Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice-Reference-Cited by-同舟云学术

Delivery of an Adeno-Associated Virus Vector into Cerebrospinal Fluid Attenuates Central Nervous System Disease in Mucopolysaccharidosis Type II Mice

Published:2016-11 Issue:11 Volume:27 Page:906-915
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Hinderer Christian¹,Katz Nathan¹,Louboutin Jean-Pierre²,Bell Peter¹,Yu Hongwei¹,Nayal Mohamad¹,Kozarsky Karen³,O'Brien W. Timothy⁴,Goode Tamara¹,Wilson James M.¹

Affiliation:

1. Gene Therapy Program, Department of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania

2. Section of Anatomy, Department of Basic Medical Sciences, University of West Indies, Kingston, Jamaica

3. REGENXBIO, Rockville, Maryland

4. Department of Neuroscience, University of Pennsylvania, Philadelphia, Pennsylvania.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2016.101

Reference28 articles.

1. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy

2. Molecular basis of mucopolysaccharidosis type II: Mutations in the iduronate-2-sulphatase gene

3. Effect of Hunter disease (mucopolysaccharidosis type II) mutations on molecular phenotypes of iduronate-2-sulfatase: Enzymatic activity, protein processing and structural analysis

4. Recognition and receptor-mediated uptake of a lysosomal enzyme, α-l-iduronidase, by cultured human fibroblasts

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