Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling

Abstract We previously reported initial clinical results of post-transcriptional gene silencing of BCL11A expression (NCT 03282656) reversing the fetal to adult hemoglobin switch. A goal of this approach is to increase fetal hemoglobin (HbF) expression while coordinately reducing sickle hemoglobin (...

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Κύριοι συγγραφείς:	Daniel C. De Souza, Nicolas Hebert, Erica B. Esrick, M. Felicia Ciuculescu, Natasha M. Archer, Myriam Armant, Étienne Audureau, Christian Brendel, Giuseppe Di Caprio, Frédéric Galactéros, Donghui Liu, Amanda McCabe, Emily Morris, Ethan Schonbrun, Dillon Williams, David K. Wood, David A. Williams, Pablo Bartolucci, John M. Higgins
Μορφή:	Artigo
Γλώσσα:	Inglês
Έκδοση:	Nature Portfolio 2023-09-01
Σειρά:	Nature Communications
Διαθέσιμο Online:	https://doi.org/10.1038/s41467-023-40923-5
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