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

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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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Nature Portfolio 2023-09-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-023-40923-5

Internet

https://doi.org/10.1038/s41467-023-40923-5

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