ASGT Press Release 

New research on the progress of gene therapy presented at the ASGT 7th Annual Meeting

Gene therapy successful in treating neurodegenerative disease

MINNEAPOLIS - For the first time, a dominant neurodegenerative disease has been successfully treated using gene therapy, according to a study presented today at the 7th Annual Meeting of the American Society of Gene Therapy (ASGT).

A research team led by Beverly Davidson, University of Iowa, investigated gene silencing by RNA interference (RNAi) as a potential therapy for Spinocerebella ataxia type 1 (SCA1), a dominant neurodegenerative disease caused by the expansion of the polyglutamine tract within the gene called ataxin-1. These are the same mechanisms underlying Huntington's disease, an inherited degenerative neuropsychiatric disorder which affects both body and mind.

Using RNAi expressed from within Adeno Associated Virus (AAV) vectors, researchers showed anatomical, pathological and functional protection from the inherited neurodegeneration in SCA1 transgenic animals. AAV vectors are present in many humans, but have never been associated with any disease, making them an excellent gene transfer vehicle.

The research provides hope for rapidly progressing towards a clinical trial for inherited dominant neurodegenerative diseases such as SCA1 and Huntington's disease.

Researchers explore effective gene transfer method

Researchers have found that a single IV injection of a new gene delivery vehicle will effectively deliver genes to the majority of muscle cells in the body, including those within the heart muscle. These findings were presented today at the 7 th Annual Meeting of the American Society of Gene Therapy (ASGT).

The results may provide a practical solution to the problem of how one might deliver gene therapy for the most common form of muscular dystrophy, a disease that causes progressive weakness of most of the muscles, including the heart.

Paul Gregorevic, PhD, and colleagues from the Muscular Dystrophy Co-operative Research Center at the University of Washington used a newly characterized harmless virus, called adeno-associated virus type 6 (vAAV6) in a new manner. Instead of doing a large number of injections into individual muscles, researchers were able to get a similar result by injecting a single bolus of the rAAV6 into a vein of a mouse. The virus was then able to spread through the bloodstream to deliver genes to the heart and most of the other muscles throughout the body.

Previous attempts at such a gene therapy in mice approached the problem by doing scores or even hundreds of injections into all of the various muscle groups, including the diaphragm and muscles in the trunk and limbs.

Studies are currently underway to demonstrate the corrective effects in mice with muscular dystrophy and could potentially improve the prospects that a practical treatment for the disease will emerge within the next several years.

Researchers develop novel vaccine concept for treating HIV

A group of researchers have developed a novel vaccine concept for HIV using cytolytic T lymphocytes, according to a study presented today at the 7th Annual Meeting of the American Society of Gene Therapy (ASGT).

Cytolytic T lymphocytes (CTL) are capable of killing cells that are infected with a virus by recognizing pieces of the virus that are displayed on the surface of the infected cell.

Hildegund C.J. Ertl, MD, and colleagues from the Wistar Institute in Philadelphia, used an adenovirus vector from a chimpanzee to deliver the gene encoding of HIV protein gag in a mouse. A single administration of the vector resulted in the generation of CTL, which initially declined, then increased. This suggested that the CTL had encountered additional or continued expression of antigen that caused their number to increase.

The American Society of Gene Therapy is the largest medical professional organization representing researchers and scientists dedicated to discovering new gene therapies. ASGT was established in 1996, and has grown to nearly 3,000 members. It is committed to promoting and fostering the general field of research involving gene therapy and to promoting professional and public education in all areas of gene therapy.

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