Infusion of recombinant human acid sphingomyelinase into Niemann‐Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology
Open Access
- 1 October 2000
- journal article
- research article
- Published by Wiley in The FASEB Journal
- Vol. 14 (13) , 1988-1995
- https://doi.org/10.1096/fj.00-0014com
Abstract
An inherited deficiency of acid sphingomyelinase (ASM) activity results in the Type A and B forms of Niemann-Pick disease (NPD). Using the ASM-deficient mouse model (ASMKO) of NPD, we evaluated the e...Keywords
Funding Information
- National Institutes of Health (HD 28607, HD 32654)
- United States - Israel Binational Science Foundation (93-00015)
- National Center for Research Resources (RR 0071)
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