Gene therapy restores the transcriptional program of hematopoietic stem cells in Fanconi anemia

Clinical trials have shown that lentiviral-mediated gene therapy can ameliorate bone marrow failure (BMF) in non-conditioned Fanconi anemia (FA) patients resulting from the proliferative advantage of corrected FA hematopoietic stem and progenitor cells (HSPC). However, it is not yet known if gene th...

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Detalles Bibliográficos
Autores principales: Lasaga, Miren, Río, Paula, Vilas-Zornoza, Amaia, Planell, Nuria, Navarro, Susana, Alignani, Diego, Fernández-Varas, Beatriz, Mouzo, Daniel, Zubicaray, Josune, Pujol, Roser M., Nicoletti, Eileen, Schwartz, Jonathan D., Sevilla, Julián, Ainciburi, Marina, Ullate-Agote, Asier, Surrallés, Jordi, Perona, Rosario, Sastre, Leandro, Prosper, Felipe, Gomez-Cabrero, David, Bueren, Juan A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Fondazione Ferrata Storti 2023
Materias:
Article - Bone Marrow Failure
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10542844/
https://www.ncbi.nlm.nih.gov/pubmed/37021532
http://dx.doi.org/10.3324/haematol.2022.282418

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10542844/
https://www.ncbi.nlm.nih.gov/pubmed/37021532
http://dx.doi.org/10.3324/haematol.2022.282418

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