Towards gene therapy for the central nervous system |
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| Affiliation: | 1. Jefferson CNS Gene Therapy Center, Dept of Neurosurgery, Jefferson Medical College, 1025 Walnut Street, Philadelphia, PA 19107, USA.;2. University of Auckland School of Medicine, Auckland, New Zealand.;1. Department of Internal Medicine, Hadassah Medical Center, Jerusalem, Israel;2. Seymour Fox School of Education, The Hebrew University of Jerusalem, Jerusalem, Israel;3. Reproductive Endocrinology and Genetics Unit, Department of Obstetrics and Gynecology, Shaare Zedek Medical Center, Jerusalem, Israel;4. Neuropediatric Unit, Department of Pediatrics, Shaare Zedek Medical Center, Jerusalem, Israel;5. Department of Pediatrics, Hadassah Medical Center, Jerusalem, Israel;1. Department of Pediatrics, Vanderbilt University Medical Center, United States;2. Vanderbilt Kennedy Center for Research on Human Development, United States;3. Vanderbilt University, United States;4. Department of Hearing and Speech Sciences, Vanderbilt University Medical Center, United States;1. Molecular Neurogenetics Unit, IRCCS Foundation Neurological Institute C. Besta, Via L. Temolo 4, 20126 Milan, Italy;2. Department of Pediatric Neurology, IRCCS Foundation Neurological Institute C. Besta, Via Celoria 11, 20133 Milan, Italy;3. Department of Medicine and Surgery, PhD Programme in Molecular and Translational Medicine, University of Milan Bicocca, Via Cadore 48, 20900 Monza, Italy;1. The Sheryl and Daniel R. Tishman Cognitive Neurophysiology Laboratory, Children’s Evaluation and Rehabilitation Center (CERC), Department of Pediatrics, Albert Einstein College of Medicine & Montefiore Medical Center, Bronx, NY 10461, USA;2. Ferkauf Graduate School of Psychology Albert Einstein College of Medicine, Bronx, NY 10461, USA;3. Department of Genetics, Albert Einstein College of Medicine, Bronx, NY 10461, USA;4. Department of Neuroscience, Rose F. Kennedy Intellectual and Developmental Disabilities Research Center, Albert Einstein College of Medicine, Bronx, NY 10461, USA;5. Ernest J. Del Monte Institute for Neuroscience, Department of Neuroscience, University of Rochester School of Medicine and Dentistry, Rochester, NY 14642, USA;1. Reta Lila Weston Institute, UCL Institute of Neurology, London, UK;2. Department of Molecular Neuroscience, UCL Institute of Neurology, London, UK;3. Department of Neurodegenerative Diseases, UCL Institute of Neurology, London, UK;4. Department of Clinical Neuroscience, UCL Institute of Neurology, London, UK;5. Neurogenetics Unit, National Hospital for Neurology and Neurosurgery, London, UK;6. Department of Clinical Neuroscience, King''s College London, Institute of Psychiatry, London, UK;7. Centre for Neuroscience and Trauma, Blizard Institute, Queen Mary University of London, North-East London and Essex Regional Motor Neuron Disease Care Centre, London, UK |
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| Abstract: | Gene therapy has generated enormous scientific, medical and public interest over the last decade. Clinical trials involving approximately 2000 patients worldwide have targeted simple genetic diseases such as cystic fibrosis, muscular dystrophy, adenosine deaminase deficiency, Gaucher's disease and familial hypercholesterolemia, as well as complex acquired diseases such as cancer and AIDS. The central nervous system is a new and particularly exciting target for gene therapy because its unique properties prevent the successful treatment of many neurological disorders by conventional means. This review discusses the potential applications of in vivo gene therapy to neurological disorders that have the greatest potential for genetic treatments. |
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