Clinically Relevant Correction of Recessive Dystrophic Epidermolysis Bullosa by Dual sgRNA CRISPR/Cas9-Mediated Gene Editing

Gene editing constitutes a novel approach for precisely correcting disease-causing gene mutations. Frameshift mutations in COL7A1 causing recessive dystrophic epidermolysis bullosa are amenable to open reading frame restoration by non-homologous end joining repair-based approaches. Efficient targete...

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Detalles Bibliográficos
Publicado en:Mol Ther
Main Authors: Bonafont, Jose, Mencía, Ángeles, García, Marta, Torres, Raúl, Rodríguez, Sandra, Carretero, Marta, Chacón-Solano, Esteban, Modamio-Høybjør, Silvia, Marinas, Lucía, León, Carlos, Escamez, María J., Hausser, Ingrid, Del Río, Marcela, Murillas, Rodolfo, Larcher, Fernando
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Gene & Cell Therapy 2019
Assuntos:
Original Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC6520462/
https://ncbi.nlm.nih.gov/pubmed/30930113
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2019.03.007
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