Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease

Author:

Mah Cathryn S,Falk Darin J,Germain Sean A,Kelley Jeffry S,Lewis Melissa A,Cloutier Denise A,DeRuisseau Lara R,Conlon Thomas J,Cresawn Kerry O,Fraites Jr Thomas J,Campbell-Thompson Martha,Fuller David D,Byrne Barry J

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference49 articles.

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3. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein;Kessler;Proc Natl Acad Sci USA,1996

4. Fatigue: an important feature of late-onset Pompe disease;Hagemans;J Neurol,2007

5. Acid α-glucosidase deficiency (glycogenosis type II, Pompe disease;Raben;Curr Mol Med,2002

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