Therapeutic gene editing in hematopoietic progenitor cells from a mouse model of Fanconi anemia
María José Pino-Barrio, Yari A. Gimenez, M Villanueva, Marcus Hildenbeutel, Rebeca Sánchez‐Domínguez, Sandra Rodríguez, Roser M. Pujol, Jordi Surrallés, Paula Rı́o, Toni Cathomen, Claudio Mussolino, Juan Antonio Bueren, 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.
bioRxiv · Jul 5, 2018 · PreprintMolecular Biology