Ontology highlight
ABSTRACT:
SUBMITTER: Rio P
PROVIDER: S-EPMC4203359 | biostudies-literature | 2014 Jun
REPOSITORIES: biostudies-literature
Rio Paula P Baños Rocio R Lombardo Angelo A Quintana-Bustamante Oscar O Alvarez Lara L Garate Zita Z Genovese Pietro P Almarza Elena E Valeri Antonio A Díez Begoña B Navarro Susana S Torres Yaima Y Trujillo Juan P JP Murillas Rodolfo R Segovia Jose C JC Samper Enrique E Surralles Jordi J Gregory Philip D PD Holmes Michael C MC Naldini Luigi L Bueren Juan A JA
EMBO molecular medicine 20140406 6
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 used zinc finger nucleases and integrase-defective lentiviral vectors to demonstrate for the first time that FANCA can be efficiently and specifically targeted into the AAVS1 safe harbor locus in fibr ...[more]