Safety Study of Mini-dystrophin Gene to Treat Duchenne Muscular Dystrophy
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|ClinicalTrials.gov Identifier: NCT00428935|
Recruitment Status : Completed
First Posted : January 30, 2007
Last Update Posted : February 5, 2013
Nationwide Children's Hospital
Asklepios Biopharmaceutical, Inc.
Information provided by (Responsible Party):
Jerry R. Mendell, Nationwide Children's Hospital
No Study Results Posted on ClinicalTrials.gov for this Study
|Recruitment Status :||Completed|
|Actual Primary Completion Date :||March 2009|
|Actual Study Completion Date :||July 2010|
Publications of Results:
Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. Dystrophin immunity in Duchenne's muscular dystrophy. N Engl J Med. 2010 Oct 7;363(15):1429-37. doi: 10.1056/NEJMoa1000228.
Bowles DE, McPhee SW, Li C, Gray SJ, Samulski JJ, Camp AS, Li J, Wang B, Monahan PE, Rabinowitz JE, Grieger JC, Govindasamy L, Agbandje-McKenna M, Xiao X, Samulski RJ. Phase 1 gene therapy for Duchenne muscular dystrophy using a translational optimized AAV vector. Mol Ther. 2012 Feb;20(2):443-55. doi: 10.1038/mt.2011.237. Epub 2011 Nov 8.
Watchko J, O'Day T, Wang B, Zhou L, Tang Y, Li J, Xiao X. Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice. Hum Gene Ther. 2002 Aug 10;13(12):1451-60.