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Review : Gene Therapy: Applications to the Neurosciences and to Neurological Disease

Department of Neurosciences University of California-San Diego La Jolla, California Veterans Affairs Medical Center San Diego, California

Gene expression is involved in some way in every human disease. As our knowledge of gene structure and function has blossomed in the last 2 decades, so too has our potential genetically-based repertoire for combating disease. Gene therapy refers to the manipulation of gene expression, either by augmenting the expression of therapeutic genes or by diminishing the expression of deleterious genes. In some neurological diseases, such as trauma, ischemia, and neurodegenerative disorders, gene therapy might be used to express genes for such substances as growth factors or neurotransmitters to prevent neuronal degeneration or to compensate for lost function, respectively. In other cases, gene therapy could be used to block the expression of genes that cause disease such as ß-amyloid precursor protein or the Huntingtin gene. In inherited diseases of the nervous system such as muscular dystrophy, normal gene copies could be placed into the nervous system to compensate for lost function resulting from abnormal gene expression. The tools for achieving well-targeted, sustained, and safe gene delivery in the nervous system are now becoming available, and this technology is likely to substantially alter the nature of neurological therapy in the future. NEUROSCIENTIST 4:398-407, 1998

Key Words: KEY WORDS Gene therapy • Alzheimer disease • Spinal cord injury • Parkinson disease • Cancer • Retrovirus • Adeno-associated virus

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