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Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.
Kanter J, Thompson AA, Pierciey FJ Jr, Hsieh M, Uchida N, Leboulch P, Schmidt M, Bonner M, Guo R, Miller A, Ribeil JA, Davidson D, Asmal M, Walters MC, Tisdale JF. Kanter J, et al. Among authors: schmidt m. Am J Hematol. 2023 Jan;98(1):11-22. doi: 10.1002/ajh.26741. Epub 2022 Oct 10. Am J Hematol. 2023. PMID: 36161320 Free PMC article.
Polyclonal long-term repopulating stem cell clones in a primate model.
Schmidt M, Zickler P, Hoffmann G, Haas S, Wissler M, Muessig A, Tisdale JF, Kuramoto K, Andrews RG, Wu T, Kiem HP, Dunbar CE, von Kalle C. Schmidt M, et al. Blood. 2002 Oct 15;100(8):2737-43. doi: 10.1182/blood-2002-02-0407. Blood. 2002. PMID: 12351380 Free article.
Parallel assessment of globin lentiviral transfer in induced pluripotent stem cells and adult hematopoietic stem cells derived from the same transplanted β-thalassemia patient.
Tubsuwan A, Abed S, Deichmann A, Kardel MD, Bartholomä C, Cheung A, Negre O, Kadri Z, Fucharoen S, von Kalle C, Payen E, Chrétien S, Schmidt M, Eaves CJ, Leboulch P, Maouche-Chrétien L. Tubsuwan A, et al. Among authors: schmidt m. Stem Cells. 2013 Sep;31(9):1785-94. doi: 10.1002/stem.1436. Stem Cells. 2013. PMID: 23712774
Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease.
Negre O, Bartholomae C, Beuzard Y, Cavazzana M, Christiansen L, Courne C, Deichmann A, Denaro M, de Dreuzy E, Finer M, Fronza R, Gillet-Legrand B, Joubert C, Kutner R, Leboulch P, Maouche L, Paulard A, Pierciey FJ, Rothe M, Ryu B, Schmidt M, von Kalle C, Payen E, Veres G. Negre O, et al. Among authors: schmidt m. Curr Gene Ther. 2015;15(1):64-81. doi: 10.2174/1566523214666141127095336. Curr Gene Ther. 2015. PMID: 25429463 Free PMC article.
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. Among authors: schmidt m. 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.
Acute Myeloid Leukemia Case after Gene Therapy for Sickle Cell Disease.
Goyal S, Tisdale J, Schmidt M, Kanter J, Jaroscak J, Whitney D, Bitter H, Gregory PD, Parsons G, Foos M, Yeri A, Gioia M, Voytek SB, Miller A, Lynch J, Colvin RA, Bonner M. Goyal S, et al. Among authors: schmidt m. N Engl J Med. 2022 Jan 13;386(2):138-147. doi: 10.1056/NEJMoa2109167. Epub 2021 Dec 12. N Engl J Med. 2022. PMID: 34898140
Long-term outcomes of lentiviral gene therapy for the β-hemoglobinopathies: the HGB-205 trial.
Magrin E, Semeraro M, Hebert N, Joseph L, Magnani A, Chalumeau A, Gabrion A, Roudaut C, Marouene J, Lefrere F, Diana JS, Denis A, Neven B, Funck-Brentano I, Negre O, Renolleau S, Brousse V, Kiger L, Touzot F, Poirot C, Bourget P, El Nemer W, Blanche S, Tréluyer JM, Asmal M, Walls C, Beuzard Y, Schmidt M, Hacein-Bey-Abina S, Asnafi V, Guichard I, Poirée M, Monpoux F, Touraine P, Brouzes C, de Montalembert M, Payen E, Six E, Ribeil JA, Miccio A, Bartolucci P, Leboulch P, Cavazzana M. Magrin E, et al. Among authors: schmidt m. Nat Med. 2022 Jan;28(1):81-88. doi: 10.1038/s41591-021-01650-w. Epub 2022 Jan 24. Nat Med. 2022. PMID: 35075288 Clinical Trial.
Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia.
Kiem HP, Sellers S, Thomasson B, Morris JC, Tisdale JF, Horn PA, Hematti P, Adler R, Kuramoto K, Calmels B, Bonifacino A, Hu J, von Kalle C, Schmidt M, Sorrentino B, Nienhuis A, Blau CA, Andrews RG, Donahue RE, Dunbar CE. Kiem HP, et al. Among authors: schmidt m. Mol Ther. 2004 Mar;9(3):389-95. doi: 10.1016/j.ymthe.2003.12.006. Mol Ther. 2004. PMID: 15006605 Free article.
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