Gene Therapy Restores the Transcriptional Program of Hematopoietic Stem Cells in Fanconi Anemia
Miren Lasaga, Paula Rı́o, Amaia Vilas‐Zornoza, Núria Planell, Susana Navarro, Diego Alignani, Beatriz Fernández-Varas, Daniel Mouzo, Josune Zubicaray, Roser M. Pujol, Eileen Nicoletti, Jonathan Schwartz, Julián Sevilla, Marina Ainciburu, Asier Ullate‐Agote, , Juan Antonio Bueren
This study shows gene therapy can restore the transcriptional program of hematopoietic stem cells in Fanconi Anemia patients, making them resemble healthy donor cells.
Haematologica · Apr 6, 2023Peer-reviewedGenetic & Genomic Medicine