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Gene Therapy and Gene Editing for β-Thalassemia.
Christakopoulos GE, Telange R, Yen J, Weiss MJ. Christakopoulos GE, et al. Among authors: weiss mj. Hematol Oncol Clin North Am. 2023 Apr;37(2):433-447. doi: 10.1016/j.hoc.2022.12.012. Hematol Oncol Clin North Am. 2023. PMID: 36907613 Free PMC article. Review.
Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1.
Mamcarz E, Zhou S, Lockey T, Abdelsamed H, Cross SJ, Kang G, Ma Z, Condori J, Dowdy J, Triplett B, Li C, Maron G, Aldave Becerra JC, Church JA, Dokmeci E, Love JT, da Matta Ain AC, van der Watt H, Tang X, Janssen W, Ryu BY, De Ravin SS, Weiss MJ, Youngblood B, Long-Boyle JR, Gottschalk S, Meagher MM, Malech HL, Puck JM, Cowan MJ, Sorrentino BP. Mamcarz E, et al. Among authors: weiss mj. N Engl J Med. 2019 Apr 18;380(16):1525-1534. doi: 10.1056/NEJMoa1815408. N Engl J Med. 2019. PMID: 30995372 Free PMC article. Clinical Trial.
Safe and efficient peripheral blood stem cell collection in patients with sickle cell disease using plerixafor.
Uchida N, Leonard A, Stroncek D, Panch SR, West K, Molloy E, Hughes TE, Hauffe S, Taylor T, Fitzhugh C, Hankins JS, Wilson M, Sharma A, Tsai SQ, Weiss MJ, Hsieh M, Tisdale JF. Uchida N, et al. Among authors: weiss mj. Haematologica. 2020 Oct 1;105(10):e497. doi: 10.3324/haematol.2019.236182. Haematologica. 2020. PMID: 33054091 Free PMC article. No abstract available.
Pharmacogenetics for Safe Codeine Use in Sickle Cell Disease.
Gammal RS, Crews KR, Haidar CE, Hoffman JM, Baker DK, Barker PJ, Estepp JH, Pei D, Broeckel U, Wang W, Weiss MJ, Relling MV, Hankins J. Gammal RS, et al. Among authors: weiss mj. Pediatrics. 2016 Jul;138(1):e20153479. doi: 10.1542/peds.2015-3479. Pediatrics. 2016. PMID: 27335380 Free PMC article.
679 results