Use of a Lower Dosage Liver-Detargeted AAV Vector to Prevent Hamster Muscular Dystrophy

The BIO14.6 hamster carries a mutation in the delta sarcoglycan gene causing muscular dystrophy and cardiomyopathy. The disease can be prevented by systemic delivery of delta sarcoglycan cDNA using adeno-associated viruses (AAVs). However, all AAVs also target the liver, raising concerns about their...

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Main Authors: Rotundo, Ida Luisa, Lancioni, Alessio, Savarese, Marco, D'Orsi, Luca, Iacomino, Michele, Nigro, Gerardo, Piluso, Giulio, Auricchio, Alberto, Nigro, Vincenzo
Format: Artigo
Language:Inglês
Published: Mary Ann Liebert, Inc. 2013
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Research Articles
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3631017/
https://ncbi.nlm.nih.gov/pubmed/23427808
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.121
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