Therapeutic gene editing in CD34(+) hematopoietic progenitors from Fanconi anemia patients

Gene targeting constitutes a new step in the development of gene therapy for inherited diseases. Although previous studies have shown the feasibility of editing fibroblasts from Fanconi anemia (FA) patients, here we aimed at conducting therapeutic gene editing in clinically relevant cells, such as h...

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Detalles Bibliográficos
Autores principales: Diez, Begoña, Genovese, Pietro, Roman‐Rodriguez, Francisco J, Alvarez, Lara, Schiroli, Giulia, Ugalde, Laura, Rodriguez‐Perales, Sandra, Sevilla, Julian, Diaz de Heredia, Cristina, Holmes, Michael C, Lombardo, Angelo, Naldini, Luigi, Bueren, Juan Antonio, Rio, Paula
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5666315/
https://www.ncbi.nlm.nih.gov/pubmed/28899930
http://dx.doi.org/10.15252/emmm.201707540

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