Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann-Pick type A disease

Niemann-Pick type A disease is a lysosomal storage disorder caused by a deficiency in acid sphingomyelinase (ASM) activity. Previously we showed that storage pathology in the ASM knockout (ASMKO) mouse brain can be corrected by adeno-associated virus serotype 2 (AAV2)-mediated gene transfer. The pre...

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Détails bibliographiques
Auteurs principaux: Dodge, James C., Clarke, Jennifer, Song, Antonius, Bu, Jie, Yang, Wendy, Taksir, Tatyana V., Griffiths, Denise, Zhao, Michael A., Schuchman, Edward H., Cheng, Seng H., O'Riordan, Catherine R., Shihabuddin, Lamya S., Passini, Marco A., Stewart, Gregory R.
Format: Artigo
Langue:Inglês
Publié: National Academy of Sciences 2005
Sujets:
Biological Sciences
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC1308930/
https://ncbi.nlm.nih.gov/pubmed/16301517
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0509062102
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