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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Библиографические подробности
Опубликовано в: :EMBO Mol Med
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: John Wiley and Sons Inc. 2017
Предметы:
Research Articles
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5666315/
https://ncbi.nlm.nih.gov/pubmed/28899930
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.201707540
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