Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of niemann

Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of niemann–pick disease

Published:2003-12 Issue:6 Volume:8 Page:876-885
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Jin Hee-Kyung,Schuchman Edward H

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

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4. Acid sphingomyelinase-deficient mice mimic the neurovisceral form of human lysosomal storage disease (Niemann–Pick disease);Otterbach;Cell,1995

5. Replacement therapy for inherited enzyme deficiency;Brady;N. Engl. J. Med.,1973

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