Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and re...
Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling
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Author / Creator
De Souza, Daniel C. , Hebert, Nicolas , Esrick, Erica B. , Ciuculescu, M. Felicia , Archer, Natasha M. , Armant, Myriam , Audureau, Étienne , Brendel, Christian , Di Caprio, Giuseppe , Galactéros, Frédéric , Liu, Donghui , McCabe, Amanda , Morris, Emily , Schonbrun, Ethan , Williams, Dillon , Wood, David K. , Williams, David A. , Bartolucci, Pablo and Higgins, John M.
Publisher
London: Nature Publishing Group UK
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Language
English
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Publisher
London: Nature Publishing Group UK
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Contents
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 (HbS) expression. The resulting combinatorial effect should...
Alternative Titles
Full title
Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling
Authors, Artists and Contributors
Author / Creator
Hebert, Nicolas
Esrick, Erica B.
Ciuculescu, M. Felicia
Archer, Natasha M.
Armant, Myriam
Audureau, Étienne
Brendel, Christian
Di Caprio, Giuseppe
Galactéros, Frédéric
Liu, Donghui
McCabe, Amanda
Morris, Emily
Schonbrun, Ethan
Williams, Dillon
Wood, David K.
Williams, David A.
Bartolucci, Pablo
Higgins, John M.
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Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_c27e3b0ea4ac4641bfd4b6b6b60347e0
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_c27e3b0ea4ac4641bfd4b6b6b60347e0
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ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-023-40923-5