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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Publicado no:Nat Med
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: 2019
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6512986/
https://ncbi.nlm.nih.gov/pubmed/30911135
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41591-019-0401-y
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