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Page 1
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: locatelli f. 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.
Challenges and opportunities for international cooperative studies in pediatric hematopoeitic cell transplantation: priorities of the Westhafen Intercontinental Group.
Schultz RK, Baker KS, Boelens JJ, Bollard CM, Egeler RM, Cowan M, Ladenstein R, Lankester A, Locatelli F, Lawitschka A, Levine JE, Loh M, Nemecek E, Niemeyer C, Prasad VK, Rocha V, Shenoy S, Strahm B, Veys P, Wall D, Bader P, Grupp SA, Pulsipher MA, Peters C. Schultz RK, et al. Among authors: locatelli f. Biol Blood Marrow Transplant. 2013 Sep;19(9):1279-87. doi: 10.1016/j.bbmt.2013.07.006. Epub 2013 Jul 21. Biol Blood Marrow Transplant. 2013. PMID: 23883618 Free PMC article.
Hematopoietic stem cell transplantation in thalassemia major and sickle cell disease: indications and management recommendations from an international expert panel.
Angelucci E, Matthes-Martin S, Baronciani D, Bernaudin F, Bonanomi S, Cappellini MD, Dalle JH, Di Bartolomeo P, de Heredia CD, Dickerhoff R, Giardini C, Gluckman E, Hussein AA, Kamani N, Minkov M, Locatelli F, Rocha V, Sedlacek P, Smiers F, Thuret I, Yaniv I, Cavazzana M, Peters C; EBMT Inborn Error and EBMT Paediatric Working Parties. Angelucci E, et al. Among authors: locatelli f. Haematologica. 2014 May;99(5):811-20. doi: 10.3324/haematol.2013.099747. Haematologica. 2014. PMID: 24790059 Free PMC article.
Will post-transplantation cell therapies for pediatric patients become standard of care?
Lankester AC, Locatelli F, Bader P, Rettinger E, Egeler M, Katewa S, Pulsipher MA, Nierkens S, Schultz K, Handgretinger R, Grupp SA, Boelens JJ, Bollard CM; Westhafen Intercontinental Group. Lankester AC, et al. Among authors: locatelli f. Biol Blood Marrow Transplant. 2015 Mar;21(3):402-11. doi: 10.1016/j.bbmt.2014.07.018. Epub 2014 Jul 23. Biol Blood Marrow Transplant. 2015. PMID: 25064748 Free article. Review.
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: locatelli f. 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: locatelli f. 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.
Ruxolitinib for Chronic Graft-versus-Host Disease. Reply.
Zeiser R, Teshima T, Locatelli F. Zeiser R, et al. Among authors: locatelli f. N Engl J Med. 2021 Oct 21;385(17):1631-1632. doi: 10.1056/NEJMc2113499. N Engl J Med. 2021. PMID: 34670053 No abstract available.
Betibeglogene Autotemcel Gene Therapy for Non-β0/β0 Genotype β-Thalassemia.
Locatelli F, Thompson AA, Kwiatkowski JL, Porter JB, Thrasher AJ, Hongeng S, Sauer MG, Thuret I, Lal A, Algeri M, Schneiderman J, Olson TS, Carpenter B, Amrolia PJ, Anurathapan U, Schambach A, Chabannon C, Schmidt M, Labik I, Elliot H, Guo R, Asmal M, Colvin RA, Walters MC. Locatelli F, et al. N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11. N Engl J Med. 2022. PMID: 34891223 Clinical Trial.
2,178 results