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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.
Frangoul H, Altshuler D, Cappellini MD, Chen YS, Domm J, Eustace BK, Foell J, de la Fuente J, Grupp S, Handgretinger R, Ho TW, Kattamis A, Kernytsky A, Lekstrom-Himes J, Li AM, Locatelli F, Mapara MY, de Montalembert M, Rondelli D, Sharma A, Sheth S, Soni S, Steinberg MH, Wall D, Yen A, Corbacioglu S. Frangoul H, et al. Among authors: de montalembert m, de la fuente j. N Engl J Med. 2021 Jan 21;384(3):252-260. doi: 10.1056/NEJMoa2031054. Epub 2020 Dec 5. N Engl J Med. 2021. PMID: 33283989 Clinical Trial.
Sickle cell disease: the price of cure.
Roberts I, de la Fuente J. Roberts I, et al. Among authors: de la fuente j. Blood. 2016 Nov 24;128(21):2486-2488. doi: 10.1182/blood-2016-10-740969. Blood. 2016. PMID: 27884835 Free article. No abstract available.
Sickle cell disease: an international survey of results of HLA-identical sibling hematopoietic stem cell transplantation.
Gluckman E, Cappelli B, Bernaudin F, Labopin M, Volt F, Carreras J, Pinto Simões B, Ferster A, Dupont S, de la Fuente J, Dalle JH, Zecca M, Walters MC, Krishnamurti L, Bhatia M, Leung K, Yanik G, Kurtzberg J, Dhedin N, Kuentz M, Michel G, Apperley J, Lutz P, Neven B, Bertrand Y, Vannier JP, Ayas M, Cavazzana M, Matthes-Martin S, Rocha V, Elayoubi H, Kenzey C, Bader P, Locatelli F, Ruggeri A, Eapen M; Eurocord, the Pediatric Working Party of the European Society for Blood and Marrow Transplantation, and the Center for International Blood and Marrow Transplant Research. Gluckman E, et al. Among authors: de la fuente j. Blood. 2017 Mar 16;129(11):1548-1556. doi: 10.1182/blood-2016-10-745711. Epub 2016 Dec 13. Blood. 2017. PMID: 27965196 Free PMC article.
Risk factors and outcomes according to age at transplantation with an HLA-identical sibling for sickle cell disease.
Cappelli B, Volt F, Tozatto-Maio K, Scigliuolo GM, Ferster A, Dupont S, Simões BP, Al-Seraihy A, Aljurf MD, Almohareb F, Belendez C, Matthes S, Dhedin N, Pondarre C, Dalle JH, Bertrand Y, Vannier JP, Kuentz M, Lutz P, Michel G, Rafii H, Neven B, Zecca M, Bader P, Cavazzana M, Labopin M, Locatelli F, Magnani A, Ruggeri A, Rocha V, Bernaudin F, de La Fuente J, Corbacioglu S, Gluckman E; Eurocord, the Cellular Therapy and Immunobiology Working Party (CTIWP) and the Paediatric Diseases Working Party (PDWP) of the EBMT. Cappelli B, et al. Among authors: de la fuente j. Haematologica. 2019 Dec;104(12):e543-e546. doi: 10.3324/haematol.2019.216788. Epub 2019 Apr 24. Haematologica. 2019. PMID: 31018975 Free PMC article. No abstract available.
Proceedings of the meeting of the Pediatric Disease Working Party (PDWP) of the European group for Blood and Marrow Transplantation (EBMT) on sickle cell disease 16-17 May 2019, Regensburg, Germany.
Corbacioglu S, Gluckman E, Alahmari A, Kassim A, de la Fuente J. Corbacioglu S, et al. Among authors: de la fuente j. Hematol Oncol Stem Cell Ther. 2020 Jun;13(2):51-52. doi: 10.1016/j.hemonc.2019.12.001. Epub 2020 Mar 12. Hematol Oncol Stem Cell Ther. 2020. PMID: 32202247 Free article. No abstract available.
The role of haematopoietic stem cell transplantation for sickle cell disease in the era of targeted disease-modifying therapies and gene editing.
de la Fuente J, Gluckman E, Makani J, Telfer P, Faulkner L, Corbacioglu S; Paediatric Diseases Working Party of the European Society for Blood and Marrow Transplantation. de la Fuente J, et al. Lancet Haematol. 2020 Dec;7(12):e902-e911. doi: 10.1016/S2352-3026(20)30283-0. Lancet Haematol. 2020. PMID: 33242447 Review.
Systematic Review/Meta-Analysis on Efficacy of Allogeneic Hematopoietic Cell Transplantation in Sickle Cell Disease: An International Effort on Behalf of the Pediatric Diseases Working Party of European Society for Blood and Marrow Transplantation and the Sickle Cell Transplantation International Consortium.
Iqbal M, Reljic T, Corbacioglu S, de la Fuente J, Gluckman E, Kumar A, Yassine F, Ayala E, El-Jawahri A, Murthy H, Almohareb F, Hashmi SK, Cappelli B, Alahmari A, Scigliuolo GM, Kassim A, Aljurf M, Kharfan-Dabaja MA. Iqbal M, et al. Among authors: de la fuente j. Transplant Cell Ther. 2021 Feb;27(2):167.e1-167.e12. doi: 10.1016/j.jtct.2020.10.007. Epub 2020 Dec 10. Transplant Cell Ther. 2021. PMID: 33830027 Free article.
Busulfan-fludarabine- or treosulfan-fludarabine-based myeloablative conditioning for children with thalassemia major.
Lüftinger R, Zubarovskaya N, Galimard JE, Cseh A, Salzer E, Locatelli F, Algeri M, Yesilipek A, de la Fuente J, Isgrò A, Alseraihy A, Angelucci E, Smiers FJ, La La Nasa G, Zecca M, Fisgin T, Unal E, Kleinschmidt K, Peters C, Lankester A, Corbacioglu S; EBMT Pediatric Diseases, Inborn Errors Working Parties. Lüftinger R, et al. Among authors: la la nasa g, de la fuente j. Ann Hematol. 2022 Mar;101(3):655-665. doi: 10.1007/s00277-021-04732-4. Epub 2022 Jan 9. Ann Hematol. 2022. PMID: 34999929
Busulfan-fludarabine- or treosulfan-fludarabine-based conditioning before allogeneic HSCT from matched sibling donors in paediatric patients with sickle cell disease: A study on behalf of the EBMT Paediatric Diseases and Inborn Errors Working Parties.
Cseh A, Galimard JE, de la Fuente J, Isgro A, Zecca M, Garwer B, Biffi A, Aljurf M, Sundin M, Belendez C, Locatelli F, Balduzzi A, Lawson S, Sengeloev H, Ifversen M, Saccardi R, Wynn R, Lankester AC, Corbacioglu S, Peters C. Cseh A, et al. Among authors: de la fuente j. Br J Haematol. 2024 Jan;204(1):e1-e5. doi: 10.1111/bjh.19122. Epub 2023 Oct 5. Br J Haematol. 2024. PMID: 37795523
1,427 results