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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Detalles Bibliográficos
Autores principales: Melo, Sandra P, Lisowski, Leszek, Bashkirova, Elizaveta, Zhen, Hanson H, Chu, Kirk, Keene, Douglas R, Marinkovich, M Peter, Kay, Mark A, Oro, Anthony E
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2014
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3982486/
https://www.ncbi.nlm.nih.gov/pubmed/24390279
http://dx.doi.org/10.1038/mt.2013.290

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3982486/
https://www.ncbi.nlm.nih.gov/pubmed/24390279
http://dx.doi.org/10.1038/mt.2013.290

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