TY  - GEN 
AU  - Gutierrez-Guerrero, A
AU  - Sanchez-Gilabert, Almudena
AU  - Galvani, G
AU  - Pinedo-Gomez, J
AU  - Sanchez-Hernandez, Sabina
AU  - Martin-Guerra, Rocio
AU  - Cobo, Mariem
AU  - Gregory, Phyllips
AU  - Holmes, Mickael
AU  - Benabdellah, Karim
AU  - Martin, Francisco
PY  - 2018
UR  - https://hdl.handle.net/10481/101722
AB  - Primary immunodeficiencies, including Wiskott-Aldrich syndrome (WAS), are a main target for genome-editing strategies using specific nucleases (SNs) because a small number of corrected hematopoietic stem cells could cure patients. In this work, we...
LA  - eng
PB  - MARY ANN LIEBERT
KW  - Lentiviral vectors
KW  - gene therapy
KW  - Edition génica
KW  - CRISPR
KW  - Zinc-Finger Nucleases
KW  - Wiskott-Aldrich syndrome
KW  - rare deiseases
TI  - Comparison of ZFNs versus CRISPR specific nucleases for genome edition of the Wiskott-Aldrich Syndrome locus
DO  - 10.1089/hum.2017.047
ER  -