High-level correction of the sickle mutation is amplified in vivo during erythroid differentiation

BACKGROUND: A point mutation in sickle cell disease (SCD) alters one amino acid in the β-globin subunit of hemoglobin, with resultant anemia and multiorgan damage that typically shortens lifespan by decades. Because SCD is caused by a single mutation, and hematopoietic stem cells (HSCs) can be harve...

Descripción completa

Detalles Bibliográficos
Autores principales: Magis, Wendy, DeWitt, Mark A., Wyman, Stacia K., Vu, Jonathan T., Heo, Seok-Jin, Shao, Shirley J., Hennig, Finn, Romero, Zulema G., Campo-Fernandez, Beatriz, Said, Suzanne, McNeill, Matthew S., Rettig, Garrett R., Sun, Yongming, Wang, Yu, Behlke, Mark A., Kohn, Donald B., Boffelli, Dario, Walters, Mark C., Corn, Jacob E., Martin, David I.K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9130532/
https://www.ncbi.nlm.nih.gov/pubmed/35633935
http://dx.doi.org/10.1016/j.isci.2022.104374

Ejemplares similares

Ejemplares similares