that they had treated a baby girl, Layla Richards, with an experimental treatment using donor T-cells genetically engineered using TALEN to attack cancer cells. One year after the treatment she was still free of her cancer (a highly aggressive form of acute lymphoblastic leukaemia [ALL]).[128] Children with highly aggressive ALL normally have a very poor prognosis and Layla’s disease had been regarded as terminal before the treatment.[129]

In December, scientists of major world academies called for a moratorium on inheritable human genome edits, including those related to CRISPR-Cas9 technologies[130] but that basic research including embryo gene editing should continue.[131]

In April the Committee for Medicinal Products for Human Use of the European Medicines Agency endorsed a gene therapy treatment called Strimvelis[132][133] and the European Commission approved it in June.[134] This treats children born with adenosine deaminase deficiency and who have no functioning immune system. This was the second gene therapy treatment to be approved in Europe.[135]

In October, Chinese scientists reported they had started a trial to genetically modify T-cells from 10 adult patients with lung cancer and reinject the modified T-cells back into their bodies to attack the cancer cells. The T-cells had the PD-1 protein (which stops or slows the immune response) removed using CRISPR-Cas9.[136][137]

A 2016 Cochrane systematic review looking at data from four trials on topical cystic fibrosis transmembrane conductance regulator (CFTR) gene therapy does not support its clinical use as a mist inhaled into the lungs to treat cystic fibrosis patients with lung infections. One of the four trials did find weak evidence that liposome-based CFTR gene transfer therapy may lead to a small respiratory improvement for people with CF. This weak evidence is not enough to make a clinical recommendation for routine CFTR gene therapy.[138]

In February Kite Pharma announced results from a clinical trial of CAR-T cells in around a hundred people with advanced Non-Hodgkin lymphoma.[139]

In March, French scientists reported on clinical research of gene therapy to treat sickle-cell disease.[140]

In August, the FDA approved tisagenlecleucel for acute lymphoblastic leukemia.[141] Tisagenlecleucel is an adoptive cell transfer therapy for B-cell acute lymphoblastic leukemia; T cells from a person with cancer are removed, genetically engineered to make a specific T-cell receptor (a chimeric T cell receptor, or “CAR-T”) that reacts to the cancer, and are administered back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells. This is the first form of gene therapy to be approved in the United States. In October, a similar therapy called axicabtagene ciloleucel was approved for non-Hodgkin lymphoma.[142]

In December the results of using an adeno-associated virus with blood clotting factor VIII to treat nine haemophilia A patients were published. Six of the seven patients on the high dose regime increased the level of the blood clotting VIII to normal levels. The low and medium dose regimes had no effect on the patient’s blood clotting levels.[143][144]

In December, the FDA approved Luxturna, the first in vivo gene therapy, for the treatment of blindness due to Leber’s congenital amaurosis.[145] The price of this treatment was 850,000 US dollars for both eyes.[146][147]

Speculated uses for gene therapy include:

Athletes might adopt gene therapy technologies to improve their performance.[148] Gene doping is not known to occur, but multiple gene therapies may have such effects. Kayser et al. argue that gene doping could level the playing field if all athletes receive equal access. Critics claim that any therapeutic intervention for non-therapeutic/enhancement purposes compromises the ethical foundations of medicine and sports.[149]

Genetic engineering could be used to cure diseases, but also to change physical appearance, metabolism, and even improve physical capabilities and mental faculties such as memory and intelligence. Ethical claims about germline engineering include beliefs that every fetus has a right to remain genetically unmodified, that parents hold the right to genetically modify their offspring, and that every child has the right to be born free of preventable diseases.[150][151][152] For parents, genetic engineering could be seen as another child enhancement technique to add to diet, exercise, education, training, cosmetics, and plastic surgery.[153][154] Another theorist claims that moral concerns limit but do not prohibit germline engineering.[155]

Possible regulatory schemes include a complete ban, provision to everyone, or professional self-regulation. The American Medical Associations Council on Ethical and Judicial Affairs stated that “genetic interventions to enhance traits should be considered permissible only in severely restricted situations: (1) clear and meaningful benefits to the fetus or child; (2) no trade-off with other characteristics or traits; and (3) equal access to the genetic technology, irrespective of income or other socioeconomic characteristics.”[156]

As early in the history of biotechnology as 1990, there have been scientists opposed to attempts to modify the human germline using these new tools,[157] and such concerns have continued as technology progressed.[158][159] With the advent of new techniques like CRISPR, in March 2015 a group of scientists urged a worldwide moratorium on clinical use of gene editing technologies to edit the human genome in a way that can be inherited.[124][125][126][127] In April 2015, researchers sparked controversy when they reported results of basic research to edit the DNA of non-viable human embryos using CRISPR.[160][161] A committee of the American National Academy of Sciences and National Academy of Medicine gave qualified support to human genome editing in 2017[162][163] once answers have been found to safety and efficiency problems “but only for serious conditions under stringent oversight.”[164]

Regulations covering genetic modification are part of general guidelines about human-involved biomedical research. There are no international treaties which are legally binding in this area, but there are recommendations for national laws from various bodies.

The Helsinki Declaration (Ethical Principles for Medical Research Involving Human Subjects) was amended by the World Medical Association’s General Assembly in 2008. This document provides principles physicians and researchers must consider when involving humans as research subjects. The Statement on Gene Therapy Research initiated by the Human Genome Organization (HUGO) in 2001 provides a legal baseline for all countries. HUGOs document emphasizes human freedom and adherence to human rights, and offers recommendations for somatic gene therapy, including the importance of recognizing public concerns about such research.[165]

No federal legislation lays out protocols or restrictions about human genetic engineering. This subject is governed by overlapping regulations from local and federal agencies, including the Department of Health and Human Services, the FDA and NIH’s Recombinant DNA Advisory Committee. Researchers seeking federal funds for an investigational new drug application, (commonly the case for somatic human genetic engineering,) must obey international and federal guidelines for the protection of human subjects.[166]

NIH serves as the main gene therapy regulator for federally funded research. Privately funded research is advised to follow these regulations. NIH provides funding for research that develops or enhances genetic engineering techniques and to evaluate the ethics and quality in current research. The NIH maintains a mandatory registry of human genetic engineering research protocols that includes all federally funded projects.

An NIH advisory committee published a set of guidelines on gene manipulation.[167] The guidelines discuss lab safety as well as human test subjects and various experimental types that involve genetic changes. Several sections specifically pertain to human genetic engineering, including Section III-C-1. This section describes required review processes and other aspects when seeking approval to begin clinical research involving genetic transfer into a human patient.[168] The protocol for a gene therapy clinical trial must be approved by the NIH’s Recombinant DNA Advisory Committee prior to any clinical trial beginning; this is different from any other kind of clinical trial.[167]

As with other kinds of drugs, the FDA regulates the quality and safety of gene therapy products and supervises how these products are used clinically. Therapeutic alteration of the human genome falls under the same regulatory requirements as any other medical treatment. Research involving human subjects, such as clinical trials, must be reviewed and approved by the FDA and an Institutional Review Board.[169][170]

Gene therapy is the basis for the plotline of the film I Am Legend[171] and the TV show Will Gene Therapy Change the Human Race?.[172] In 1994, gene therapy was a plot element in The Erlenmeyer Flask, The X-Files’ first-season finale. It is also used in Stargate as a means of allowing humans to use Ancient technology.[173]

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Gene therapy – Wikipedia

Gene therapy – Mayo Clinic

Overview

Gene therapy involves altering the genes inside your body’s cells in an effort to treat or stop disease.

Genes contain your DNA the code that controls much of your body’s form and function, from making you grow taller to regulating your body systems. Genes that don’t work properly can cause disease.

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body’s ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.

Researchers are still studying how and when to use gene therapy. Currently, in the United States, gene therapy is available only as part of a clinical trial.

Gene therapy is used to correct defective genes in order to cure a disease or help your body better fight disease.

Researchers are investigating several ways to do this, including:

Gene therapy has some potential risks. A gene can’t easily be inserted directly into your cells. Rather, it usually has to be delivered using a carrier, called a vector.

The most common gene therapy vectors are viruses because they can recognize certain cells and carry genetic material into the cells’ genes. Researchers remove the original disease-causing genes from the viruses, replacing them with the genes needed to stop disease.

This technique presents the following risks:

The gene therapy clinical trials underway in the U.S. are closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research.

Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body.

Your specific procedure will depend on the disease you have and the type of gene therapy being used.

For example, in one type of gene therapy:

Viruses aren’t the only vectors that can be used to carry altered genes into your body’s cells. Other vectors being studied in clinical trials include:

The possibilities of gene therapy hold much promise. Clinical trials of gene therapy in people have shown some success in treating certain diseases, such as:

But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment, including:

Gene therapy continues to be a very important and active area of research aimed at developing new, effective treatments for a variety of diseases.

Explore Mayo Clinic studies testing new treatments, interventions and tests as a means to prevent, detect, treat or manage this disease.

Dec. 29, 2017

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Gene therapy – Mayo Clinic

What is Gene Therapy? – Learn.Genetics

Could the condition be corrected by adding one or a few functional genes?For you to even consider gene therapy, the answer must be “yes.” For instance, genetic disorders caused by mutations in single genes tend to be good candidates for gene therapy, while diseases involving many genes and environmental factors tend to be poor candidates.

Do you know which genes are involved?If you plan to treat a genetic flaw, you need to know which gene(s) to pursue. You must also have a DNA copy of the gene available in your laboratory.

Do you understand the biology of the disorder?To design the best possible approach, you need to learn all you can about how the gene factors into the disorder. For example, which tissues the disorder affects, what role the protein encoded by the gene plays within the cells of that tissue, and exactly how mutations in the gene affect the protein’s function.

Will adding a normal copy of the gene fix the problem in the affected tissue? Or could getting rid of the defective gene fix it?Sometimes when a gene is defective, no functional protein is being made from it. In cases like these, adding a functional copy of the gene could correct the problem. But sometimes a defective gene codes for a protein that starts doing something it shouldn’t or prevents another protein from doing its job. In order to correct the problem, you would need to get rid of the misbehaving protein.

Can you deliver the gene to cells of the affected tissue?The answer will come from several pieces of information, including the tissue’s accessibility and molecular signatures.

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What is Gene Therapy? – Learn.Genetics

Gene Therapy Retrovirus Vectors Explained

A retrovirus is any virus belonging to the viral family Retroviridae. All The genetic material in retroviruses is in the form of RNA molecules, while the genetic material of their hosts is in the form of DNA. When a retrovirus infects a host cell, it will introduce its RNA together with some enzymes into the cell. This RNA molecule from the retrovirus must produce a DNA copy from its RNA molecule before it can be considered part of the genetic material of the host cell. Retrovirus genomes commonly contain these three open reading frames that encode for proteins that can be found in the mature virus. Group-specific antigen (gag) codes for core and structural proteins of the virus, polymerase (pol) codes for reverse transcriptase, protease and integrase, and envelope (env) codes for the retroviral coat proteins (see figure 1). Figure 1. Genome organisation of retroviruses.

The process of producing a DNA copy from an RNA molecule is termed reverse transcription. It is carried out by one of the enzymes carried in the virus, called reverse transcriptase. After this DNA copy is produced and is free in the nucleus of the host cell, it must be incorporated into the genome of the host cell. That is, it must be inserted into the large DNA molecules in the cell (the chromosomes). This process is done by another enzyme carried in the virus called integrase (see figure 2).

Now that the genetic material of the virus is incorporated and has become part of the genetic material of the host cell, we can say that the host cell is now modified to contain a new gene. If this host cell divides later, its descendants will all contain the new genes. Sometimes the genes of the retrovirus do not express their information immediately.

Retroviral vectors are created by removal op the retroviral gag, pol, and env genes. These are replaced by the therapeutic gene. In order to produce vector particles a packaging cell is essential. Packaging cell lines provide all the viral proteins required for capsid production and the virion maturation of the vector. These packaging cell lines have been made so that they contain the gag, pol and env genes. Early packaging cell lines contained replication competent retroviral genomes and a single recombination event between this genome and the retroviral DNA vector could result in the production of a wild type virus. Following insertion of the desired gene into in the retroviral DNA vector, and maintainance of the proper packaging cell line, it is now a simple matter to prepare retroviral vectors (see figure 3).

One of the problems of gene therapy using retroviruses is that the integrase enzyme can insert the genetic material of the virus in any arbitrary position in the genome of the host. If genetic material happens to be inserted in the middle of one of the original genes of the host cell, this gene will be disrupted (insertional mutagenesis). If the gene happens to be one regulating cell division, uncontrolled cell division (i.e., cancer) can occur. This problem has recently begun to be addressed by utilizing zinc finger nucleases or by including certain sequences such as the beta-globin locus control region to direct the site of integration to specific chromosomal sites.

Gene therapy trials to treat severe combined immunodeficiency (SCID) were halted or restricted in the USA when leukemia was reported in three of eleven patients treated in the French X-linked SCID (X-SCID) gene therapy trial. Ten X-SCID patients treated in England have not presented leukemia to date and have had similar success in immune reconstitution. Gene therapy trials to treat SCID due to deficiency of the Adenosine Deaminase (ADA) enzyme continue with relative success in the USA, Italy and Japan.

As a reaction to the adverse events in the French X-SCID gene therapy trial, the Recombinant DNA Advisory Committee (RAC) sent a letter to Principal Investigators Conveying RAC Recommendations in 2003. In addition, the RAC published conclusions and recommendations of the RAC Gene Transfer Safety Symposium in 2005. A joint working party of the Gene Therapy Advisory Committee and the Committee on Safety of Medicines (CSM) in the UK lead to the publication of an updated recommendations of the GTAC/CSM working party on retroviruses in 2005.

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Gene Therapy Retrovirus Vectors Explained

Gene Therapy Net – News, Conferences, Vectors, Literature …

Posted on: 18 July 2018, source: European CommissionThe EU’s Regulation on advanced therapies, is designed to ensure the free movement of advanced therapy products within Europe, to facilitate access to the EU market, and to foster the competitiveness of European companies in the field, while guaranteeing the highest level of health protection for patients. A Good Practice document on the assessment of GMO-related aspects in the context of clinical trials with human cells genetically modified has been developed by the national competent authorities and the Commission services.

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Gene Therapy Net – News, Conferences, Vectors, Literature …

A Stem Cell Transplant Let a Wheelchair-Bound Man Dance Again

Stand Up Guy

For 10 years, Roy Palmer had no feeling in his lower extremities. Two days after receiving a stem cell transplant, he cried tears of joy because he could feel a cramp in his leg.

The technical term for the procedure the British man underwent is hematopoietic stem cell transplantation (HSCT). And while risky, it’s offering new hope to people like Palmer, who found himself wheelchair-bound after multiple sclerosis (MS) caused his immune system to attack his nerves’ protective coverings.

Biological Reboot

Ever hear the IT troubleshooting go-to of turning a system off and on again to fix it? The HSCT process is similar, but instead of a computer, doctors attempt to reboot a patient’s immune system.

To do this, they first remove stem cells from the patient’s body. Then the patient undergoes chemotherapy, which kills the rest of their immune system. After that, the doctors use the extracted stem cells to reboot the patient’s immune system.

It took just two days for the treatment to restore some of the feeling in Palmer’s legs. Eventually, he was able to walk on his own and even dance. He told the BBC in a recent interview that he now feels like he has a second chance at life.

“We went on holiday, not so long ago, to Turkey. I walked on the beach,” said Palmer. “Little things like that, people do not realize what it means to me.”

Risk / Reward

Still, HSCT isn’t some miracle cure for MS. Though it worked for Palmer, that’s not always the case, and HSCT can also cause infections and infertility. The National MS Society still considers HSCT to be an experimental treatment, and the Food and Drug Administration has yet to approve the therapy in the U.S.

However, MS affects more than 2.3 million people, and if a stem cell transplant can help even some of those folks the way it helped Palmer, it’s a therapy worth exploring.

READ MORE: Walking Again After Ten Years With MS [BBC]

More on HCST: New Breakthrough Treatment Could “Reverse Disability” for MS Patients

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A Stem Cell Transplant Let a Wheelchair-Bound Man Dance Again

AI Dreamed Up These Nightmare Fuel Halloween Masks

Nightmare Fuel

Someone programmed an AI to dream up Halloween masks, and the results are absolute nightmare fuel. Seriously, just look at some of these things.

“What’s so scary or unsettling about it is that it’s not so detailed that it shows you everything,” said Matt Reed, the creator of the masks, in an interview with New Scientist. “It leaves just enough open for your imagination to connect the dots.”

A selection of masks featured on Reed’s twitter. Credit: Matt Reed/Twitter

Creative Horror

To create the masks, Reed — whose day job is as a technologist at a creative agency called redpepper — fed an open source AI tool 5,000 pictures of Halloween masks he sourced from Google Images. He then instructed the tool to generate its own masks.

The fun and spooky project is yet another sign that AI is coming into its own as a creative tool. Just yesterday, a portrait generated by a similar system fetched more than $400,000 at a prominent British auction house.

And Reed’s masks are evocative. Here at the Byte, if we looked through the peephole and saw one of these on a trick or treater, we might not open our door.

READ MORE: AI Designed These Halloween Masks and They Are Absolutely Terrifying [New Scientist]

More on AI-generated art: Generated Art Will Go on Sale Alongside Human-Made Works This Fall

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AI Dreamed Up These Nightmare Fuel Halloween Masks

Robot Security Guards Will Constantly Nag Spectators at the Tokyo Olympics

Over and Over

“The security robot is patrolling. Ding-ding. Ding-ding. The security robot is patrolling. Ding-ding. Ding-ding.”

That’s what Olympic attendees will hear ad nauseam when they step onto the platforms of Tokyo’s train stations in 2020. The source: Perseusbot, a robot security guard Japanese developers unveiled to the press on Thursday.

Observe and Report

According to reporting by Kyodo News, the purpose of the AI-powered Perseusbot is to lower the burden on the stations’ staff when visitors flood Tokyo during the 2020 Olympics.

The robot is roughly 5.5 feet tall and equipped with security cameras that allow it to note suspicious behaviors, such as signs of violence breaking out or unattended packages, as it autonomous patrols the area. It can then alert security staff to the issues by sending notifications directly to their smart phones.

Prior Prepration

Just like the athletes who will head to Tokyo in 2020, Perseusbot already has a training program in the works — it’ll patrol Tokyo’s Seibu Shinjuku Station from November 26 to 30. This dry run should give the bot’s developers a chance to work out any kinks before 2020.

If all goes as hoped, the bot will be ready to annoy attendees with its incessant chant before the Olympic torch is lit. And, you know, keep everyone safe, too.

READ MORE: Robot Station Security Guard Unveiled Ahead of 2020 Tokyo Olympics [Kyodo News]

More robot security guards: Robot Security Guards Are Just the Beginning

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Robot Security Guards Will Constantly Nag Spectators at the Tokyo Olympics

People Would Rather a Self-Driving Car Kill a Criminal Than a Dog

Snap Decisions

On first glance, a site that collects people’s opinions about whose life an autonomous car should favor doesn’t tell us anything we didn’t already know. But look closer, and you’ll catch a glimpse of humanity’s dark side.

The Moral Machine is an online survey designed by MIT researchers to gauge how the public would want an autonomous car to behave in a scenario in which someone has to die. It asks questions like: “If an autonomous car has to choose between killing a man or a woman, who should it kill? What if the woman is elderly but the man is young?”

Essentially, it’s a 21st century update on the Trolley Problem, an ethical thought experiment no doubt permanently etched into the mind of anyone who’s seen the second season of “The Good Place.”

Ethical Dilemma

The MIT team launched the Moral Machine in 2016, and more than two million people from 233 countries participated in the survey — quite a significant sample size.

On Wednesday, the researchers published the results of the experiment in the journal Nature, and they really aren’t all that surprising: Respondents value the life of a baby over all others, with a female child, male child, and pregnant woman following closely behind. Yawn.

It’s when you look at the other end of the spectrum — the characters survey respondents were least likely to “save” — that you’ll see something startling: Survey respondents would rather the autonomous car kill a human criminal than a dog.

moral machine
Image Credit: MIT

Ugly Reflection

While the team designed the survey to help shape the future of autonomous vehicles, it’s hard not to focus on this troubling valuing of a dog’s life over that of any human, criminal or not. Does this tell us something important about how society views the criminal class? Reveal that we’re all monsters when hidden behind the internet’s cloak of anonymity? Confirm that we really like dogs?

The MIT team doesn’t address any of these questions in their paper, and really, we wouldn’t expect them to — it’s their job to report the survey results, not extrapolate some deeper meaning from them. But whether the Moral Machine informs the future of autonomous vehicles or not, it’s certainly held up a mirror to humanity’s values, and we do not like the reflection we see.

READ MORE: Driverless Cars Should Spare Young People Over Old in Unavoidable Accidents, Massive Survey Finds [Motherboard]

More on the Moral Machine: MIT’s “Moral Machine” Lets You Decide Who Lives & Dies in Self-Driving Car Crashes

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People Would Rather a Self-Driving Car Kill a Criminal Than a Dog

Scientists Say New Material Could Hold up an Actual Space Elevator

Space Elevator

It takes a lot of energy to put stuff in space. That’s why one longtime futurist dream is a “space elevator” — a long cable strung between a geostationary satellite and the Earth that astronauts could use like a dumbwaiter to haul stuff up into orbit.

The problem is that such a system would require an extraordinarily light, strong cable. Now, researchers from Beijing’s Tsinghua University say they’ve developed a carbon nanotube fiber so sturdy and lightweight that it could be used to build an actual space elevator.

Going Up

The researchers published their paper in May, but it’s now garnering the attention of their peers. Some believe the Tsinghua team’s material really could lead to the creation of an elevator that would make it cheaper to move astronauts and materials into space.

“This is a breakthrough,” colleague Wang Changqing, who studies space elevators at Northwestern Polytechnical University, told the South China Morning Post.

Huge If True

There are still countless galling technical problems that need to be overcome before a space elevator would start to look plausible. Wang pointed out that it’d require tens of thousands of kilometers of the new material, for instance, as well as a shield to protect it from space debris.

But the research brings us one step closer to what could be a true game changer: a vastly less expensive way to move people and spacecraft out of Earth’s gravity.

READ MORE: China Has Strongest Fibre That Can Haul 160 Elephants – and a Space Elevator? [South China Morning Post]

More on space elevators: Why Space Elevators Could Be the Future of Space Travel

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Scientists Say New Material Could Hold up an Actual Space Elevator

Report Identifies China as the Source of Ozone-Destroying Emissions

Emissions Enigma

For years, a mystery puzzled environmental scientists. The world had banned the use of many ozone-depleting compounds in 2010. So why were global emission levels still so high?

The picture started to clear up in June. That’s when The New York Times published an investigation into the issue. China, the paper claimed, was to blame for these mystery emissions. Now it turns out the paper was probably right to point a finger.

Accident or Incident

In a paper published recently in the journal Geophysical Research Letters, an international team of researchers confirms that eastern China is the source of at least half of the 40,000 tonnes of carbon tetrachloride emissions currently entering the atmosphere each year.

They figured this out using a combination of ground-based and airborne atmospheric concentration data from near the Korean peninsula. They also relied on two models that simulated how the gases would move through the atmosphere.

Though they were able to narrow down the source to China, the researchers weren’t able to say exactly who’s breaking the ban and whether they even know about the damage they’re doing.

Pinpoint

“Our work shows the location of carbon tetrachloride emissions,” said co-author Matt Rigby in a press release. “However, we don’t yet know the processes or industries that are responsible. This is important because we don’t know if it is being produced intentionally or inadvertently.”

If we can pinpoint the source of these emissions, we can start working on stopping them and healing our ozone. And given that we’ve gone nearly a decade with minimal progress on that front, there’s really no time to waste.

READ MORE: Location of Large ‘Mystery’ Source of Banned Ozone Depleting Substance Uncovered [University of Bristol]

More on carbon emissions: China Has (Probably) Been Pumping a Banned Gas Into the Atmosphere

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Report Identifies China as the Source of Ozone-Destroying Emissions

An AI Conference Refusing a Name Change Highlights a Tech Industry Problem

Name Game

There’s a prominent artificial intelligence conference that goes by the suggestive acronym NIPS, which stands for “Neural Information Processing Systems.”

After receiving complaints that the acronym was alienating to women, the conference’s leadership collected suggestions for a new name via an online poll, according to WIRED. But the conference announced Monday that it would be sticking with NIPS all the same.

Knock It Off

It’s convenient to imagine that this acronym just sort of emerged by coincidence, but let’s not indulge in that particular fantasy.

It’s more likely that tech geeks cackled maniacally when they came up with the acronym, and the refusal to do better even when people looking up the conference in good faith are bombarded with porn is a particularly telling failure of the AI research community.

Small Things Matter

This problem goes far beyond a silly name — women are severely underrepresented in technology research and even more so when it comes to artificial intelligence. And if human decency — comforting those who are regularly alienated by the powers that be — isn’t enough of a reason to challenge the sexist culture embedded in tech research, just think about what we miss out on.

True progress in artificial intelligence cannot happen without a broad range of diverse voices — voices that are silenced by “locker room talk” among an old boy’s club. Otherwise, our technological development will become just as stuck in place as our cultural development often seems to be.

READ MORE: AI RESEARCHERS FIGHT OVER FOUR LETTERS: NIPS [WIRED]

More on Silicon Valley sexism: The Tech Industry’s Gender Problem Isn’t Just Hurting Women

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An AI Conference Refusing a Name Change Highlights a Tech Industry Problem

Scientists Are Hopeful AI Could Help Predict Earthquakes

Quake Rate

Earlier this year, I interviewed U.S. Geological Survey geologist Annemarie Baltay for a story about why it’s incredibly difficult to predict earthquakes.

“We don’t use that ‘p word’ — ‘predict’ — at all,” she told me. “Earthquakes are chaotic. We don’t know when or where they’ll occur.”

Neural Earthwork

That could finally be starting to change, according to a fascinating feature in The New York Times.

By feeding seismic data into a neural network — a type of artificial intelligence that learns to recognize patterns by scrutinizing examples — researchers say they can now predict moments after a quake strikes how far its aftershocks will travel.

And eventually, some believe, they’ll be able to listen to signals from fault lines and predict when an earthquake will strike in the first place.

Future Vision

But like Baltay, some researchers aren’t convinced we’ll ever be able to predict earthquakes.University of Tokyo seismologist Robert Geller told the Times that until an algorithm actually predicts an upcoming quake, he’ll remain skeptical.

“There are no shortcuts,” he said. “If you cannot predict the future, then your hypothesis is wrong.”

READ MORE: A.I. Is Helping Scientist Predict When and Where the Next Big Earthquake Will Be [The New York Times]

More on earthquake AI: A New AI Detected 17 Times More Earthquakes Than Traditional Methods

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Scientists Are Hopeful AI Could Help Predict Earthquakes

This AI Lie Detector Flags Falsified Police Reports

Minority Report

Imagine this: You file a police report, but back at the station, they feed it into an algorithm — and it accuses you of lying, as though it had somehow looked inside your brain.

That might sound like science fiction, but Spain is currently rolling out a very similar program, called VeriPol, in many of its police stations. VeriPol’s creators say that when it flags a report as false, it turns out to be correct more than four-fifths of the time.

Lie Detector

VeriPol is the work of researchers at Cardiff University and Charles III University of Madrid.

In a paper published earlier this year in the journal Knowledge-Based Systems, they describe how they trained the lie detector with a data set of more than 1,000 robbery reports — including a number that police identified as false — to identify subtle signs that a report wasn’t true.

Thought Crime

In pilot studies in Murcia and Malaga, Quartz reported, further investigation showed that the algorithm was correct about 83 percent of the time that it suspected a report was false.

Still, the project raises uncomfortable questions about allowing algorithms to act as lie detectors. Fast Company reported earlier this year that authorities in the United States, Canada, and the European Union are testing a separate system called AVATAR that they want to use to collect biometric data about subjects at border crossings — and analyze it for signs that they’re not being truthful.

Maybe the real question isn’t whether the tech works, but whether we want to permit authorities to act upon what’s essentially a good — but not perfect — assumption that someone is lying.

READ MORE: Police Are Using Artificial Intelligence to Spot Written Lies [Quartz]

More on lie detectors: Stormy Daniels Took a Polygraph. What Do We Do With the Results?

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This AI Lie Detector Flags Falsified Police Reports

These Bacteria Digest Food Waste Into Biodegradable Plastic

Factory Farm

Plastics have revolutionized manufacturing, but they’re still terrible for the environment.

Manufacturing plastics is an energy-intensive slog that ends in mountains of toxic industrial waste and greenhouse gas emissions. And then the plastic itself that we use ends up sitting in a garbage heap for thousands of years before it biodegrades.

Scientists have spent years investigating ways to manufacture plastics without ruining the planet, and a Toronto biotech startup called Genecis says it’s found a good answer: factories where vats of bacteria digest food waste and use it to form biodegradable plastic in their tiny microbial guts.

One-Two Punch

The plastic-pooping bacteria stand to clean up several kinds of pollution while churning out usable materials, according to Genecis.

That’s because the microbes feed on waste food or other organic materials — waste that CBC reported gives off 20 percent of Canada’s methane emissions as it sits in landfills.

Then What?

The plastic that the little buggers produce isn’t anything new. It’s called PHA and it’s used in anything that needs to biodegrade quickly, like those self-dissolving stitches. What’s new here is that food waste is much cheaper than the raw materials that usually go into plastics, leading Genecis to suspect it can make the same plastics for 40 percent less cost.

There are a lot of buzzworthy new alternative materials out there, but with a clear environmental and financial benefit, it’s possible these little bacteria factories might be here to stay.

READ MORE: Greener coffee pods? Bacteria help turn food waste into compostable plastic [CBC]

More on cleaning up plastics: The EU Just Voted to Completely ban Single-Use Plastics

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These Bacteria Digest Food Waste Into Biodegradable Plastic

You Can Now Preorder a $150,000 Hoverbike

Please, Santa?

It’s never too early to start writing your Christmas wish list, right? Because we know what’s now at the top of ours: a hoverbike.

We’ve had our eyes on Hoversurf’s Scorpion-3 since early last year — but now, the Russian drone start-up is accepting preorders on an updated version of the vehicle.

Flying Bike

The S3 2019 is part motorcycle and part quadcopter. According to the Hoversurf website, the battery-powered vehicle weighs 253 pounds and has a flight time of 10 to 25 minutes depending on operator weight. Its maximum legal speed is 60 mph — though as for how fast the craft can actually move, that’s unknown. Hoversurf also notes that the vehicle’s “safe flight altitude” is 16 feet, but again, we aren’t sure how high it can actually soar.

What we do know: The four blades that provide S3 with its lift spin at shin level, and while this certainly looks like it would be a safety hazard, the U.S. Department of Transportation’s Federal Aviation Administration approved the craft for legal use as an ultralight vehicle in September.

That means you can only operate an S3 for recreational or sports purposes — but you can’t cruise to work on your morning commute.

Plummeting Bank Account

You don’t need a pilot’s license to operate an S3, but you will need a decent amount of disposable income — the Star Wars-esque craft will set you back $150,000.

If that number doesn’t cause your eyes to cross, go ahead and slap down the $10,000 deposit needed to claim a spot in the reservation queue. You’ll then receive an email when it’s time to to place your order. You can expect to receive your S3 2019 two to six months after that, according to the company website.

That means there’s a pretty good chance you won’t be able to hover around your front yard this Christmas morning, but a 2019 jaunt is a genuine possibility.

READ MORE: For $150,000 You Can Now Order Your Own Hoverbike [New Atlas]

More on Hoversurf: Watch the World’s First Rideable Hoverbike in Flight

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You Can Now Preorder a $150,000 Hoverbike

FBI’s Tesla Criminal Probe Reportedly Centers on Model 3 Production

Ups and Downs

Can we please get off Mr. Musk’s Wild Ride now? We don’t know how much more of this Tesla rollercoaster we can take.

In 2018 alone, Elon Musk’s clean energy company has endured a faulty flufferbot, furious investors, and an SEC probe and settlement. But there was good news, too. Model 3 deliveries reportedly increased, and just this week, we found out that Tesla had a historic financial quarter, generating $312 million in profit.

And now we’re plummeting again.

Closing In

On Friday, The Wall Street Journal reported that the Federal Bureau of Investigation (FBI) is deepening a criminal probe into whether Tesla “misstated information about production of its Model 3 sedans and misled investors about the company’s business going back to early 2017.”

We’ve known about the FBI’s Tesla criminal probe since September 18, but this is the first report confirming that Model 3 production is at the center of the investigation.

According to the WSJ’s sources, FBI agents have been reaching out to former Tesla employees in recent weeks to ask if they’d be willing to testify in the criminal case, though no word yet on whether any have agreed.

Casual CEO

We might be having trouble keeping up with these twists and turns, but Musk seems to be taking the FBI’s Tesla criminal probe all in stride — he spent much of Friday afternoon joking around with his Twitter followers about dank memes.

Clearly he has the stomach for this, but it’d be hard to blame any Tesla investors for deciding they’d had enough.

READ MORE: Tesla Faces Deepening Criminal Probe Over Whether It Misstated Production Figures [The Wall Street Journal]

More on Tesla: Elon Musk Says Your Tesla Will Earn You Money While You Sleep

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FBI’s Tesla Criminal Probe Reportedly Centers on Model 3 Production


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