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1st appeared 15 September 1998 Breakthrough in Gene Therapy Offers Hope for Some Neurodegenerative Diseases A study co-authored by UCSF researchers offers the first evidence that a gene therapy technique involving ribozymes, molecules that disrupt protein production, can be used to slow neurodegeneration in an animal model, signaling a possible approach for overcoming one of the monumental hurdles of gene therapy. In the study, reported in the August issue of Nature Medicine, researchers demonstrated that ribozymes could be used to reduce the production of a faulty gene product that leads to the degeneration of light-sensitive rod cells in the eye. The degeneration occurs in association with autosomal dominant retinitis pigmentosa, an untreatable condition of progressive blindness. Importantly, the approach could potentially also be used to treat other diseases resulting from a destructive protein, including some forms of glaucoma, Huntington's disease, amyotrophic lateral sclerosis (ALS) and Warfan's syndrome, according to the senior author of the study, Matthew M. LaVail, a professor of anatomy and ophthalmology at UCSF. The finding represents an important breakthrough in the still fledgling field of gene therapy, which has been particularly challenged by disorders caused by abnormal, or mutant, proteins, so-called autosomal dominant diseases, said LaVail. Links: source: Jennifer O'Brien, News Services |
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