Lysosomal Storage Disease

Transplanted neural progenitor cells decrease lysosomal storage pathology

Published in Gene Therapy Weekly, February 17th, 2005

Transplanted neural progenitor cells decrease lysosomal storage pathology.

"Niemann-Pick disease is caused by a genetic deficiency in acid sphingomyelinase (ASM) leading to the intracellular accumulation of sphingomyelin and cholesterol in lysosomes. In the present study, we evaluated the effects of direct intracerebral transplantation of neural progenitor cells (NPCs) on the brain storage pathology in the ASM knock-out (ASMKO) mouse model of Type A Niemann-Pick disease," scientists in the United States report.

"NPCs derived from adult mouse brain were genetically modified to express human ASM (hASM) and were transplanted into multiple regions of the...

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Source: Gene Therapy Weekly (2005-02-17)

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