Widespread Correction of Lysosomal Storage Following Intrahepatic Injection of a Recombinant Adeno-associated Virus in the Adult MPS VII Mouse-Reference-Cited by-同舟云学术

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Widespread Correction of Lysosomal Storage Following Intrahepatic Injection of a Recombinant Adeno-associated Virus in the Adult MPS VII Mouse

Published:2004-09 Issue:3 Volume:10 Page:478-491
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Sferra Thomas J.,Backstrom Kristin,Wang Chuansong,Rennard Rachel,Miller Matt,Hu Yan

Publisher

Elsevier BV

Subject

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

Reference53 articles.

1. The mucopolysaccharidoses;Neufeld,2001

2. Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines;Peters;Bone Marrow Transplant,2003

3. Enzyme replacement therapy for the mucopolysaccharide storage disorders;Kakkis;Expert Opin. Invest. Drugs,2002

4. β-Glucuronidase deficiency: report of clinical, radiologic and biochemical features of a new mucopolysaccharidosis;Sly;J. Pediatr,1973

5. Murine mucopolysaccharidosis type VII: characterization of a mouse with β-glucuronidase deficiency;Birkenmeier;J. Clin. Invest,1989

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