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  Vol. 291 No. 23, June 16, 2004 TABLE OF CONTENTS
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Gene Therapy and ALS

Joan Stephenson, PhD

JAMA. 2004;291:2809.

Since this article does not have an abstract, we have provided the first 150 words of the full text and any section headings.

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.

The VEGF treatment increased the animals' life expectancy by 30%, with no apparent toxic effects. "We believe that this approach may have potential as a safe and practical treatment for many of . . . [Full Text of this Article]



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