Preclinical safety and efficacy of lentiviral-mediated gene therapy for leukocyte adhesion deficiency type I by Cristina Mesa-Núñez, Carlos Damián, María Fernández-García, Begoña Díez, Gayatri Rao, Jonathan D. Schwartz, Ken M. Law, Julián Sevilla, Paula Río, Rosa Yáñez, Juan A. Bueren, Elena Almarza

Get full text

Therapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients by Begoña Diez, Pietro Genovese, Francisco J Roman‐Rodriguez, Lara Alvarez, Giulia Schiroli, Laura Ugalde, Sandra Rodriguez‐Perales, Julian Sevilla, Cristina Diaz de Heredia, Michael C Holmes, Angelo Lombardo, Luigi Naldini, Juan Antonio Bueren, Paula Rio

Get full text

Targeted gene therapy and cell reprogramming in Fanconi anemia by 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, Juan A Bueren

Get full text