Gene therapy for ALS delivers

• Published in: Trends in neurosciences (Regular ed.) Vol. 27; no. 5; pp. 235 - 238
• Main Authors: Boillée, Séverine, Cleveland, Don W.
• Format: Journal Article
• Language: English
• Published: Oxford Elsevier Ltd 01.05.2004; Elsevier Science; Elsevier Sequoia S.A
• 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; Human; Neurotrophin; Nervous system diseases; Rodentia; Motor neuron disease; Transgenic animal; Amyotrophic lateral sclerosis; Motor neuron; Review; Insulin like growth factor 1; Genetic disease; Vertebrata; Mammalia; Treatment; Mouse; Animal; Central nervous system disease; Degenerative disease; Gene therapy; Spinal cord disease
• ISSN: 0166-2236; 1878-108X
• DOI: 10.1016/j.tins.2004.03.002

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.