TY  - GEN
AU  - Río, Paula
AU  - Zubicaray, Josune
AU  - Navarro, Susana
AU  - Gálvez, Eva
AU  - Sánchez-Domínguez, Rebeca
AU  - Nicoletti, Eileen
AU  - Sebastián, Elena
AU  - Rothe, Michael
AU  - Pujol, Roser
AU  - Bogliolo, Massimo
AU  - John-Neek, Philipp
AU  - Bastone, Antonella Lucía
AU  - Schambach, Axel
AU  - Wang, Wei
AU  - Schmidt, Manfred
AU  - Larcher, Lise
AU  - Segovia, José C
AU  - Yáñez, Rosa M
AU  - Alberquilla, Omaira
AU  - Díez, Begoña
AU  - Fernández-García, María
AU  - García-García, Laura
AU  - Ramírez, Manuel
AU  - Galy, Anne
AU  - Lefrere, Francois
AU  - Cavazzana, Marina
AU  - Leblanc, Thierry
AU  - García de Andoin, Nagore
AU  - López-Almaraz, Ricardo
AU  - Catalá, Albert
AU  - Barquinero, Jordi
AU  - Rodriguez Perales, Sandra
AU  - Rao, Gayatri
AU  - Surrallés, Jordi
AU  - Soulier, Jean
AU  - Díaz-de-Heredia, Cristina
AU  - Schwartz, Jonathan D
AU  - Sevilla, Julián
AU  - Bueren, Juan A
PY  - 2025
UR  - https://hdl.handle.net/20.500.12105/26179
AB  - Allogeneic haematopoietic stem-cell transplantation is the standard treatment for bone marrow failure (BMF) in patients with Fanconi anaemia, but transplantation-associated complications such as an increased incidence of subsequent cancer are...
AB  - This was an open-label, investigator-initiated phase 1/2 clinical trial (FANCOLEN-1) and long-term follow-up trial (up to 7 years post-treatment) in Spain. Mobilised peripheral blood (PB) CD34 cells from nine patients with Fanconi anaemia-A in the...
AB  - There were eight evaluable treated patients with Fanconi anaemia-A. Patients were recruited between Jan 7, 2016 and April 3, 2019. The primary endpoint was met in five of the eight evaluable patients (62·50%). The median number of therapeutic vector...
AB  - These results show for the first time that haematopoietic gene therapy without genotoxic conditioning enables sustained engraftment and reversal of BMF progression in patients with Fanconi anaemia.
AB  - European Commission, Instituto de Salud Carlos III, and Rocket Pharmaceuticals.
LA  - eng
PB  - Elsevier
KW  - CELL TRANSPLANTATION
KW  - CANCER
KW  - ENGRAFTMENT
TI  - Haematopoietic gene therapy of non-conditioned patients with Fanconi anaemia-A: results from open-label phase 1/2 (FANCOLEN-1) and long-term clinical trials.
TY  - research article
ER  -