Using mutant protein, CHOP hematologist safely removes unwanted antibodies, reverses hemophilia in dog model of bleeding disease

Using gene therapy to produce a mutant human protein with unusually high blood-clotting power, scientists have successfully treated dogs with the bleeding disorder hemophilia, without triggering an unwanted immune response. In addition, the "turbocharged" clotting factor protein eliminated pre-existing antibodies that often weaken conventional treatments for people with hemophilia.

"Our findings may provide a new approach to gene therapy for hemophilia and perhaps other genetic diseases that have similar complications from inhibiting antibodies," said the study leader, Valder R. Arruda, M.D., Ph.D., a hematology researcher at The Children's Hospital of Philadelphia (CHOP).

Arruda and colleagues published their animal study results in the print edition of Blood on March 5.

Hemophilia is an inherited bleeding disorder that famously affected European royal families descended from Queen Victoria. Most commonly occurring in two types, hemophilia A and hemophilia B, the disease impairs the blood's ability to clot, sometimes fatally. When not fatal, severe hemophilia causes painful, often disabling internal bleeding and joint damage.

Doctors treat hemophilia with frequent intravenous infusions of blood clotting proteins called clotting factors, but these treatments are expensive and time-consuming. Moreover, some patients develop inhibiting antibodies that negate the effectiveness of the infusions.

For more than two decades, many research teams, including at CHOP, have investigated gene therapy strategies that deliver DNA sequences carrying genetic code to produce clotting factor in patients. However, this approach has been frustrated by the body's immune response against vectors--the non-disease-causing viruses used to carry the DNA. Those responses, which defeated initial benefits seen in experimental human gene therapy, were dose-dependent: higher amounts of vectors caused more powerful immune responses.

Arruda and colleagues therefore investigated gene therapy that used lower dosages of vector (adeno-associated viral-8 vector, or AAV-8 vector) to produce a more potent clotting factor--a variant protein called FIX-Padua.

Arruda was part of a scientific team in 2009 that discovered FIX-Padua in a young Italian man who had thrombosis, excessive clotting that can dangerously obstruct blood vessels. A mutation produced the mutant clotting factor, called FIX-Padua, named after the patient's city of residence. This was the first mutation in the factor IX gene found to cause thrombosis. All previously discovered FIX mutations lead to hemophilia, the opposite of thrombosis.

FIX-Padua is hyperfunctional--it clots blood 8 to 12 times more strongly than normal, wild-type factor IX. In the current study, the researchers thus needed to strike a balance--to relieve severe hemophilia in dogs, by using a dose strong enough to allow clotting, but not enough to cause thrombosis or stimulate immune reactions. "Our ultimate goal is to translate this approach to humans," said Arruda, "by adapting this variant protein found in one patient to benefit other patients with the opposite disease."

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Study bolsters 'turbocharged' protein as a promising tool in hemophilia gene therapy

Gene-editing companies say research on altering the DNA of human reproductive cells is dangerous and unethical.

Officials of a biotechnology industry group have called for a voluntary moratorium on using new DNA-editing techniques to change the genetic characteristics of human embryos in laboratory research.

In an editorial published today by the journal Nature, Edward Lanphier, CEO of the biotechnology company Sangamo Biosciences, and four colleagues write that scientists should agree not to modify the DNA of human reproductive cells because it raises safety and ethical risks including the danger of unpredictable effects on future generations.

New gene-editing techniques, in particular one called CRISPR, have given scientists powerful and useful new ways to swap and change DNA letters inside of living cells for the first time (see Genome Surgery).

Recently, some scientific teams have started to study whether CRISPR would be able to correct disease genes in future generations of peoplefor instance, by repairing genes during in vitro fertilization, or in eggs or sperm. The idea of such germ line modification would be to install healthy versions of genes, which children would be born with.

The emergence of active research around germ-line editing, which is taking place in China, at Harvard University, and at a publicly traded biotechnology company called OvaScience, were described last week by MIT Technology Review (see Engineering the Perfect Baby).

But the idea of using editing technology to improve children is as controversial as it is medically powerful. In their editorial, Lanphier, whose coauthors include Fyodor Urnov, co-developer of a different gene-editing system, raise the concern that such techniques might be exploited for non-therapeutic modifications. That could mean, for instance, changing the physical traits of children.

The availability of technology to carry out genetic engineering in human germ-line cells is driving intense debate in scientific circles and may eventually become a legal issue in the United States and other countries.

The authors call for a cessation of basic research is unusual and likely to be opposed by scientists as an intrusion on the quest for scientific knowledge.

George Church, a professor at Harvard Medical School whose laboratory studies CRISPR and germ-line editing, says a voluntary moratorium would be weak compared with existing regulations that nearly all countries impose on the use of new medical technologies until they are proven safe and effective in animals or human [tests]. Church was referring to rules governing the birth of actual gene-edited children, not basic research.

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Industry Body Calls for Gene-Editing Moratorium

FRESNO, Calif. (KFSN) --

Six-year-old Andy Moorhead is learning how to read. But instead of using his eyes, he's using his fingers. Andy told ABC30, "Well, I read the letters with my fingers."

Andy is blind. Andy's Mother, Heather Ingram-Moorhead explained, "He was around nine months, and we started to notice his eyes were twitching."

Andy has leber congenital amaurosis, or LCA. It's the most common type of childhood blindness and is caused by genetic mutations.

"It is just very hard. It's taken us a while to really understand the condition and do everything to help Andy," Heather told ABC30.

Andy's whole family is hands-on. Even his sister Valerie gives him guidance. But despite their efforts, his mom says gene therapy is their only hope.

University of Florida scientist Shannon E. Boye, PhD, is using a $900,000 grant to perfect a gene therapy that could restore vision.

"It's not an attempt just to slow the progression of the disease. It's actually an attempt to halt the progression and make the patient better by delivering them the gene they don't have," Boye told ABC30.

Boye says the therapy has worked in animals. "We're able to show, via what's called an electra retinal gram, that the retinal function has been restored to the mice," she explained.

Gene therapy is still an investigational treatment with risks and only available for those in a clinical trial. Right now there are hundreds of studies underway to treat conditions like LCA, cancer and HIV. It's hope that one day Andy could put down his cane and see his family for the first time.

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Gene therapy: Hope for the blind?

An effective vaccine for HIV has eluded researchers for several decades, due to the pathogen's infamous shape-shifting abilities.

Even though researchers have identified certain broadly neutralizing antibodies that can conquer multiple strains of the human immunodeficiency virus, many strains of rapidly mutating HIV remain resistant to the these super antibodies.

In recent years however,researches have proposed a new method of battling the virus that involves gene therapy.

Instead of using a vaccine to stimulate the body's own immune system, so that it produces HIV antibodies, scientists are bypassing the immune system entirely.

In experiments involving rats and monkeys, the researchers have used non-life-threatening viruses to alter the animals' genome so that its cells produce designer molecules capable of neutralizing HIV.

In a paper published Wednesday in the journal Nature, a team of researchers said they had used the technique to protect rhesus macaques from repeated intravenous injections of a SHIV, a combination of simian immunodeficiency virus and humanimmunodeficiency virus.

The technique, researchers said, "can function like an effective HIV-1 vaccine." (HIV-1 is the main family of the virus, and accounts for most infections worldwide.)

When HIV enters the body, it attacks specific immune cells. As the virus copies itself over and over, and kills more and more host cells, the immune system grows progressively weaker. If left untreated, this progressive weakening will give rise to AIDS.

In most cases, the HIV virus begins its attack by latching onto two separate protein structures on the surface of its target white blood cells. One of these structures is called CD4, and the other is called CCR5.

In the Nature study, researchers set out to engineer an antibody-like molecule that would mimic both of these proteins, so that it would act as decoy of sorts for the virus. Instead of latching onto a host cell, HIV would latch onto a specially enhanced protein molecule, or eCD4-Ig, that was released by the cell.

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With genetic engineering, scientists use decoy molecule to trick HIV

An effective vaccine for HIV has eluded researchers for several decades, due to the pathogen's infamous shape-shifting abilities.

Even though researchers have identified certain broadly neutralizing antibodies that can conquer multiple strains of the human immunodeficiency virus, many strains of rapidly mutating HIV remain resistant to the these super antibodies.

In recent years however,researches have proposed a new method of battling the virus that involves gene therapy.

Instead of using a vaccine to stimulate the body's own immune system, so that it produces HIV antibodies, scientists are bypassing the immune system entirely.

In experiments involving rats and monkeys, the researchers have used non-life-threatening viruses to alter the animals' genome so that its cells produce designer molecules capable of neutralizing HIV.

In a paper published Wednesday in the journal Nature, a team of researchers said they had used the technique to protect rhesus macaques from repeated intravenous injections of a SHIV, a combination of simian immunodeficiency virus and humanimmunodeficiency virus.

The technique, researchers said, "can function like an effective HIV-1 vaccine." (HIV-1 is the main family of the virus, and accounts for most infections worldwide.)

When HIV enters the body, it attacks specific immune cells. As the virus copies itself over and over, and kills more and more host cells, the immune system grows progressively weaker. If left untreated, this progressive weakening will give rise to AIDS.

In most cases, the HIV virus begins its attack by latching onto two separate protein structures on the surface of its target white blood cells. One of these structures is called CD4, and the other is called CCR5.

In the Nature study, researchers set out to engineer an antibody-like molecule that would mimic both of these proteins, so that it would act as decoy of sorts for the virus. Instead of latching onto a host cell, HIV would latch onto a specially enhanced protein molecule, or eCD4-Ig, that was released by the cell.

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A new twist on HIV vaccines shows results in monkeys, study says

Paying for gene therapy are annuities the next big thing
Paying for gene therapy are annuities the next big thing.

By: Reuters-BBC-Aljazeera

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Paying for gene therapy are annuities the next big thing - Video

Home Books Author Jeanne Ryan discusses Charisma and gene therapy

Author Jeanne Ryan talks with Hypable about her book Charisma and whether or not she would take her fictional drug if given the chance.

Charisma follows the story of Aislyn, a painfully shy junior in high school who suffers from social anxiety. Speaking up in class and trying to have fun with her friends at a party are well outside her comfort zone. She finds out about a new underground gene therapy treatment in the form of a drug called Charisma. Aislyns personality completely changes, and suddenly shes exactly the person she wanted to be all along.

But theres one problem. The therapy appears to be contagious and deadly. Worse yet, the doctor who gave her and a select number of other patients the drug suddenly disappears. Read our review.

Tell us 5 random facts about yourself.

Two of my brothers have the same name, kind of by accident. Just after my mom gave birth to her sixth kid in less than seven years, the nurse asked the name of her baby. Thinking the nurse meant the baby at home, my sleep-deprived mom gave her his name, and it ended up on the birth certificate.

I could swear that when I was a kid a certain soup company ran a commercial where the main lyric in its jingle was Constipation got you down? However, to this day, I have never been able to find anyone else who remembers this.

Until more recently than I care to admit, I thought narwhals were fictional. (In my defense, they look totally fake in photos!)

Id love to go to Burning Man one day.

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Author Jeanne Ryan discusses Charisma and gene therapy

S75RS04 Gene Therapy
Science Technology ITV Schools circa 1986.

By: Lammas Science

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S75RS04 Gene Therapy - Video

The Gene Therapy Plan: Taking Control of Your Genetic Destiny with Diet and Lifestyle
By Mitchell L. Gaynor MD (Author), Mehmet C. Oz M.D. (Foreword) http://genechanger.com/gene-therapy-plan-taking-control-genetic-destiny-diet-lifestyle/ From ...

By: Mitchell Gaynor M.D.

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The Gene Therapy Plan: Taking Control of Your Genetic Destiny with Diet and Lifestyle - Video

Cystic Fibrosis Gene Therapy
A short animation to briefly explain the research being conducted at the Gene Therapy Department of the Adelaide Women #39;s and Children #39;s Hospital. Donations c...

By: WCH Gene Therapy

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Cystic Fibrosis Gene Therapy - Video

Advances in gene therapy and the deepening understanding of cancer will see the oft-fatal disease becoming treatable in two decades, said cancer researcher Inder M. Verma.

Cancer mutations are being exposed cancer is in retreat through a combination of surgery, radiation, chemotherapy, molecular and genetic therapy, cancer will become a chronic disease rather than a terminal one, said Dr. Verma, a professor in Laboratory of Genetics at the Salk Institute for Biological Studies, at the Infosys Science Foundation Lecture at the National Centre for Biological Sciences here on Wednesday.

His optimism was elaborated through an intriguing cat-and-mouse game that played out for over five years of research into the Glioblastomas multiforme (GBM), a lethal form of brain cancer that kills the patient within 14 months.

Understanding GBM was critical as relapse, even after surgery or treatment, was certainty, said Dr. Verma.

The researchers at the Salk Institute developed a novel genetic technique to switch on genes in around five cells of a mouse brain to make them into cancer cells. The cells grew to all parts of the brain, but more importantly, they started to exhibit stem cell characteristics, said Dr. Verma.

Unlike the normal cell, a stem cell can divide into specialised cells a phenomenon that explains the resurgent ability of the GBM cancer. Even if you surgically remove the tumour, one cell is enough to recreate the cancer again, he explained.

Using gene therapy, the team of scientists attempted to block this ability as well as use drugs to block blood supply to the cancer cell. While the tumour did become smaller, it became even more invasive. Though the treatment did not work, the cancer cell did reveal the genes responsible for its invasiveness.

We began to genetically cut out the cancers invasiveness, and for the first time, experiments showed GBM cancer could be controlled This is an exciting area that can be possibly used to treat other forms of cancer, said Dr. Verma.

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Cancer set to become treatable: expert

Walking barefoot on sand "felt like walking on glass" for Keith Wenckowski, who has lived with type-one diabetes for more than two decades.

One of the participants in a new Northwestern Medicine study who suffered from painful diabetic neuropathy (PDN), Wenckowski finally found relief from the constant foot pain that required him to wear shoes at all times, even to the beach.

The study found that those with PDN who received two low dose rounds of a non-viral gene therapy called VM202 had significant improvement of their pain that lasted for months.

"I can now go to a beach and walk on the sand without feeling like I am walking on glass," Wenckowski said.

The results of this phase two, double-blind, placebo-controlled study will be published March 5 in the journal Annals of Clinical and Translation Neurology.

Right now there is no treatment for this disease of the peripheral nerves that affects 20 to 25 percent of diabetics. Patients with the most extreme form of the disease feel intense pain with a slight graze or touch. The pain can interfere with daily activities, sleep, mood and can diminish quality of life.

"Those who received the therapy reported more than a 50 percent reduction in their symptoms and virtually no side effects," said Dr. Jack Kessler, lead author of the study. "Not only did it improve their pain, it also improved their ability to perceive a very, very light touch."

Kessler is the Ken and Ruth Davee Professor of Stem Cell Biology in the department of neurology and a professor in the department of pharmacology at Northwestern University Feinberg School of Medicine. He also is an attending physician at Northwestern Memorial Hospital.

VM202 contains human hepatocyte growth factor (HGF) gene. Growth factor is a naturally occurring protein in the body that acts on cells -- in this case nerve cells -- to keep them alive, healthy and functioning. Future study is needed to investigate if the therapy can actually regenerate damaged nerves, reversing the neuropathy.

Wenckowski had continuous numbness, but now, more than a year since he received the therapy, his symptoms have not returned. "I am hoping the effects I am feeling do not cease," he said.

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Neuropathy: Relief for diabetics with painful condition

DUBLIN, Mar. 03, 2015 /PRNewswire/ --Research and Markets

(http://www.researchandmarkets.com/research/gxqhg9/gene_therapy) has announced the addition of Jain PharmaBiotech's new report "Gene Therapy - Technologies, Markets and Companies" to their offering.

Gene therapy technologies are described in detail including viral vectors, nonviral vectors and cell therapy with genetically modified vectors. Gene therapy is an excellent method of drug delivery and various routes of administration as well as targeted gene therapy are described. There is an introduction to technologies for gene suppression as well as molecular diagnostics to detect and monitor gene expression.

Clinical applications of gene therapy are extensive and cover most systems and their disorders. Full chapters are devoted to genetic syndromes, cancer, cardiovascular diseases, neurological disorders and viral infections with emphasis on AIDS. Applications of gene therapy in veterinary medicine, particularly for treating cats and dogs, are included.

Research and development is in progress in both the academic and the industrial sectors. The National Institutes of Health (NIH) of the US is playing an important part. As of 2014, over 2050 clinical trials have been completed, are ongoing or have been approved worldwide.A breakdown of these trials is shown according to the geographical areas and applications.

Since the death of Jesse Gelsinger in the US following a gene therapy treatment, the FDA has further tightened the regulatory control on gene therapy. A further setback was the reports of leukemia following use of retroviral vectors in successful gene therapy for adenosine deaminase deficiency. Several clinical trials were put on hold and many have resumed now. The report also discusses the adverse effects of various vectors, safety regulations and ethical aspects of gene therapy including germline gene therapy.

The markets for gene therapy are difficult to estimate as there is only one approved gene therapy product and it is marketed in China since 2004. Gene therapy markets are estimated for the years 2014-2024. The estimates are based on epidemiology of diseases to be treated with gene therapy, the portion of those who will be eligible for these treatments, competing technologies and the technical developments anticipated in the next decades. In spite of some setbacks, the future for gene therapy is bright.The markets for DNA vaccines are calculated separately as only genetically modified vaccines and those using viral vectors are included in the gene therapy markets

The voluminous literature on gene therapy was reviewed and selected 750 references are appended in the bibliography.The references are constantly updated. The text is supplemented with 75 tables and 15 figures.

Profiles of 181 companies involved in developing gene therapy are presented along with 223 collaborations. There were only 44 companies involved in this area in 1995. In spite of some failures and mergers, the number of companies has increased more than 4-fold within a decade.

Key Topics Covered:

Continue reading here:

Gene Therapy Market Report 2014-2024 - Technologies, Markets and Companies

DUBLIN, Mar. 03, 2015 /PRNewswire/ --Research and Markets

(http://www.researchandmarkets.com/research/rcv4lq/gene_therapy) has announced the addition of the "Gene Therapy Market, 2015 - 2025" report to their offering.

The "Gene Therapy Market, 2015-2025" report provides an extensive study on the marketed and pipeline gene therapies. A lot of research has been carried out in this field for over a decade but there are only five approved therapies (four available in Asian markets; one approved in the EU). There are many promising therapies which are currently being developed worldwide; the approach is likely to result in several commercial success stories in the foreseen future. The report covers various aspects, such as key players, marketed gene therapy products, products in clinical / pre-clinical research, associated ethical issues, likely future developments and upcoming opportunities for a variety of stakeholders.

Several disorders that arise inside the body are a result of either a direct genetic aberration or a dysfunctional/non-functional protein. The attempt to use nucleic acids to correct or delete the genes causing a particular disease is known as gene therapy. Although gene therapy has not contributed significantly to the global pharmaceutical market yet, it is anticipated to grow at a fast pace over the next decade.

Gendicine, developed by SiBiono GeneTech, was the foremost gene therapy that entered market in 2004 in China. Since then four more therapies have received approval in China, Philippines, Russia and the EU. This number for approved / marketed therapies seems weak at present; however, the strong and highly populated pipeline holds tremendous potential. There are 12 gene therapies in late stage of clinical development for the treatment of cancer, ocular and cardiovascular disorders.

There are several concerns that remain to be answered; examples include insertional mutagenesis, treatment of multigene disorders, curbing the risk of immune reactions, eugenics, high cost of therapy and ethical concerns related to making alterations at the genetic level. Despite this, gene therapy does offer a ray of hope for patients who either have no treatment options or show no benefits with drugs that are currently available. Such a benefit far outweighs any disadvantages that may be associated with this upcoming therapeutic field.

Key Topics Covered:

1. Preface

2. Executive Summary

3. Introduction

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Global Gene Therapy Market Report 2015-2025 - Extensive Study on the Marketed and Pipeline Gene Therapies

Carlsbad, CA (PRWEB) February 28, 2015

A study out of the University of Oxford has developed a promising new gene therapy procedure for the treatment of a hereditary ocular condition that leads to progressive blindness in patients. Authored by Dr. Robert MacLaren, professor of ophthalmology at the University of Oxford and published in the Lancet Medical Journal on January 16, 2014, the study treated six patients suffering from choroideremia, a rare genetic disorder that mostly affects men and leads to the degeneration of the choroid and retina, eventually causing blindness. Night blindness and loss of peripheral vision are the most common symptoms of choroideremia. For the study, researchers injected the subjects with healthy genetic material in an attempt to repair the damaged portions of the patients own gene, halting the onset of blindness.

Developing technologies and gene therapy are the future of ophthalmology, said Carlsbad LASIK surgeon Dr. Michael Tracy. In the past, there was little that could be done for certain patients facing blindness due to genetic and degenerative conditions. Gene therapy can lead to treatments that can stop the onset of blindness in certain patients before its too late.

For the Oxford study, researchers used virus particles to add the genetic material to the fluid behind the retina. The goal of the therapy was for the new genetic material to fix or patch flaws in the patients damaged DNA. Timing was an important element of the study. The participants in the study were all treated before their visual acuity had been irreversibly diminished, giving the therapy a greater chance of success. Of the six participants, two reported significant improvement in visual acuity, the other four reported improvement in night vision and maintained the same level of visual acuity as before the treatment in the affected eye. The therapy does involve the risk of further or permanent damage as it requires detachment of the retina, but none of the participants reported further degeneration of visual acuity in the treated eye.

This research is very important because it can pave the way for more gene therapy studies for treating other debilitating conditions that can lead to blindness, such as macular degeneration, added Dr. Tracy.

Dr. Michael Tracy is a board-certified ophthalmologist in Carlsbad, CA. He completed his residency training in ophthalmology at the Scheie Eye Institute at the University of Pennsylvania and his fellowship in Corneal and Refractive Surgery at the Bascom Palmer Eye Institute. Dr. Tracy specializes in LASIK and PRK surgery at his private practice at Carlsbad Eye Care.

To contact Carlsbad Eye Care, please visit http://carlsbad-eye-care.com/ or call (760) 603-9910.

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New Gene Therapy Procedure Could Hold Key to Preventing Blindness for Certain Patients

Cancer researchers at Indiana University report that about 15 percent of people with pancreatic cancer may benefit from therapy targeting a newly identified gene signature.

Using data from the Cancer Genome Atlas, Murray Korc, M.D., the Myles Brand Professor of Cancer Research at the Indiana University School of Medicine and a researcher at the Indiana University Melvin and Bren Simon Cancer Center, and colleagues found that a sub-group of pancreatic cancer patients who possess a strong angiogenic gene signature could benefit from personalized therapies that cut off the pathways that feed the cancer's growth.

This particular gene signature enables abnormal blood vessels to form in tumors, which feeds the tumor's growth.

The finding, published online Feb. 25 in the journal Oncotarget, is new because the prevalence of this signature was not previously known. The authors also demonstrated for the first time that endothelial cells, the main type of cell found in the inside lining of blood vessels, can produce molecules that directly stimulate the growth of pancreatic cancer cells.

"We showed that endothelial cells can stimulate the growth of pancreatic cancer cells and that by silencing or inhibiting certain pathways -- JAK1-2 and STAT3 -- we can alter that effect," Dr. Korc explained. "We demonstrated that it is possible to target these pathways and prolong the survival of genetically modified mice whose pancreatic cancers also have a strong pro-angiogenic gene signature."

Thus, for people with a strong pro-angiogenic gene signature, the finding suggests that they may benefit from targeted therapy that is directed against one of these pathways.

An important feature of the study was to demonstrate that it is possible to implant in mice small biopsy samples obtained from patients undergoing endoscopic procedures and to generate human tumors in these mice. When the original human tumor had evidence for angiogenesis, the implanted human tumor also exhibited angiogenesis in the mouse. Additional studies are necessary to confirm that these approaches could guide the design of precision medicine using targeted therapies, Dr. Korc said.

The need for new therapies for pancreatic cancer patients is great as only 7 percent of people with the disease survive more than five years after diagnosis. According to the American Cancer Society, there will be an estimated 48,960 new cases of pancreatic cancer and 40,560 deaths from the disease in 2015.

Co-authors of the study were Jesse Gore, Ph.D.; Stuart Sherman, M.D.; Harvey Cramer, M.D.; Hai Nguyen, M.D.; Kelly Craven, Monica Cheng, and Julie Wilson, all of IU School of Medicine, and Gregory Cote M.D. M.S., formerly of IU School of Medicine and now at the Medical University of South Carolina.

The study was made possible, in part, by grant CA-075059 awarded by the National Cancer Institute of the National Institutes of Health.

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Pancreatic cancer patients who benefit from personalized treatment identified

Albany, NewYork (PRWEB) February 25, 2015

Researchmoz.us announces the latest addition to its growing database of analytical market research reports. The new report, titled Gene Therapy Cardiovascular Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape, looks into the value chain of gene therapy and its association with cardiovascular treatments.

The report goes over the fundamentals of gene therapy in brief, explaining its biological mechanism and providing some historic background to better understand the growing influence of gene therapy in the healthcare sector and more particularly in the application of cardiovascular gene therapy. It also covers some of the jargon of the gene therapy sector for better comprehension of the detailed discussion of pipeline products.

Read Full Report With TOC at http://www.researchmoz.us/gene-therapy-cardiovascular-insight-pipeline-assessment-market-trend-technology-and-competitive-landscape-report.html

The report goes on to describe the determinant conditions in the cardiovascular gene therapy market, including the competitive landscape of the market and a detailed examination of the potential impact of market drivers and restraining factors. It describes the major products currently dominating the cardiovascular gene therapy market as well as the most potentially influential products and therapies in the pipeline. The analysis of the major pipeline products includes an evaluation of the competitive landscape concerning the products in questionthe market players developing them and the impact they could have on the markets competitive hierarchy.

The report elaborates upon the product description and technological background of the cardiovascular gene therapy in question, as well as the corporate aspect of it such as development partners and licensors and collaborators, and the stage of development which the product currently occupies.

The publication also includes a detailed overview of the pre-clinical and clinical outcomes of gene therapies and various biochemical aspects of the therapy such as the vector used in the procedure, the gene targeted by the therapy and localization, and an in-depth explanation of the mechanism by which the therapy operates. The report also covers dormant and discontinued products, which helps gain an understanding of what the market isnt ready for, or wasnt ready for at the time of the discontinuation. The pipeline cardiovascular gene therapies covered in the report include Gendicine, Rexin G, and Glybera.

Through the detailed analysis of all aspects related to the cardiovascular gene therapy market, the report provides in-depth insights on which to base winning market strategies.

In all, the report covers more than 25 cardiovascular gene therapy products from more than 20 manufacturers. The companies use 8 distinct technological support systems that have their own set of strengths and weaknesses, which are profiled in the report. As far as vectors are concerned, 50% of the total vectors studies are non-viral vectors, 46% are viral vectors, while a minuscule 4% are RNAi therapeutics.

All Latest Market Research Report at http://www.researchmoz.us/latest-report.html

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'Gene Therapy Cardiovascular Insight: Pipeline Assessment, Market Trend, Technology and Competitive Landscape'

A Stem Cell Cure for Bubble Baby Disease (SCID)
Visit: http://www.uctv.tv/) On November 18th, 2014, a UCLA research team led by Donald Kohn, M.D., announced a breakthrough gene therapy and stem cell cure ...

By: University of California Television (UCTV)

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A Stem Cell Cure for Bubble Baby Disease (SCID) - Video

Cancer Gene Therapy -- 8News Anchor Amy Lacey/VCU Massey Cancer Center
It can be devastating to be diagnosed with cancer, but what if you could take drugs tailored to your DNA, drugs that can buy you precious time? 8News Anchor Amy Lacey has more on the new test...

By: Amy Lacey

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Cancer Gene Therapy -- 8News Anchor Amy Lacey/VCU Massey Cancer Center - Video

Gene therapy leads to promising avenue for HIV vaccine
Gene therapy leads to promising avenue for HIV vaccine.

By: 7BILLIONHUMANBEING

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Gene therapy leads to promising avenue for HIV vaccine - Video