GENE THERAPY OF CANAVAN DISEASE

$0M01FY2000RRNIH

Yale University, New Haven CT

Investigators

Linked publications & trials

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Abstract

Treatment with a gene therapy approach for children with the childhood progressive leukodystrophy, Canavan's Disease, will be developed and characterized. This leukodystrophy results in profound psychomotor retardation and spongiform degenereration of the brain, and there is no specific treatment of the disease at present.

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