New findings from a study of a mouse model of amyotrophic lateral sclerosis
(ALS) suggest a possible therapeutic strategy for treating the disease. ALS
usually leads to paralysis and death within 3 to 5 years after onset (Nature. 2004;429:413-417).
Recent studies have indicated that reduced concentrations of vascular
endothelial growth factor (VEGF), a substance involved in angiogenesis and
the protection of neurons, predispose mice and humans to ALS. In the new study,
researchers in England and Belgium reported that a single injection of a genetically
modified virus bearing a gene for VEGF delayed ALS onset and progression in
a strain of ALS-prone mice, even when treatment was started after the onset
Stephenson J. Gene Therapy and ALS. JAMA. 2004;291(23):2809. doi:10.1001/jama.291.23.2809-d
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