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.

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

Glybera gene therapy reduces pancreatitis risk in Lipoprotein Lipase Deficiency patients

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today released data showing that its gene therapy Glybera® (alipogene tiparvovec) significantly reduces the risk of pancreatitis in patients with Lipoprotein Lipase Deficiency (LPLD). Pancreatitis, or inflammation of the pancreas, is a major clinical symptom of LPLD.

29 Jun 2011

Genetic tools that enable cell type-specific delivery hold promise for combating incurable neurologic 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. Five of the most exciting developments in this rapidly advancing field are presented in a series of articles in the June issue of Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc..

29 Jun 2011

Circulation journal publishes Celladon's MYDICAR Phase 2 trial on advanced heart failure

Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, announces that the data from its completed Phase 2 CUPID clinical study of MYDICAR for the treatment of advanced heart failure is being published today online in Circulation, an American Heart Association journal.

28 Jun 2011

Flamel, Digna Biotech enter joint development agreement for multiple products

Flamel Technologies SA and Digna Biotech SL today announced that the two companies have entered into a joint development agreement for the pre-clinical and clinical development of multiple products.

27 Jun 2011

In vivo genome editing restores hemostasis in mouse model of blood clotting disorder

Using an innovative gene therapy technique called genome editing that hones in on the precise location of mutated DNA, scientists have treated the blood clotting disorder hemophilia in mice.

27 Jun 2011

AMT receives CHMP opinion on Glybera Marketing Authorisation Application

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that it has received an opinion on its Marketing Authorisation Application (MAA) for Glybera as a potential therapy for Lipoprotein Lipase Deficiency.

24 Jun 2011

New surprising details about flow of neurotransmitters between brain neurons

Two studies featuring research from Weill Cornell Medical College have uncovered surprising details about the complex process that leads to the flow of neurotransmitters between brain neurons -- a dance of chemical messages so delicate that missteps often lead to neurological dysfunction.

23 Jun 2011

Telaprevir drug shows promise in curing hepatitis C infection

The drug telaprevir (Incivek) provides a dramatic improvement in the treatment of the most common form of hepatitis C infection, says an international team of investigators led by Dr. Ira M. Jacobson of NewYork-Presbyterian Hospital/Weill Cornell Medical Center.

23 Jun 2011