Dr. Campbell: New gene therapy may replace pacemaker implants
July 30, 2014 WNCN.

By: DrKevinCampbellMD

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Dr. Campbell: New gene therapy may replace pacemaker implants - Video

Joint research projects by NUIG and the Mayo Clinic will focus on a number of key strategic areas, including adult stem-cell therapy, gene therapy, biomaterials and biomedical engineering, the two institutes have said. Illustration: Getty

NUI Galway and the Mayo Clinic in the US plan to collaborate on clinical trials using regenerative medicine, following the signing of a memorandum of understanding between the two institutes.

The joint research projects will focus on a number of key strategic areas, including adult stem-cell therapy, gene therapy, biomaterials and biomedical engineering, the two institutes have said.

The Mayo Clinic and NUIGs Regenerative Medicine Institute have worked closely with each other for a number of years.

Both have licensed cell manufacturing facilities, and student and staff exchange programmes between Galway and the US will continue.

Welcoming the agreement, NUIG president Dr Jim Browne has noted that his university has Irelands only facility licensed to produce stem cells for human use.

A new clinical and translational research facility for conducting clinical trials with patients will be complete in early 2015, he said.

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NUI Galway in joint stem cell project with Mayo Clinic

3 Minute Thesis 2014 Winner - Gene therapy spray: A breath of fresh air - Presentation
Presenter: Harshavardini Padmanabhan Faculty of Health Sciences School of Paediatrics Thesis area: Aerosol Airway Gene Transfer Technique for Clinical Use Su...

By: University of Adelaide

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3 Minute Thesis 2014 Winner - Gene therapy spray: A breath of fresh air - Presentation - Video

3 Minute Thesis 2014 Winner - Harshavardini Padmanabhan - Interview
Gene therapy spray: A breath of fresh air Presenter: Harshavardini Padmanabhan Faculty of Health Sciences School of Paediatrics Thesis area: Aerosol Airway G...

By: University of Adelaide

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3 Minute Thesis 2014 Winner - Harshavardini Padmanabhan - Interview - Video

Sainik School Bijapur, Mahaling, Gene Therapy to Adjust Heartbeat,9 Sept 2014
Sainik School Bijapur, Mahaling, Gene Therapy to Adjust Heartbeat,9 Sept 2014.

By: D.Vijaya Kumar Kumar

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Sainik School Bijapur, Mahaling, Gene Therapy to Adjust Heartbeat,9 Sept 2014 - Video

27 - Gene Therapy by Jasmine Costa
27 - Gene Therapy by Jasmine Costa.

By: Bruce Rasmussen

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27 - Gene Therapy by Jasmine Costa - Video

Xenon Pharmaceuticals, an early-stage biotech with a gene therapy discovery platform for rare diseases, filed on Wednesday with the SEC to raise up to $52 million in an initial public offering. Xenon has a number of collaboration and licensing agreements with large pharmaceuticals including Teva, Genentech and Merck for its preclinical research.

The company's discovery platform was used to develop uniQure's Glybera treatment for orphan disease lipoprotein lipase deficiency, the first gene therapy approved in the EU. February IPO uniQure ( QURE ) priced above its range but ended the first day down 14% and now trades down 36% below the IPO price. Xenon is eligible to receive mid single-digit royalties on net sales of Glybera. Teva is in Phase 2 trials for a gene therapy discovered by Xenon that is being developed to treat osteoarthritis. Genentech was cleared to begin a Phase 1 trial for Xenon's pain treatment and Merck is in preclinical development for cardiovascular disease. Xenon also has a variety of preclinical therapies for both orphan diseases (such as Dravet Syndrome) and large-market conditions (including acne).

Primary shareholders include Medpace (16%), Lipterx (11%), InterWest Partners (9%), Fidelity (7%), Invesco (5%) and CEO Simon Pimstone (4%).

The Burnaby, Canada-based company, which was founded in 1996 and booked $27 million in collaboration revenue for the 12 months ended June 30, 2014, plans to list on the NASDAQ under the symbol XENE. Xenon Pharmaceuticals initially filed confidentially on August 16, 2013. Jefferies and Wells Fargo Securities are the joint bookrunners on the deal. No pricing terms were disclosed.

Investment Disclosure: The information and opinions expressed herein were prepared by Renaissance Capital's research analysts and do not constitute an offer to buy or sell any security. Renaissance Capital, the Renaissance IPO ETF (symbol: IPO) or the Global IPO Fund (symbol: IPOSX) , may have investments in securities of companies mentioned.

The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of The NASDAQ OMX Group, Inc.

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Rare disease biotech Xenon Pharmaceuticals files for a $52 million IPO

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Newswise PHILADELPHIA - Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this months issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments.

Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit, says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. Were proposing a different approach that spreads payments out and only keep coming if the patient continues to do well.

Wilson and co-author Troyen A. Brennan, MD, JD, MPH, chief medical officer of CVS Health, note that while large single payments for gene therapy may be the simplest approach, they carry substantial encumbrances. For example, approval of gene therapy treatments is unavoidably based on data derived from trials carried out over several years at most -- considerably shorter than the expected duration of the therapy. Payers may therefore be unwilling to pay large up-front sums for treatments whose long-term benefit has not been established. Additionally, large payments for medications, such as the $84,000-a-patient cost of the hepatitis C treatment Sovaldi, have been criticized in the prevailing climate of curbing health care costs. This, despite the fact that effective gene therapy may reduce the overall financial burden to the health care system.

Wilson and Brennan further note that while a liver transplant, for example, can cost up to $300,000, physicians and hospitals that transplant livers know they will be compensated at market rates through existing contracts -- gene developers lack that assurance. Annuity payments, they say, could help address these problems.

An example of an annuity-type disbursement could be a hypothetical payment of $150,000 per year for a certain number of years for gene-therapy-based protein replacement for patients with hemophilia B -- so long as the therapy continues to work. According to the authors, the cumulative amount should be less than the cost of a one-time payment of $4-6 million, which would be the expected rate for a gene-based therapy to be comparatively priced to existing, conventional therapies for hemophilia B. One would presume, they write, that gene therapy will have to represent a discount in order for insurers to approve its use.

The annuity model that were proposing would eliminate the misguided incentive to invest in drugs and treatments with ongoing revenue streams but which require continuing, perhaps lifetime daily administration, with all the attendant inconveniences and burdens to patients and their families, as well as direct and indirect costs to the nations health system, says Wilson.

The authors point out that gene therapy differs substantially from the case of orphan drugs. Development of the latter, which target rare diseases affecting small patient populations, is supported by the Orphan Drug Act of 1983, which provides pharmaceutical manufacturers with grants, tax credits, and an extended period of market exclusivity for their medications. Whats more, in virtually all of these cases, the business costs of developing the drugs are further attenuated by ongoing administration of -- and payment for -- the medication over the lifetime of the patient. The contrast with gene therapy, especially that which produces a durable cure with one administration, the authors write, is clear.

Adding further details to their proposal, the authors write that The original annuity payment could be set with certain types of re-opener clauses, such as with patent expiration [death], or if a less expensive new therapy came on line -- thus subjecting the gene therapy annuity to the same vagaries of market competition that standard pharmaceuticals face.

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Penn Researcher and CVS Health Physician Urge New Payment Model for Costly Gene Therapy Treatments

Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this month's issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments.

"Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit," says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. "Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. We're proposing a different approach that spreads payments out and only keep coming if the patient continues to do well."

Wilson and co-author Troyen A. Brennan, MD, JD, MPH, chief medical officer of CVS Health, note that while large single payments for gene therapy may be the simplest approach, they carry substantial encumbrances. For example, approval of gene therapy treatments is unavoidably based on data derived from trials carried out over several years at most -- considerably shorter than the expected duration of the therapy. Payers may therefore be unwilling to pay large up-front sums for treatments whose long-term benefit has not been established. Additionally, large payments for medications, such as the $84,000-a-patient cost of the hepatitis C treatment Sovaldi, have been criticized in the prevailing climate of curbing health care costs. This, despite the fact that effective gene therapy may reduce the overall financial burden to the health care system.

Wilson and Brennan further note that while a liver transplant, for example, can cost up to $300,000, physicians and hospitals that "transplant livers know they will be compensated at market rates through existing contracts -- gene developers lack that assurance." Annuity payments, they say, could help address these problems.

An example of an annuity-type disbursement could be a hypothetical payment of $150,000 per year for a certain number of years for gene-therapy-based protein replacement for patients with hemophilia B -- so long as the therapy continues to work. According to the authors, the cumulative amount should be less than the cost of a one-time payment of $4-6 million, which would be the expected rate for a gene-based therapy to be comparatively priced to existing, conventional therapies for hemophilia B. "One would presume," they write, "that gene therapy will have to represent a discount in order for insurers to approve its use."

"The annuity model that we're proposing would eliminate the misguided incentive to invest in drugs and treatments with ongoing revenue streams but which require continuing, perhaps lifetime daily administration, with all the attendant inconveniences and burdens to patients and their families, as well as direct and indirect costs to the nation's health system," says Wilson.

The authors point out that gene therapy differs substantially from the case of "orphan" drugs. Development of the latter, which target rare diseases affecting small patient populations, is supported by the Orphan Drug Act of 1983, which provides pharmaceutical manufacturers with grants, tax credits, and an extended period of market exclusivity for their medications. What's more, in virtually all of these cases, the business costs of developing the drugs are further attenuated by ongoing administration of -- and payment for -- the medication over the lifetime of the patient. "The contrast with gene therapy, especially that which produces a durable cure with one administration," the authors write, "is clear."

Adding further details to their proposal, the authors write that "The original annuity payment could be set with certain types of 're-opener' clauses, such as with patent expiration [death], or if a less expensive new therapy came on line -- thus subjecting the gene therapy annuity to the same vagaries of market competition that standard pharmaceuticals face."

A crucial issue would be the calculation of the annual annuity payment. One option would be for the government to set the price through the Medicare program, since many of the patients with rare diseases are disabled and thus qualify for Medicare. The Medicare rate could in turn become a benchmark for the commercial market.

Another key test in developing an annuity model is determining the correct linkage between payments and the therapy's continued effectiveness and safety. In most diseases, this would entail identifying a biomarker reasonably correlated with efficacy, for example, plasma measures of clotting in hemophilia patients treated with gene therapy.

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New payment model for gene therapy needed, experts say

PUBLIC RELEASE DATE:

9-Sep-2014

Contact: Karen Kreeger karen.kreeger@uphs.upenn.edu 215-349-5658 University of Pennsylvania School of Medicine http://www.twitter.com/PennMedNews

PHILADELPHIA - Hoping to encourage sufficient investments by pharmaceutical companies in expensive gene therapies, which often consist of a single treatment, a Penn researcher and the chief medical officer of CVS Health outline an alternative payment model in this month's issue of Nature Biotechnology. They suggest annuity payments over a defined period of time and contingent on evidence that the treatment remains effective. The approach would replace the current practice of single, usually large, at-point-of-service payments.

"Unlike most rare disease treatments that can continue for decades, gene therapy is frequently administered only once, providing many years, even a lifetime, of benefit," says James M. Wilson, MD, PhD, professor of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania. "Under current reimbursement policies, private insurers and the government typically pay for this therapy once: when it is administered. But these individual payments could reach several million dollars each under current market conditions. We're proposing a different approach that spreads payments out and only keep coming if the patient continues to do well."

Wilson and co-author Troyen A. Brennan, MD, JD, MPH, chief medical officer of CVS Health, note that while large single payments for gene therapy may be the simplest approach, they carry substantial encumbrances. For example, approval of gene therapy treatments is unavoidably based on data derived from trials carried out over several years at most -- considerably shorter than the expected duration of the therapy. Payers may therefore be unwilling to pay large up-front sums for treatments whose long-term benefit has not been established. Additionally, large payments for medications, such as the $84,000-a-patient cost of the hepatitis C treatment Sovaldi, have been criticized in the prevailing climate of curbing health care costs. This, despite the fact that effective gene therapy may reduce the overall financial burden to the health care system.

Wilson and Brennan further note that while a liver transplant, for example, can cost up to $300,000, physicians and hospitals that "transplant livers know they will be compensated at market rates through existing contracts -- gene developers lack that assurance." Annuity payments, they say, could help address these problems.

An example of an annuity-type disbursement could be a hypothetical payment of $150,000 per year for a certain number of years for gene-therapy-based protein replacement for patients with hemophilia B -- so long as the therapy continues to work. According to the authors, the cumulative amount should be less than the cost of a one-time payment of $4-6 million, which would be the expected rate for a gene-based therapy to be comparatively priced to existing, conventional therapies for hemophilia B. "One would presume," they write, "that gene therapy will have to represent a discount in order for insurers to approve its use."

"The annuity model that we're proposing would eliminate the misguided incentive to invest in drugs and treatments with ongoing revenue streams but which require continuing, perhaps lifetime daily administration, with all the attendant inconveniences and burdens to patients and their families, as well as direct and indirect costs to the nation's health system," says Wilson.

The authors point out that gene therapy differs substantially from the case of "orphan" drugs. Development of the latter, which target rare diseases affecting small patient populations, is supported by the Orphan Drug Act of 1983, which provides pharmaceutical manufacturers with grants, tax credits, and an extended period of market exclusivity for their medications. What's more, in virtually all of these cases, the business costs of developing the drugs are further attenuated by ongoing administration of -- and payment for -- the medication over the lifetime of the patient. "The contrast with gene therapy, especially that which produces a durable cure with one administration," the authors write, "is clear."

Excerpt from:

Penn researcher and CVS Health physician urge new payment model for gene therapy

PUBLIC RELEASE DATE:

9-Sep-2014

Contact: Evy Vierstraete info@vib.be 32-924-46611 VIB (the Flanders Institute for Biotechnology)

For the first time the Jeffrey Modell Foundation is giving a research grant to a Belgian laboratory. The team of Adrian Liston from VIB-KU Leuven will use the grant to develop a gene therapy to cure children that suffer from IPEX syndrome, a rare and fatal autoimmune disorder in which the immune system attacks the body's own tissues and organs. At the moment, the only successful therapy to treat the syndrome is a bone marrow transplantation, which is not available for all children.

"This is a real chance for a cure", said group leader Adrian Liston. "The gene responsible for this disease was identified 13 years ago, but for the first time we may have learned enough about the basic biology to solve it. We should know within a year whether the gene therapy works in mice, after which we can move to patients at top speed."

The Jeffrey Modell Foundation (JMF)

JMF is a global non-profit organization for patients who suffer from Primary Immunodeficiency (PI) and their relatives. The organization is devoted to early and precise diagnosis, meaningful treatments and, ultimately, cures. Through clinical and basic research, physician education, patient support, advocacy, public awareness and new-born screening they want to make a difference in the lives of patients with PI.

Vicki and Fred Modell established the Foundation in 1987, in memory of their son Jeffrey, who died at the age of fifteen from complications of PI. During the years, the foundation has created a network of the world's leading expert immunologists. Two years ago the Child Immune Deficiencies Department of UZ Leuven was given the first certification as a "Jeffrey Modell Foundation Diagnostic and Research Center for Primary Immunodeficiencies" in Belgium.

IPEX and primary immunodeficiency (PI)

IPEX is an acronym for immune dysregulation, polyendocrinopathy (diseases affecting multiple endocrine glands), enteropathy (disorder of the intestines), and X-linked (pattern of inheritance).

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Jeffrey Modell Foundation supports Belgian research on primary immunodeficiency

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Newswise ANN ARBOR, Mich. University of Michigan Health System doctors have started testing a unique new approach to fighting brain tumors -- one that delivers a one-two punch designed to knock out the most dangerous brain cancer.

The experimental approach, based on U-M research, delivers two different genes directly into the brains of patients following the operation to remove the bulk of their tumors.

The idea: trigger immune activity within the brain itself to kill remaining tumor cells -- the ones neurosurgeons cant take out, which make this type of tumor so dangerous.

Its the first time this gene therapy approach is being tried in humans, after more than a decade of research in experimental models.

One of the genes is designed to kill tumor cells directly, and is turned on when the patient takes a certain drug. The other gene spurs the bodys own immune system to attack remaining cancer cells. Both are delivered into brain cells via a harmless virus.

The Phase I clinical trial has already enrolled two patients who have tolerated the gene delivery without complications. All patients in the study must have a presumptive diagnosis of WHO grade 3 or 4 malignant primary glioma, such as glioblastoma multiforme; patients must not have been treated yet by any therapy. They must also meet other criteria for inclusion in the trial.

More patients will be able to enroll at a pace of about one every three weeks, through a careful selection process. In addition to surgery and gene therapy at U-M, each will receive standard chemotherapy and radiation therapy as well as follow-up assessments for up to two years.

Were very pleased to see our years of research lead to a clinical trial, because based on our prior work we believe this combination of cell-killing and immune-stimulating approaches holds important promise, says principal investigator Pedro Lowenstein, M.D., Ph.D., the U-M Medical School Department of Neurosurgery professor who has co-led the basic research effort to develop and test the strategy.

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A One-Two Punch for Brain Tumors? New Clinical Trial Opens at U-M

First published in News Last updated by Robert Merrick, Parliamentary Correspondent

PROTESTS by a group of MPs have failed to derail plans for a controversial gene therapy, to stop incurable diseases passing to the next generations.

Ministers vowed to plough ahead with preparations for the DNA-altering procedure, which is being pioneered by a team at Newcastle University.

However, the department of health declined to say when the issue would be put to a vote in Parliament, despite suggestions that it could be before the end of the year.

The treatment involves replacing faulty mitochondria responsible for inherited diseases, including muscle wasting, heart problems, vision loss, organ failure and epilepsy.

Embryos are given healthy DNA from donor eggs, meaning a baby has the DNA of three people from two parents, plus less than one per cent from the donor.

Professor Doug Turnbull, who leads the Newcastle team, has urged the Government to draw up legislation as soon as possible, because of the number of patients waiting for treatment.

But, in the Commons, MPs brought forward a motion demanding further research and for new regulations to be delayed in light of public safety concerns.

Fiona Bruce, a Conservative backbencher, claimed the Human Fertilisation and Embryology Authority (HFEA) wanted further research, saying: This is a case of genetic engineering.

It is the alteration of a potential human being - the removal of certain genes and their replacement with others, to create children.

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MPs' protests fail to derail gene therapy plans

ABC Erin Griffin was an advocate for childhood cancer awareness.

A 14-year-old girl who campaigned to raise awareness about childhood cancer has died after making history through her participation in a gene therapy trial.

Erin Griffin died at the Children's Hospital in Adelaide with her parents, brother and grandmother by her side.

In 2013, Erin received a Children's Week Award for her advocacy work in raising childhood cancer awareness.

Erin was born in Scotland and moved to Australia when she was six.

She was diagnosed with Diffuse Intrinsic Pontine Glioma (DIPG), an incurable brain cancer, on February 18, 2012.

"We're all very sad to lose this special girl," Erin's specialist oncologist Dr Geoff McCowage told the ABC.

Dr McCowage, who runs a gene therapy trial at the Children's Hospital at Westmead in Sydney, said Erin made history.

"She was only the second child in the world to take part [in the trial]," he said.

"Throughout her ordeal she stayed courageous, she took two trips to the United States, one to Scotland and she was active on the internet telling the story of what she was going through and offering support to others going through the same thing.

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14-year-old childhood cancer campaigner Erin Griffin dies in Adelaide

Gene Therapy EDTECH

By: Krittamet Tatsanapornsatith

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Gene Therapy EDTECH - Video

The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing and maintaining the anti-serums in large quantities.

New research led by Charles Shoemaker, Ph.D., professor in the Department of Infectious Disease and Global Health at the Cummings School of Veterinary Medicine at Tufts University, shows that gene therapy may offer significant advantages in prevention and treatment of botulism exposure over current methods. The findings of the National Institutes of Health funded study appear in the August 29 issue of PLOS ONE.

Shoemaker has been studying gene therapy as a novel way to treat diseases such as botulism, a rare but serious paralytic illness caused by a nerve toxin that is produced by the bacterium Clostridium botulinum. Despite the relatively small number of botulism poisoning cases nationally, there are global concerns that the toxin can be produced easily and inexpensively for bioterrorism use. Botulism, like E. coli food poisoning and C. difficile infection, is a toxin-mediated disease, meaning it occurs from a toxin that is produced by a microbial infection.

Shoemaker's previously reported antitoxin treatments use proteins produced from the genetic material extracted from alpacas that were immunized against a toxin. Alpacas, which are members of the camelid family, produce an unusual type of antibody that is particularly useful in developing effective, inexpensive antitoxin agents. A small piece of the camelid antibody -- called a VHH -- can bind to and neutralize the botulism toxin. The research team has found that linking two or more different toxin-neutralizing VHHs results in VHH-based neutralizing agents (VNAs) that have extraordinary antitoxin potency and can be produced as a single molecule in bacteria at low cost. Additionally, VNAs have a longer shelf life than traditional antibodies so they can be better stored until needed.

The newly published PLOS ONE study assessed the long-term efficacy of the therapy and demonstrated that a single gene therapy treatment led to prolonged production of VNA in blood and protected the mice from subsequent exposures to C. botulinum toxin for up to several months. Virtually all mice pretreated with VNA gene therapy survived when exposed to a normally lethal dose of botulinum toxin administered up to nine weeks later. Approximately 40 percent survived when exposed to this toxin as late as 13 or 17 weeks post-treatment. With gene therapy the VNA genetic material is delivered to animals by a vector that induces the animals to produce their own antitoxin VNA proteins over a prolonged period of time, thus preventing illness from toxin exposures.

The second part of the study showed that mice were rapidly protected from C. botulinum toxin exposure by the same VNA gene therapy, surviving even when treated 90 minutes after the toxin exposure.

"We envision this treatment approach having a broad range of applications such as protecting military personnel from biothreat agents or protecting the public from other toxin-mediated diseases such as C. difficile and Shiga toxin-producing E. coli infections," said Shoemaker, the paper's senior author. "More research is being conducted with VNA gene therapy and it's hard to deny the potential of this rapid-acting and long-lasting therapy in treating these and several other important illnesses."

Story Source:

The above story is based on materials provided by Tufts University. Note: Materials may be edited for content and length.

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Efficacy of new gene therapy approach for toxin exposures shown in mouse study

PUBLIC RELEASE DATE:

29-Aug-2014

Contact: Rushmie A Nofsinger rushmie.nofsinger@tufts.edu 508-839-7910 Tufts University, Health Sciences Campus

NORTH GRAFTON, Mass. (August 29, 2014, 2 PM US Eastern Time)The current method to treat acute toxin poisoning is to inject antibodies, commonly produced in animals, to neutralize the toxin. But this method has challenges ranging from safety to difficulties in developing, producing and maintaining the anti-serums in large quantities.

New research led by Charles Shoemaker, Ph.D., professor in the Department of Infectious Disease and Global Health at the Cummings School of Veterinary Medicine at Tufts University, shows that gene therapy may offer significant advantages in prevention and treatment of botulism exposure over current methods. The findings of the National Institutes of Health funded study appear in the August 29 issue of PLOS ONE.

Shoemaker has been studying gene therapy as a novel way to treat diseases such as botulism, a rare but serious paralytic illness caused by a nerve toxin that is produced by the bacterium Clostridium botulinum. Despite the relatively small number of botulism poisoning cases nationally, there are global concerns that the toxin can be produced easily and inexpensively for bioterrorism use. Botulism, like E. coli food poisoning and C. difficile infection, is a toxin-mediated disease, meaning it occurs from a toxin that is produced by a microbial infection.

Shoemaker's previously reported antitoxin treatments use proteins produced from the genetic material extracted from alpacas that were immunized against a toxin. Alpacas, which are members of the camelid family, produce an unusual type of antibody that is particularly useful in developing effective, inexpensive antitoxin agents. A small piece of the camelid antibody called a VHH can bind to and neutralize the botulism toxin. The research team has found that linking two or more different toxin-neutralizing VHHs results in VHH-based neutralizing agents (VNAs) that have extraordinary antitoxin potency and can be produced as a single molecule in bacteria at low cost. Additionally, VNAs have a longer shelf life than traditional antibodies so they can be better stored until needed.

The newly published PLOS ONE study assessed the long-term efficacy of the therapy and demonstrated that a single gene therapy treatment led to prolonged production of VNA in blood and protected the mice from subsequent exposures to C. botulinum toxin for up to several months. Virtually all mice pretreated with VNA gene therapy survived when exposed to a normally lethal dose of botulinum toxin administered up to nine weeks later. Approximately 40 percent survived when exposed to this toxin as late as 13 or 17 weeks post-treatment. With gene therapy the VNA genetic material is delivered to animals by a vector that induces the animals to produce their own antitoxin VNA proteins over a prolonged period of time, thus preventing illness from toxin exposures.

The second part of the study showed that mice were rapidly protected from C. botulinum toxin exposure by the same VNA gene therapy, surviving even when treated 90 minutes after the toxin exposure.

"We envision this treatment approach having a broad range of applications such as protecting military personnel from biothreat agents or protecting the public from other toxin-mediated diseases such as C. difficile and Shiga toxin-producing E. coli infections," said Shoemaker, the paper's senior author. "More research is being conducted with VNA gene therapy and it's hard to deny the potential of this rapid-acting and long-lasting therapy in treating these and several other important illnesses."

Read more from the original source:

Mice study shows efficacy of new gene therapy approach for toxin exposures

"How to Predict and Improve Your Future Health with Gene Therapy" by Devin Stone at MetroMD
"How to Predict and Improve Your Future Health with Gene Therapy" by Devin Stone at MetroMD.

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"How to Predict and Improve Your Future Health with Gene Therapy" by Devin Stone at MetroMD - Video

Combine some microbiologists with some mechanical engineers and you never know what might happen.

A recent collaboration of those two fields of scientists at Brigham Young University produced a machine so small it takes a microscope to see it.

It's not just for fun, however. It could pave the way for research into diseases including Alzheimer's, cancer and diabetes.

For decades scientists have conducted gene research by transferring genetic material into a new cell by a process called microinjection. It was just considered an unfortunate by-product of the process to have 40 percent of the cells die.

Enter BYU and a different process called nanoinjection, which occurs by transferring material including DNA into cells.

"Because DNA is naturally negatively charged, it is attracted to the outside of the lance using positive voltage," said Brian Jensen, a BYU professor of mechanical engineering, in a release. "Once we insert the lance into a cell, we simply reverse the polarity of the electrical force and the lance releases the DNA."

The lance being used is 10 times smaller than what was used previously. In the past, researchers used a hollow needle to pump a DNA-filled liquid into an egg cell nucleus, but the extra fluid caused many of the cells to swell and die.

With the new method, the team found that nearly 80 percent of the cells proceeded to the next stage of development, a significant increase from the previous 60 percent success rate.

No extra fluid is used and the cells undergo less stress and have a higher survival rate.

"It is really great engineering stuff," said Sandra Burnett, associate professor of microbiology at BYU.

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Smaller is better: BYU creates new gene therapy technology

Gene Therapy Cure Cancer
This is my final project for science subject at my school. I learning about biotechnology. My topic is about " How gene therapy can cure cancer?" and i have to fin the solution. I am very...

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Gene Therapy Cure Cancer - Video