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 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.
Boillée
Séverine
S
Ludwig Institute for Cancer Research and Departments of Cellular and Molecular Medicine and Neurosciences, University of California, 9500 Gilman Drive, La Jolla, CA 92093-0670, USA.
Cleveland
Don W
DW
D000690:Amyotrophic Lateral Sclerosis / Q000628:therapy*; D000818:Animals; D002986:Clinical Trials as Topic; D000229:Dependovirus; D004195:Disease Models, Animal; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic; D009046:Motor Neurons / Q000187:drug effects; D013002:Somatomedins / Q000627:therapeutic use
D013002:Somatomedins