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Literature DB >> 21194026

Systemic gene transfer to skeletal muscle using reengineered AAV vectors.

Jana L Phillips¹, Julia Hegge, Jon A Wolff, R Jude Samulski, Aravind Asokan.

Abstract

Gene therapy of musculoskeletal disorders warrants efficient gene transfer to a wide range of muscle groups. Reengineered adeno-associated viral (AAV) vectors that selectively transduce muscle tissue following systemic administration are attractive candidates for such applications. Here we provide examples of several lab-derived AAV vectors that display systemic tissue tropism in mice. Methods to evaluate the efficiency of gene transfer to skeletal muscle following intravenous or isolated limb infusion of AAV -vectors in mice are discussed in detail.

Entities: Chemical Disease Gene Species

Mesh：

Substances：
Capsid Proteins

Year: 2011 PMID： 21194026 PMCID： PMC3253371 DOI： 10.1007/978-1-61737-982-6_9

Source DB: PubMed Journal: Methods Mol Biol ISSN： 1064-3745

20 in total

1. A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs.

Authors: James E Hagstrom; Julia Hegge; Guofeng Zhang; Mark Noble; Vladimir Budker; David L Lewis; Hans Herweijer; Jon A Wolff
Journal: Mol Ther Date: 2004-08 Impact factor: 11.454

2. Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity.

Authors: Joseph E Rabinowitz; Fabienne Rolling; Chengwen Li; Hervè Conrath; Weidong Xiao; Xiao Xiao; R Jude Samulski
Journal: J Virol Date: 2002-01 Impact factor: 5.103

3. Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors.

Authors: Cathryn Mah; Christina A Pacak; Kerry O Cresawn; Lara R Deruisseau; Sean Germain; Melissa A Lewis; Denise A Cloutier; David D Fuller; Barry J Byrne
Journal: Mol Ther Date: 2007-01-23 Impact factor: 11.454

4. Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors.

Authors: C Mah; K O Cresawn; T J Fraites; C A Pacak; M A Lewis; I Zolotukhin; B J Byrne
Journal: Gene Ther Date: 2005-09 Impact factor: 5.250

5. Production and characterization of adeno-associated viral vectors.

Authors: Joshua C Grieger; Vivian W Choi; R Jude Samulski
Journal: Nat Protoc Date: 2006 Impact factor: 13.491

Review 6. Pathophysiology of duchenne muscular dystrophy: current hypotheses.

Authors: Nicolas Deconinck; Bernard Dan
Journal: Pediatr Neurol Date: 2007-01 Impact factor: 3.372

7. Sustained whole-body functional rescue in congestive heart failure and muscular dystrophy hamsters by systemic gene transfer.

Authors: Tong Zhu; Liqiao Zhou; Satsuki Mori; Zhong Wang; Charles F McTiernan; Chunping Qiao; Chunlian Chen; Dao Wen Wang; Juan Li; Xiao Xiao
Journal: Circulation Date: 2005-10-17 Impact factor: 29.690

Review 8. AAV-directed muscular dystrophy gene therapy.

Authors: Ying Tang; James Cummins; Johnny Huard; Bing Wang
Journal: Expert Opin Biol Ther Date: 2010-03 Impact factor: 4.388

9. Myodys, a full-length dystrophin plasmid vector for Duchenne and Becker muscular dystrophy gene therapy.

Authors: Dongsheng Duan
Journal: Curr Opin Mol Ther Date: 2008-02

10. Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway.

Authors: Andrew M Davidoff; Catherine Y C Ng; Junfang Zhou; Yunyu Spence; Amit C Nathwani
Journal: Blood Date: 2003-03-13 Impact factor: 22.113

4 in total