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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Published in | Trends in neurosciences (Regular ed.) Vol. 27; no. 5; pp. 235 - 238 |
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Main Authors | , |
Format | Journal Article |
Language | English |
Published |
Oxford
Elsevier Ltd
01.05.2004
Elsevier Science Elsevier Sequoia S.A |
Subjects | |
Online Access | Get full text |
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Summary: | 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. |
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Bibliography: | ObjectType-Article-2 SourceType-Scholarly Journals-1 ObjectType-Feature-3 content type line 23 ObjectType-Review-1 |
ISSN: | 0166-2236 1878-108X |
DOI: | 10.1016/j.tins.2004.03.002 |