Safety Study of Adeno-Associated Virus Serotype 2-Mediated Human Acid Sphingomyelinase Expression in the Nonhuman Primate Brain-Reference-Cited by-同舟云学术

Safety Study of Adeno-Associated Virus Serotype 2-Mediated Human Acid Sphingomyelinase Expression in the Nonhuman Primate Brain

Published:2012-08 Issue:8 Volume:23 Page:891-902
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Salegio Ernesto A.¹,Samaranch Lluis¹,Jenkins Russell W.²,Clarke Christopher J.²³,Lamarre Clementine¹,Beyer Janine¹,Kells Adrian P.¹,Bringas John¹,Sebastian Waldy San¹,Richardson R. Mark¹,Rosenbluth Kate H.¹,Hannun Yusuf A.²³,Bankiewicz Krystof S.¹,Forsayeth John¹

Affiliation:

1. Department of Neurosurgery, University of California San Francisco, San Francisco, CA 94103.

2. Department of Biochemistry and Molecular Biology, Medical University of South Carolina, Charleston, SC 29425.

3. Present address: Department of Medicine and the Stony Brook University Cancer Center, Stony Brook University, Health Science Center, Stony Brook, NY 11794.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2012.052

Reference36 articles.

1. Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid- -glucosidase

2. High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency

3. Gene therapy for lysosomal storage disorders

4. Simultaneous quantitative analysis of bioactive sphingolipids by high-performance liquid chromatography-tandem mass spectrometry

5. Reversal of Pathology in the Entire Brain of Mucopolysaccharidosis Type VII Mice after Lentivirus-Mediated Gene Transfer

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