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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Vydáno v:EMBO Mol Med
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2017
Témata:
Research Articles
On-line přístup: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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