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

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) expr...

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Detalles Bibliográficos
Autores principales: 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, Higgins, John M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10511721/
https://www.ncbi.nlm.nih.gov/pubmed/37730674
http://dx.doi.org/10.1038/s41467-023-40923-5

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10511721/
https://www.ncbi.nlm.nih.gov/pubmed/37730674
http://dx.doi.org/10.1038/s41467-023-40923-5

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