Below are the most recent publications written about "beta-Thalassemia" by people in Profiles.
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Flotte TR. Gene Therapy in Global Health: Meeting the Needs of Chinese Patients with Beta-Thalassemia. Hum Gene Ther. 2021 May; 32(9-10):449-450.
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Zeng J, Wu Y, Ren C, Bonanno J, Shen AH, Shea D, Gehrke JM, Clement K, Luk K, Yao Q, Kim R, Wolfe SA, Manis JP, Pinello L, Joung JK, Bauer DE. Therapeutic base editing of human hematopoietic stem cells. Nat Med. 2020 04; 26(4):535-541.
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Wu Y, Zeng J, Roscoe BP, Liu P, Yao Q, Lazzarotto CR, Clement K, Cole MA, Luk K, Baricordi C, Shen AH, Ren C, Esrick EB, Manis JP, Dorfman DM, Williams DA, Biffi A, Brugnara C, Biasco L, Brendel C, Pinello L, Tsai SQ, Wolfe SA, Bauer DE. Highly efficient therapeutic gene editing of human hematopoietic stem cells. Nat Med. 2019 May; 25(5):776-783.
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Xu S, Luk K, Yao Q, Shen AH, Zeng J, Wu Y, Luo HY, Brendel C, Pinello L, Chui DHK, Wolfe SA, Bauer DE. Editing aberrant splice sites efficiently restores ?-globin expression in ?-thalassemia. Blood. 2019 05 23; 133(21):2255-2262.
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Taher AT, Saliba AN, Kuo KH, Giardina PJ, Cohen AR, Neufeld EJ, Aydinok Y, Kwiatkowski JL, Jeglinski BI, Pietropaolo K, Berk G, Viprakasit V. Safety and pharmacokinetics of the oral iron chelator SP-420 in ?-thalassemia. Am J Hematol. 2017 Dec; 92(12):1356-1361.
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Gaines P, Halene S. Rusty neutrophils in ?-thalassemia: no traction! Blood. 2017 06 08; 129(23):3046-3047.
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Wilburn CR, Bernard DW, Zieske AW, Andrieni J, Miller T, Wang P. The Prevalence and Role of Hemoglobin Variants in Biometric Screening of a Multiethnic Population: One Large Health System's Experience. Am J Clin Pathol. 2017 Jun 01; 147(6):589-595.
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Bahal R, Ali McNeer N, Quijano E, Liu Y, Sulkowski P, Turchick A, Lu YC, Bhunia DC, Manna A, Greiner DL, Brehm MA, Cheng CJ, L?pez-Gir?ldez F, Ricciardi A, Beloor J, Krause DS, Kumar P, Gallagher PG, Braddock DT, Mark Saltzman W, Ly DH, Glazer PM. In vivo correction of anaemia in ?-thalassemic mice by ?PNA-mediated gene editing with nanoparticle delivery. Nat Commun. 2016 10 26; 7:13304.
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Spitzer B, Giardina PJ, O'Reilly RJ, Boulad F. Late complications of mixed chimerism following allogeneic bone marrow transplantation for thalassemia major. Pediatr Blood Cancer. 2015 Jul; 62(7):1303-4.
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Boulad F, Wang X, Qu J, Taylor C, Ferro L, Karponi G, Bartido S, Giardina P, Heller G, Prockop SE, Maggio A, Sadelain M, Rivi?re I. Safe mobilization of CD34+ cells in adults with ?-thalassemia and validation of effective globin gene transfer for clinical investigation. Blood. 2014 Mar 06; 123(10):1483-6.