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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Bibliografiske detaljer
Udgivet i:EMBO Mol Med
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2017
Fag:
Research Articles
Online adgang: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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