Highly efficient therapeutic gene editing of human hematopoietic stem cells

Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by induction of fetal hemoglobin (HbF, α(2)γ(2))(1). Previously we and others have shown that core sequences at the BCL11A ery...

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Detalles Bibliográficos
Autores principales: Wu, Yuxuan, Zeng, Jing, Roscoe, Benjamin P., Liu, Pengpeng, Yao, Qiuming, Lazzarotto, Cicera R., Clement, M. Kendell, Cole, Mitchel A., Luk, Kevin, Baricordi, Cristina, Shen, Anne H., Ren, Chunyan, Esrick, Erica B., Manis, John P., Dorfman, David M., Williams, David A., Biffi, Alessandra, Brugnara, Carlo, Biasco, Luca, Brendel, Christian, Pinello, Luca, Tsai, Shengdar Q., Wolfe, Scot A., Bauer, Daniel E.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6512986/
https://www.ncbi.nlm.nih.gov/pubmed/30911135
http://dx.doi.org/10.1038/s41591-019-0401-y

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6512986/
https://www.ncbi.nlm.nih.gov/pubmed/30911135
http://dx.doi.org/10.1038/s41591-019-0401-y

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