Infusion of recombinant human acid sphingomyelinase into Niemann‐Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology-Reference-Cited by-同舟云学术

Infusion of recombinant human acid sphingomyelinase into Niemann‐Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology

Published:2000-10 Issue:13 Volume:14 Page:1988-1995
ISSN:0892-6638
Container-title:The FASEB Journal
language:en
Short-container-title:FASEB j.

Author:

Miranda Silvia R. P.¹,He Xingxuan¹,Simonaro Calogera M.¹,Gatt Shimon²,Dagan Arie²,Desnick Robert J.¹³,Schuchman Edward H.¹³

Affiliation:

1. Department of Human GeneticsInstitute for Gene Therapy and Molecular MedicineMount Sinai School of MedicineNew York 10029 USA

2. Department of BiochemistryHebrew University‐Hadassah School of MedicineJerusalem Israel 92710

3. Institute for Gene Therapy and Molecular MedicineInstitute for Gene Therapy and Molecular MedicineMount Sinai School of MedicineNew York 10029 USA

Funder

National Institutes of Health

United States - Israel Binational Science Foundation

National Center for Research Resources

Publisher

Wiley

Subject

Genetics,Molecular Biology,Biochemistry,Biotechnology

Link

https://onlinelibrary.wiley.com/doi/pdf/10.1096/fj.00-0014com

Reference39 articles.

1. The metabolism of sphingomyelin. II. Evidence of an enzymatic deficiency in Niemann-Pick diseae.

2. Sphingomyelinase in normal human spleens and in spleens from subjects with Niemann-Pick disease;Schneider P. B.;J. Lipid Res.,1967

3. Inherited Disorders of Lysosomal Metabolism

4. The uptake of enzymes into lysosomes: an overview;Neufeld E. F.;Birth Defects Orig. Artic. Ser.,1980

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