Gene Therapy News and Research

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Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery.

PharmaNet to assist BrainStorm in obtaining FDA approval for ALS human clinical trials

BrainStorm Cell Therapeutics Inc., an innovative developer of adult stem cell technologies and CNS therapeutics, announced today that it has signed an agreement with PharmaNet LLC, an inVentiv Health Company, to assist in the development of the protocol and IND file for submission to the FDA for approval of BrainStorm's ALS human clinical trials in the United States.

26 Jul 2011

Promising drug for Duchenne muscular dystrophy

Duchenne muscular dystrophy is a severely debilitating and lethal condition that affects 1 in 3,000. Females and males are affected, though females are rarely affected and are more often carriers. It is caused due to a defective gene that normally codes for the protein dystrophin, which helps provide a protective membrane around muscle fibers. Without this skin, muscle fibers become damaged and eventually die.

25 Jul 2011

FDA approves U.S. launch of Wiskott-Aldrich gene therapy clinical trial

The U.S. Food & Drug Administration (FDA) approved the launching in the U.S. of a clinical trial for gene therapy for a rare immunodeficiency, Wiskott-Aldrich syndrome.

22 Jul 2011

NIAID awards $36M grant to BIDMC for AIDS vaccine research in nonhuman primates

Beth Israel Deaconess Medical Center (BIDMC) has been awarded a grant from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, to lead a Consortium for AIDS Vaccine Research in Nonhuman Primates.

21 Jul 2011

Cell biologists identify infection mechanism of adenoviruses

Mucosal epithelia do not have any receptors on the outer membrane for the absorption of viruses like hepatitis C, herpes, the adenovirus or polio, and are thus well-protected against pathogenic germs. However, certain viruses, such as the human immunodeficiency virus HIV, still manage to enter the body via the mucous membrane.

21 Jul 2011

Localized one-time gene therapy might effectively protect against atherosclerosis

A one-dose method for delivering gene therapy into an arterial wall effectively protects the artery from developing atherosclerosis despite ongoing high blood cholesterol.

21 Jul 2011

Camel blood can help scientists fight against cancer

Nanobodies produced from camel blood have unique properties, which can be used in future drug development. New research published in Journal of Controlled Release confirms that camel blood can help scientists in the fight against cancer.

15 Jul 2011

NIH grants $70M over five years to help search for HIV cure

NIH announced on Monday it will provide $70 million over five years to three collaborations searching for an HIV/AIDS cure, making it "the largest single investment yet … into finding a way to rid the virus from the body or at least reduce levels to the point that infected people can stop taking anti-HIV drugs - which many researchers until recently viewed as a hopeless quest," ScienceInsider reports.

13 Jul 2011

New research strategy for eradicating HIV from the body

An international team led by researchers from the University of California, San Francisco and the nonprofit Vaccine and Gene Therapy Institute of Port St. Lucie, Fla., has received a major grant from the National Institutes of Health to develop a strategy to eradicate HIV from the body.

12 Jul 2011

Merck researchers to participate in two new collaborative efforts to study HIV latency

Merck, known as MSD outside the United States and Canada, today announced that company researchers will participate in two new collaborative efforts led by the prominent academic institutions of the University of North Carolina (UNC) Chapel Hill and the University of California San Francisco (UCSF) to develop new approaches towards eradicating HIV, the virus that causes AIDS.

12 Jul 2011

genOway acquires worldwide exclusive license for RMCE technology from bluebird bio for genetically modified rodents

genOway and bluebird bio report today genOway acquisition of an exclusive worldwide license for the RMCE technology (developed at the Massachusetts Institute of Technology) in the field of genetically modified rodents.

12 Jul 2011

NIAID awards $14 million a year for new HIV research

Three research teams focused on developing strategies that could help to rid the body of HIV are receiving grants totaling more than $14 million a year, for up to five years, the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health announced today.

11 Jul 2011

New technique may help scientists probe mysteries of many different stem cells

Researchers at the Salk Institute have been able to genetically incorporate "unnatural" amino acids, such as those emitting green fluorescence, into neural stem cells, which then differentiate into brain neurons with the incandescent "tag" intact.

8 Jul 2011

Gene therapy may help prevent progression of type 1 diabetes

Increasing a specific protein in areas of the pancreas that produce insulin blocks the immune attack that causes type 1 diabetes, researchers reported in the August issue of the Journal of Clinical Investigation, published early online.

7 Jul 2011

Freeze-dried gene therapy system safe and effective for treating brain cancer

Researchers at the Johns Hopkins University School of Medicine have developed a technique that delivers gene therapy into human brain cancer cells using nanoparticles that can be freeze-dried and stored for up to three months prior to use. The shelf-stable particles may obviate the need for virus-mediated gene therapy, which has been associated with safety concerns.

7 Jul 2011

Small molecule treatment holds promise for Usher syndrome

New treatment approach shall soon be ready for use in Usher syndrome patients / Publication in "Human Gene Therapy".

1 Jul 2011

Mary Ann Liebert peer-reviewed journal highlights advances in gene therapy for neurological diseases

Novel tools and methods for delivering therapeutic genes to cells in the central nervous system hold great promise for the development of new treatments to combat incurable neurologic diseases.

30 Jun 2011

Pregenen, Cellectis partner to develop meganucleases

Cellectis, the French genome engineering specialist, announced today the signing of a collaboration agreement with Pregenen, an American biotechnology start-up company dedicated to the creation of innovative tools for genome engineering.

30 Jun 2011

Mount Sinai gene therapy improves cardiac function in people with severe heart failure

Researchers at Mount Sinai School of Medicine have found in a Phase II trial that a gene therapy developed at Mount Sinai stabilized or improved cardiac function in people with severe heart failure. Patients receiving a high dose of the therapy, called SERCA2a, experienced substantial clinical benefit and significantly reduced cardiovascular hospitalizations, addressing a critical unmet need in this population.

29 Jun 2011

Researchers discover herpes virus-associated disease in monkeys

Researchers at Oregon Health & Science University have discovered a naturally occurring disease in monkeys that is very much like multiple sclerosis in humans - a discovery that could have a major impact on efforts to understand the cause of multiple sclerosis.

29 Jun 2011