Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes

Clonal gene therapy protocols based on the precise manipulation of epidermal stem cells require highly efficient gene-editing molecular tools. We have combined adeno-associated virus (AAV)-mediated delivery of donor template DNA with transcription activator-like nucleases (TALE) expressed by adenovi...

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Detalhes bibliográficos
Publicado no:Mol Ther Nucleic Acids
Main Authors: Chamorro, Cristina, Mencía, Angeles, Almarza, David, Duarte, Blanca, Büning, Hildegard, Sallach, Jessica, Hausser, Ingrid, Del Río, Marcela, Larcher, Fernando, Murillas, Rodolfo
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5014520/
https://ncbi.nlm.nih.gov/pubmed/27045209
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2016.19
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