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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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.
פורמט: Artigo
שפה:Inglês
יצא לאור: National Academy of Sciences 2005
נושאים:
Biological Sciences
גישה מקוונת: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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