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.

Gene therapy helps damaged heart cells regain strength and beat normally

Researchers at Jefferson Medical College and Duke University have used gene therapy to help damaged heart cells regain strength and beat normally again in the laboratory. The work takes the scientists one step closer to eventual clinical trials in humans.

12 Apr 2004

University of Florida researchers have used a common gel to successfully deliver gene therapy to the diaphragm

The technique, described in the current issue of Molecular Therapy, could eventually lead to a method to correct genetic conditions in humans that cause diaphragm weakness and respiratory failure -- a leading cause of death in tens of thousands of patients with forms of muscular dystrophy, including Pompe’s disease. Thousands of Americans with muscle-weakening diseases are placed on ventilators each year, according to the Muscular Dystrophy Association.

7 Apr 2004

Genetic trick makes gene therapy vectors more versatile

A genetic trick used by viruses to replicate themselves has been adapted for laboratory use to build complex protein structures required by immune system cells, according to investigators at St. Jude Children’s Research Hospital.

7 Apr 2004

Oxford Biomedica to win Department of Health gene funding

Oxford spinout Oxford Biomedica was yesterday awarded £0.5 million by the Department of Health to conduct the first British gene therapy haemophilia trial.

31 Mar 2004

£1.6m for research into a possible cure for muscular dystrophy

A consortium which includes Oxford researchers has won £1.6m in government funding for ground-breaking research into a possible cure for muscular dystrophy.

30 Mar 2004

Oregon Stem Cell Center Hunt for Human Disease Therapies

Oregon Health & Science University's fast-growing stem cell research program, which already has made significant strides in the hunt for human disease therapies, now has a place to call home.

23 Mar 2004

Tiny Ribozyme package could be future treatment of Hepatitis B virus

Penn State College of Medicine researchers have developed a tiny package that searches for and destroys up to 80 percent of hepatitis B virus in the livers of mice.

22 Mar 2004

AMT Obtains Orphan Drug Designation for Its LPL Gene Therapy Product

AMT today announced that its adeno-associated viral vector (AAV) expressing lipoprotein lipase (LPL) product has been designated as an orphan medicinal product for the treatment of LPL deficiency by the European Commission. This decision follows a positive recommendation by the European Agency for the Evaluation of Medicinal Products.

22 Mar 2004