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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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
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2012
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Original Article
Online dostop: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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