Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells

Autor: Chu, Simon N. ^{1, 2, 3}, Soupene, Eric ⁴, Sharma, Devesh ^{1, 2}, Sinha, Roshani ^{1, 2}, McCreary, Travis ^{1, 2}, Hernandez, Britney ⁵, Shen, Huifeng ⁵, Wienert, Beeke ⁶, Bowman, Chance ^{1, 2, 7}, Yin, Han ^{1, 3}, Lesch, Benjamin J. ^{1, 2, 7}, Jia, Kun ^{1, 2}, Romero, Kathleen A. ⁵, Kostamo, Zachary ⁵, Zhang, Yankai ⁵, Tran, Tammy ^{1, 2}, Cordero, Marco ^{1, 2}, Homma, Shota ⁸, Hampton, Jessica P. ⁹, Gardner, James M. ^{1, 3}, Conklin, Bruce R. ^{6, 10}, MacKenzie, Tippi C. ^{1, 2}, Sheehan, Vivien A. ⁵, Porteus, Matthew H. ⁹, Cromer, M. Kyle ^{1, 2, 7, 11, ∗}

Publikováno v: In Cell Reports 28 January 2025 44(1)

Dual α-globin and truncated EPO receptor knockin restores hemoglobin production in α-thalassemia-derived red blood cells.

Autor: Chu SN; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA.; Diabetes Center, University of California, San Francisco, San Francisco, CA., Soupene E; Department of Pediatrics, University of California, San Francisco, Oakland, CA., Wienert B; Gladstone Institutes, San Francisco, CA., Yin H; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Diabetes Center, University of California, San Francisco, San Francisco, CA., Sharma D; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA., McCreary T; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA., Jia K; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA., Homma S; Department of Genetics, Stanford University, Stanford, CA., Hampton JP; Department of Pediatrics, Stanford University, Stanford, CA., Gardner JM; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Diabetes Center, University of California, San Francisco, San Francisco, CA., Conklin BR; Gladstone Institutes, San Francisco, CA.; Department of Medicine, University of California, San Francisco, San Francisco, CA., MacKenzie TC; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA., Porteus MH; Department of Pediatrics, Stanford University, Stanford, CA., Cromer MK; Department of Surgery, University of California, San Francisco, San Francisco, CA.; Eli & Edythe Broad Center for Regeneration Medicine, University of California, San Francisco, San Francisco, CA.; Department of Bioengineering & Therapeutic Sciences, University of California, San Francisco,San Francisco, CA.

Publikováno v: BioRxiv : the preprint server for biology [bioRxiv] 2024 May 07. Date of Electronic Publication: 2024 May 07.