Journal
TRENDS IN NEUROSCIENCES
Volume 27, Issue 5, Pages 235-238Publisher
ELSEVIER SCIENCE LONDON
DOI: 10.1016/j.tins.2004.03.002
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Funding
- NINDS NIH HHS [R37 NS027036] Funding Source: Medline
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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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