Gene Therapy for Mucopolysaccharidosis Type VI Is Effective in Cats Without Pre-Existing Immunity to AAV8

Liver gene transfer with adeno-associated viral (AAV) 2/8 vectors is being considered for therapy of systemic diseases like mucopolysaccharidosis type VI (MPS VI), a lysosomal storage disease due to deficiency of arylsulfatase B (ARSB). We have previously reported that liver gene transfer with AAV2/...

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Main Authors: Ferla, Rita, O'Malley, Thomas, Calcedo, Roberto, O'Donnell, Patricia, Wang, Ping, Cotugno, Gabriella, Claudiani, Pamela, Wilson, James M., Haskins, Mark, Auricchio, Alberto
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2013
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3581049/
https://ncbi.nlm.nih.gov/pubmed/23194248
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.179
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