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Targeted gene therapy and cell reprogramming in Fanconi anemia

Abstract 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 s...

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Principais autores: Paula Rio, Rocio Baños, Angelo Lombardo, Oscar Quintana‐Bustamante, Lara Alvarez, Zita Garate, Pietro Genovese, Elena Almarza, Antonio Valeri, Begoña Díez, Susana Navarro, Yaima Torres, Juan P Trujillo, Rodolfo Murillas, Jose C Segovia, Enrique Samper, Jordi Surralles, Philip D Gregory, Michael C Holmes, Luigi Naldini, Juan A Bueren
Format: Artigo
Jezik:Inglês
Izdano: Springer Nature 2014-05-01
Serija:EMBO Molecular Medicine
Teme:
cell reprogramming
Fanconi anemia
gene‐targeting
iPSCs
zinc finger nucleases
Online dostop:https://doi.org/10.15252/emmm.201303374
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