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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 and Juan A Bueren Targeted gene therapy and cell reprogramming in Fanconi anemia EMBO Molecular Medicine 6

Version of Record online: 23 MAY 2014 | DOI: 10.15252/emmm.201303374

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This study shows for the first time the possibility of performing targeted gene therapy in a z-repair deficiency syndrome, known as Fanconi anemia. By reprogramming targeted cells, asymptomatic gene-edited iPSCs and hematopoietic progenitor cells are generated.

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