Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia-Reference-Cited by-同舟云学术

Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

Published:2019-09 Issue:9 Volume:25 Page:1396-1401
ISSN:1078-8956
Container-title:Nature Medicine
language:en
Short-container-title:Nat Med

Author:

Río Paula,Navarro Susana,Wang Wei,Sánchez-Domínguez Rebeca,Pujol Roser M.,Segovia José C.,Bogliolo Massimo,Merino Eva,Wu Ning,Salgado Rocío,Lamana María L.,Yañez Rosa M.,Casado José A.,Giménez Yari,Román-Rodríguez Francisco J.,Álvarez Lara,Alberquilla Omaira,Raimbault Anna,Guenechea Guillermo,Lozano M. Luz,Cerrato Laura,Hernando Miriam,Gálvez Eva,Hladun Raquel,Giralt Irina,Barquinero Jordi,Galy Anne,García de Andoín Nagore,López Ricardo,Catalá Albert,Schwartz Jonathan D.,Surrallés Jordi,Soulier Jean,Schmidt Manfred,Díaz de Heredia Cristina,Sevilla Julián^ORCID,Bueren Juan A.^ORCID

Publisher

Springer Science and Business Media LLC

Subject

General Biochemistry, Genetics and Molecular Biology,General Medicine

Link

http://www.nature.com/articles/s41591-019-0550-z.pdf

Reference40 articles.

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2. Knies, K. et al. Biallelic mutations in the ubiquitin ligase RFWD3 cause Fanconi anemia. J. Clin. Invest. 127, 3013–3027 (2017).

3. Casado, J. A. et al. A comprehensive strategy for the subtyping of Fanconi anemia patients: conclusions from the Spanish Fanconi Anemia research network. J. Med. Genet. 44, 241–249 (2007).

4. Taniguchi, T. & D’Andrea, A. D. Molecular pathogenesis of Fanconi anemia: recent progress. Blood 107, 4223–4233 (2006).

5. Butturini, A. et al. Hematologic abnormalities in Fanconi anemia: an International Fanconi Anemia Registry study. Blood 84, 1650–1655 (1994).

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