TY  - JOUR
AU  - Rodríguez-Merchan, E. C.
AU  - De Pablo Moreno, Juan Andrés
AU  - Liras, Antonio
PY  - 2021
DO  - 10.3390/ijms22147647
SN  - 1661-6596, ESSN: 1422-0067
UR  - https://hdl.handle.net/20.500.14352/4757
T2  - International Journal of Molecular Sciences
AB  - Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring...
LA  - eng
M2  - 1
PB  - MDPI
KW  - Hemophilia
KW  - Advanced therapies
KW  - Gene therapy
KW  - FVIII transgene
KW  - FIX transgene
KW  - Adenoassociated virus
KW  - Lentiviral vectors
TI  - Gene therapy in hemophilia: recent advances
TY  - journal article
VL  - 22
ER  -