Retroviral-mediated transfer of the human acid sphingomyelinase cDNA: correction of the metabolic defect in cultured Niemann-Pick disease cells.

Types A and B Niemann-Pick disease (NPD) result from inherited deficiencies of the lysosomal hydrolase, acid sphingomyelinase (ASM; sphingomyelin cholinephosphohydrolase, EC 3.1.4.12). To evaluate the feasibility of somatic gene therapy for the treatment of these disorders, retroviral-mediated gene...

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Main Authors: Suchi, M, Dinur, T, Desnick, R J, Gatt, S, Pereira, L, Gilboa, E, Schuchman, E H
Formato: Artigo
Idioma:Inglês
Publicado: 1992
Assuntos:
Research Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC48839/
https://ncbi.nlm.nih.gov/pubmed/1565614
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