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Esrick, Erica B; Lehmann, Leslie E; Biffi, Alessandra; Achebe, Maureen; Brendel, Christian; Ciuculescu, Marioara F; Daley, Heather; MacKinnon, Brenda; Morris, Emily; Federico, Amy; Abriss, Daniela; Boardman, Kari; Khelladi, Radia; Shaw, Kit; Negre, Helene; Negre, Olivier; Nikiforow, Sarah; Ritz, Jerome; Pai, Sung-Yun; London, Wendy B; Dansereau, Colleen; Heeney, Matthew M; Armant, Myriam; Manis, John P; Williams, David A
The New England journal of medicine, 01/2021, Volume: 384, Issue: 3Journal Article
The switch from fetal to adult hemoglobin expression in humans is related to the active repression of HbF expression by BCL11A . A self-inactivating lentivirus vector with a short hairpin RNA designed to interfere with BCL11A expression was inserted into autologous hematopoietic stem cells of patients with sickle cell disease, resulting in increased expression of HbF and a decline in the frequency of sickle cell crises.
