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Targeted gene therapy and cell reprogramming in Fanconi anemia

Gene targeting is progressively becoming a realistic therapeutic alternative in clinics. It is unknown, however, whether this technology will be suitable for the treatment of DNA repair deficiency syndromes such as Fanconi anemia (FA), with defects in homology-directed DNA repair. In this study, we...

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Detalles Bibliográficos
Autores principales: Rio, Paula, Baños, Rocio, Lombardo, Angelo, Quintana-Bustamante, Oscar, Alvarez, Lara, Garate, Zita, Genovese, Pietro, Almarza, Elena, Valeri, Antonio, Díez, Begoña, Navarro, Susana, Torres, Yaima, Trujillo, Juan P, Murillas, Rodolfo, Segovia, Jose C, Samper, Enrique, Surralles, Jordi, Gregory, Philip D, Holmes, Michael C, Naldini, Luigi, Bueren, Juan A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Blackwell Publishing Ltd 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4203359/
https://www.ncbi.nlm.nih.gov/pubmed/24859981
http://dx.doi.org/10.15252/emmm.201303374