MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy-Reference-Cited by-同舟云学术

MRI roadmap-guided transendocardial delivery of exon-skipping recombinant adeno-associated virus restores dystrophin expression in a canine model of Duchenne muscular dystrophy

Published:2012-05-03 Issue:3 Volume:20 Page:274-282
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Barbash I M,Cecchini S,Faranesh A Z,Virag T,Li L,Yang Y,Hoyt R F,Kornegay J N,Bogan J R,Garcia L,Lederman R J,Kotin R M

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.nature.com/articles/gt201238.pdf

Reference44 articles.

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2. Emery AE . Population frequencies of inherited neuromuscular diseases--a world survey. Neuromuscul disord 1991; 1: 19–29.

3. Koenig M, Hoffman EP, Bertelson CJ, Monaco AP, Feener C, Kunkel LM et al. Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell 1987; 50: 509–517.

4. Hoffman EP, Brown RH, Kunkel LM . Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell 1987; 51: 919–928.

5. Bushby K, Finkel R, Birnkrant DJ, Case LE, Clemens PR, Cripe L et al. Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management. Lancet Neurol 2010; 9: 77–93.

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