TY  - JOUR
AU  - Diez, Begoña
AU  - Genovese, Pietro
AU  - Roman-Rodriguez, Francisco J
AU  - Alvarez, Lara
AU  - Schiroli, Giulia
AU  - Ugalde, Laura
AU  - Rodriguez Perales, Sandra
AU  - Sevilla, Julian
AU  - Diaz de Heredia, Cristina
AU  - Holmes, Michael C
AU  - Lombardo, Angelo
AU  - Naldini, Luigi
AU  - Bueren, Juan Antonio
AU  - Rio, Paula
PY  - 2017
DO  - 10.15252/emmm.201707540
SN  - 1757-4676
UR  - http://hdl.handle.net/20.500.12105/7133
AB  - 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...
LA  - eng
PB  - Wiley
KW  - CD34+ cells
KW  - Fanconi anemia
KW  - Gene editing
KW  - Hematopoietic stem and progenitor cells
KW  - Zinc finger nucleases
KW  - Animals
KW  - Antigens, CD34
KW  - Base Sequence
KW  - Cells, Cultured
KW  - Dependovirus
KW  - Fanconi Anemia
KW  - Fanconi Anemia Complementation Group A Protein
KW  - Fetal Blood
KW  - Gene Editing
KW  - Genetic Vectors
KW  - Hematopoietic Stem Cell Transplantation
KW  - Hematopoietic Stem Cells
KW  - Humans
KW  - Mice
KW  - Mice, Inbred NOD
KW  - Mice, SCID
KW  - Mice, Transgenic
KW  - Reactive Oxygen Species
KW  - Zinc Finger Nucleases
TI  - Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients
TY  - journal article
ER  -