Somatic Correction of Junctional Epidermolysis Bullosa by a Highly Recombinogenic AAV Variant

Definitive correction of disease causing mutations in somatic cells by homologous recombination (HR) is an attractive therapeutic approach for the treatment of genetic diseases. However, HR-based somatic gene therapy is limited by the low efficiency of gene targeting in mammalian cells and replicati...

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Bibliographic Details
Main Authors: Melo, Sandra P, Lisowski, Leszek, Bashkirova, Elizaveta, Zhen, Hanson H, Chu, Kirk, Keene, Douglas R, Marinkovich, M Peter, Kay, Mark A, Oro, Anthony E
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2014
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Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3982486/
https://ncbi.nlm.nih.gov/pubmed/24390279
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.290
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