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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

Paula Rio,Josune Zubicaray,Susana Navarro,Eva Galvez,Rebeca Sanchez-Dominguez,Eileen Nicoletti,Elena Sebastian,Michael Rothe,Roser Pujol,Massimo Bogliolo,Philipp John-Neek, Antonella Lucia Bastone,Axel Schambach,Wei Wang,Manfred Schmidt,Lise Larcher,Jose C. Segovia,Rosa M. Yanez,Omaira Alberquilla,Begona Diez, Maria Fernandez-Garcia, Laura Garcia-Garcia, Manuel Ramirez, Anne Galy, Francois Lefrere, Marina Cavazzana, Thierry Leblanc, Nagore Garcia de Andoin, Ricardo Lopez-Almaraz, Albert Catala, Jordi Barquinero, Sandra Rodriguez-Perales, Gayatri Rao, Jordi Surralles, Jean Soulier, Cristina Diaz-de-Heredia, Jonathan D. Schwartz, Julian Sevilla, Juan A. Bueren

LANCET(2024)

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