Molecular BiologybioRxivPreprint
Therapeutic gene editing in hematopoietic progenitor cells from a mouse model of Fanconi anemia
María José Pino-Barrio, Yari A. Gimenez, M Villanueva…·Susana Navarro
This paper demonstrates efficient gene editing in FA mouse models using TALENs and a donor template at a safe harbor locus, achieving phenotypic correction of FA cells.
DOI 10.1101/362251