Retroviral Mediated Transfer of the cDNA for Human Glucocerebrosidase into Hematopoietic Stem Cells of Patients with Gaucher Disease. A Phase I Study. National Institutes of Health, Bethesda, Maryland
Author:
Affiliation:
1. CHB, NHLBI
2. Childrens Hospital of Los Angeles
3. NINDS
4. DTM, NIH
5. NHLBI
6. University of Southern California
7. Genetic Therapy, Inc.
Publisher
Mary Ann Liebert Inc
Subject
Genetics,Molecular Biology,Molecular Medicine
Link
http://www.liebertpub.com/doi/pdf/10.1089/hum.1996.7.2-231
Reference41 articles.
1. Gaucher's Disease
2. Metabolism of glucocerebrosides II. Evidence of an enzymatic deficiency in Gaucher's disease
3. Demonstration of a deficiency of glucocerebroside-cleaving enzyme in Gaucher's disease.
4. BENEFICIAL EFFECT OF PRE-TRANSPLANT SPLENECTOMY ON DISPLACEMENT BONE MARROW TRANSPLANTATION FOR GAUCHER'S SYNDROME
5. LONG-TERM FOLLOW-UP OF THE FIRST SUCCESSFUL BONE MARROW TRANSPLANTATION IN GAUCHER DISEASE
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