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

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

Abstract 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,...

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Main Authors: Begoña Diez, Pietro Genovese, Francisco J Roman‐Rodriguez, Lara Alvarez, Giulia Schiroli, Laura Ugalde, Sandra Rodriguez‐Perales, Julian Sevilla, Cristina Diaz de Heredia, Michael C Holmes, Angelo Lombardo, Luigi Naldini, Juan Antonio Bueren, Paula Rio
Format: Article
Language:English
Published: Springer Nature 2017-09-01
Series:EMBO Molecular Medicine
Subjects:
CD34+ cells
Fanconi anemia
gene editing
hematopoietic stem and progenitor cells
zinc finger nucleases
Online Access:https://doi.org/10.15252/emmm.201707540
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https://doi.org/10.15252/emmm.201707540

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