Merits of Combination Cortical, Subcortical, and Cerebellar Injections for the Treatment of Niemann-Pick Disease Type A

Niemann-Pick disease Type A (NPA) is a neuronopathic lysosomal storage disease (LSD) caused by the loss of acid sphingomyelinase (ASM). The goals of the current study are to ascertain the levels of human ASM that are efficacious in ASM knockout (ASMKO) mice, and determine whether these levels can be...

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Detalhes bibliográficos
Main Authors: Bu, Jie, Ashe, Karen M, Bringas, John, Marshall, John, Dodge, James C, Cabrera-Salazar, Mario A, Forsayeth, John, Schuchman, Edward H, Bankiewicz, Krystof S, Cheng, Seng H, Shihabuddin, Lamya S., Passini, Marco A
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2012
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3464981/
https://ncbi.nlm.nih.gov/pubmed/22828503
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.118
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