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The following appeared in the Oct 3rd 1994 issue of the Durham North Carolina Herald-Sun (Slightly abbreviated): "Two studies involving the UNC medical school provide evidence that gene therapy may help patients with Parkinson's Disease and a blood disorder called Fanconi Anemia. The studies are reported in the Oct.`93 issues of Nature Genetics and the Journal of Clinical Investigation respectively. Our study of Parkinson's Disease is the first demonstration that gene therapy with adeno-associated virus safely can transfer and then express a normal gene to the brain. This is a new delivery approach to gene transfer in the central nervous system" said R. Jude Samulski, director of the UNC at Chapel Hill Gene Therapy Center and associate prof. of Pharmacology. Samulski is a member of the Lineberger Comprehensive Cancer Center and the Program in Molecular Biology and Biotechnology. (paragraph describing PD symptoms deleted) Gene therapy is a form of molecular surgery in which a normally functioning gene is placed into cells with a defective gene, Samulski said. Viruses have been the main tool used to replace defective genes. `Using (adeno- associated virus) to carry properly functioning genes to replace mal- functioning ones has potential for numerous deseases, not just these two', Samulski said. `Unlike many institutions that are concentrating on using a specific viral carrier to treat or potentially cure one disease, we are studying (adeno-associated virus) for use in many different diseases that could be amenable to gene therapy.' Adeno-associated virus has the potential to replace a defective gene for the life of a person. It is safe to use, does not cause disease on its own and can enter dividing and non-dividing cells. Samulski and colleages demonstrated that L-dopamine, a neurotransmitter that is diminished in PD was produced in a rat model of the disease after administering adeno-associated virus. `Compared to a group of control animals, the rats given (adeno-associated virus) had significant behavioral improvement,' he said. When using adeno- associated virus as a viral carrier for human genes, all of the adeno- associated virus genes were removed and replaced with a gene that causes cells to produce L-dopamine when a specific protein is present." A phone call was made to Professor Samulski by Crayton Thompson, one of the leaders of the Raleigh NC PD Support Group. Samulski indicated they may ready for human trials in a year or two. They are working with monkeys at the present time. The NIH, FDA, etc. will review the monkey data and decide when human trials can begin. Entered by Bruce McCallum, [log in to unmask]