A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy

BACKGROUND: Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder with monogenic mutations setting the stage for successful gene therapy treatment. We have completed a study that directly deals with the following key issues that can be directly adapted to a gene therapy clinical trial...

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Hauptverfasser: Rodino-Klapac, Louise R, Janssen, Paul ML, Montgomery, Chrystal L, Coley, Brian D, Chicoine, Louis G, Clark, K Reed, Mendell, Jerry R
Format: Artigo
Sprache:Inglês
Veröffentlicht: BioMed Central 2007
Schlagworte:
Research
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2082019/
https://ncbi.nlm.nih.gov/pubmed/17892583
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1479-5876-5-45
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