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Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-Associated Virus 1 Human Acid α-Glucosidase

A biodistribution and toxicology study was performed to test the acute toxicities of intradiaphragmatic injection of a recombinant adeno-associated virus (rAAV) 2/1-human acid alpha-Glucosidase (hGAA) driven by a cytomegalovirus (CMV) promoter (rAAV1-CMV-hGAA) in New Zealand white rabbits and in the...

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Bibliographic Details
Main Authors: Conlon, Thomas J., Erger, Kirsten, Porvasnik, Stacy, Cossette, Travis, Roberts, Cheryl, Combee, Lynn, Islam, Saleem, Kelley, Jeffry, Cloutier, Denise, Clément, Nathalie, Abernathy, Corinne R., Byrne, Barry J.
Format: Artigo
Language:Inglês
Published: Mary Ann Liebert, Inc. 2013
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Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC4003472/
https://ncbi.nlm.nih.gov/pubmed/24021025
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2013.147
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