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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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Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:EMBO Mol Med
Egile Nagusiak: 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
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: John Wiley and Sons Inc. 2017
Gaiak:
Sarrera elektronikoa: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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