COL7A1 Editing via CRISPR/Cas9 in Recessive Dystrophic Epidermolysis Bullosa

Designer nucleases allow specific and precise genomic modifications and represent versatile molecular tools for the correction of disease-associated mutations. In this study, we have exploited an ex vivo CRISPR/Cas9-mediated homology-directed repair approach for the correction of a frequent inherite...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Hainzl, Stefan, Peking, Patricia, Kocher, Thomas, Murauer, Eva M., Larcher, Fernando, Del Rio, Marcela, Duarte, Blanca, Steiner, Markus, Klausegger, Alfred, Bauer, Johann W., Reichelt, Julia, Koller, Ulrich
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5675435/
https://ncbi.nlm.nih.gov/pubmed/28800953
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.07.005
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