ACID SPHINGOMYELINASE AND NIEMANN PICK DISEASE

$0M01FY2000RRNIH

Mount Sinai School Of Medicine Of Cuny, New York NY

Investigators

Linked publications & trials

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Abstract

The overall objectives of the proposed research are to: 1) characterize the bio-chemical and molecular genetics of human acid sphingomyelinase, the lysosomal hydrolase deficient in the neuronopathic (Type A) and non-neuronopathic (Type B) forms of Niemann-Pick disease (NPD), and 2) use the murine model of NPD for the development and evaluation of enzyme replacement and somatic gene transfer strategies for the treatment of this lysosomal storage disease.

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