First Trial of Gene Therapy for Advanced Heart Failure Shows Promising Results

Phase I Results Presented at the American Heart Association Scientific Sessions Affirm Safety and Show Promise for Clinical Improvements

NewYork-Presbyterian Hospital/Columbia University Medical Center and Other Study Centers Begin Phase II

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Scientist Clears Hurdles for Muscular Dystrophy Therapy

Approximately 250,000 people in the United States have some form of muscular dystrophy. Duchenne muscular dystrophy (DMD) is the most common type of the disease, predominantly affecting males. Boys with DMD will lose the ability to walk by their teens and typically die before the age of 30. For years, scientists have studied the use of gene therapy as a possible way to correct the muscle deterioration, but hurdles such as the need to treat all muscles in the body, including both skeletal and heart muscle, have challenged researchers looking for an effective therapy until now.

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Gene therapy restores vision to mice with retinal degeneration

Technique may someday treat retinitis pigmentosa, macular degeneration

Massachusetts General Hospital (MGH) researchers have used gene therapy to restore useful vision to mice with degeneration of the light-sensing retinal rods and cones, a common cause of human blindness. Their report, appearing in the Oct. 14 Proceedings of the National Academy of Sciences, describes the effects of broadly expressing a light-sensitive protein in other neuronal cells found throughout the retina.

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New safer way to make stem cells reported

Harvard scientists yesterday reported a new way to turn adult cells into stem cells, without using harmful viruses that can cause cancer.

Using a type of virus employed in gene therapy to deliver genes to mouse cells, researchers were able to transform adult cells into embryonic-like stem cells - capable of developing into any cell in the body. That virus had not previously been used in stem cell production.

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Gene Therapy For Chronic Pain trial begins

University of Michigan scientists are beginning a phase 1 clinical trial for the treatment of cancer-related pain, using a novel gene transfer vector injected into the skin to deliver a pain-relieving gene to the nervous system.

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