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Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia.
Thompson AA, Walters MC, Kwiatkowski J, Rasko JEJ, Ribeil JA, Hongeng S, Magrin E, Schiller GJ, Payen E, Semeraro M, Moshous D, Lefrere F, Puy H, Bourget P, Magnani A, Caccavelli L, Diana JS, Suarez F, Monpoux F, Brousse V, Poirot C, Brouzes C, Meritet JF, Pondarré C, Beuzard Y, Chrétien S, Lefebvre T, Teachey DT, Anurathapan U, Ho PJ, von Kalle C, Kletzel M, Vichinsky E, Soni S, Veres G, Negre O, Ross RW, Davidson D, Petrusich A, Sandler L, Asmal M, Hermine O, De Montalembert M, Hacein-Bey-Abina S, Blanche S, Leboulch P, Cavazzana M. Thompson AA, et al. Among authors: soni s. N Engl J Med. 2018 Apr 19;378(16):1479-1493. doi: 10.1056/NEJMoa1705342. N Engl J Med. 2018. PMID: 29669226 Free article. Clinical Trial.
Gene Therapy in a Patient with Sickle Cell Disease.
Ribeil JA, Hacein-Bey-Abina S, Payen E, Magnani A, Semeraro M, Magrin E, Caccavelli L, Neven B, Bourget P, El Nemer W, Bartolucci P, Weber L, Puy H, Meritet JF, Grevent D, Beuzard Y, Chrétien S, Lefebvre T, Ross RW, Negre O, Veres G, Sandler L, Soni S, de Montalembert M, Blanche S, Leboulch P, Cavazzana M. Ribeil JA, et al. Among authors: soni s. N Engl J Med. 2017 Mar 2;376(9):848-855. doi: 10.1056/NEJMoa1609677. N Engl J Med. 2017. PMID: 28249145 Free article.
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: soni s. 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.
Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.
Shenoy S, Walters MC, Ngwube A, Soni S, Jacobsohn D, Chaudhury S, Grimley M, Chan K, Haight A, Kasow KA, Parikh S, Andreansky M, Connelly J, Delgado D, Godder K, Hale G, Nieder M, Pulsipher MA, Trachtenberg F, Neufeld E, Kwiatkowski JL, Thompson AA. Shenoy S, et al. Among authors: soni s. Biol Blood Marrow Transplant. 2018 Jun;24(6):1216-1222. doi: 10.1016/j.bbmt.2018.01.023. Epub 2018 Jan 31. Biol Blood Marrow Transplant. 2018. PMID: 29374585 Free PMC article. Clinical Trial.
Veno-occlusive disease after high-dose busulfan-melphalan in neuroblastoma.
Schechter T, Perez-Albuerne E, Lin TF, Irwin MS, Essa M, Desai AV, Frangoul H, Yanik G, Dupuis LL, Jacobsohn D, Kletzel M, Ranalli M, Soni S, Seif AE, Grupp S, Dvorak CC. Schechter T, et al. Among authors: soni s. Bone Marrow Transplant. 2020 Mar;55(3):531-537. doi: 10.1038/s41409-018-0298-y. Epub 2018 Sep 4. Bone Marrow Transplant. 2020. PMID: 30181580
1,047 results