Gene therapy for ALS delivers
Amyotrophic lateral sclerosis (ALS) is a fatal, progressive neurodegenerative disease that kills motor neurons. Despite a long disappointing history of human trials with neurotrophins, including insulin-like growth factor 1 (IGF-1), Kaspar and colleagues have successfully slowed disease in transgeni...
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| Veröffentlicht in: | Trends in neurosciences (Regular ed.) 2004-05, Vol.27 (5), p.235-238 |
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| container_issue | 5 |
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| container_title | Trends in neurosciences (Regular ed.) |
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| creator | Boillée, Séverine Cleveland, Don W. |
| description | Amyotrophic lateral sclerosis (ALS) is a fatal, progressive neurodegenerative disease that kills motor neurons. Despite a long disappointing history of human trials with neurotrophins, including insulin-like growth factor 1 (IGF-1), Kaspar and colleagues have successfully slowed disease in transgenic ALS mice by forcing motor neurons to produce IGF-1 following retrograde delivery of recombinant adeno-associated virus (AAV) injected into muscle. With the clinical safety of both IGF-1 and AAV already established, this provides real hope for an effective treatment of ALS. |
| doi_str_mv | 10.1016/j.tins.2004.03.002 |
| format | Article |
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| fulltext | fulltext |
| identifier | ISSN: 0166-2236 |
| ispartof | Trends in neurosciences (Regular ed.), 2004-05, Vol.27 (5), p.235-238 |
| issn | 0166-2236 1878-108X |
| language | eng |
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| source | MEDLINE; Access via ScienceDirect (Elsevier) |
| subjects | Amyotrophic lateral sclerosis Amyotrophic Lateral Sclerosis - therapy Animals Biological and medical sciences Clinical Trials as Topic Degenerative and inherited degenerative diseases of the nervous system. Leukodystrophies. Prion diseases Dependovirus Disease Models, Animal Gene therapy Genetic Therapy - methods Humans Medical sciences Mice Mice, Transgenic Motor Neurons - drug effects Neurology Neurons Rodents Somatomedins - therapeutic use |
| title | Gene therapy for ALS delivers |
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