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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Detalhes bibliográficos
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2005
Assuntos:
Biological Sciences
Acesso em linha: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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