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The World in Medicine
June 16, 2004

Gene Therapy and ALS

JAMA. 2004;291(23):2809. doi:10.1001/jama.291.23.2809-d

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 of paralysis.

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