Gene replacement of α-globin with β-globin restores hemoglobin balance in β-thalassemia-derived hematopoietic stem and progenitor cells

β-Thalassemia pathology is due not only to loss of β-globin (HBB), but also to erythrotoxic accumulation and aggregation of the β-globin-binding partner, α-globin (HBA1/2). Here we describe a Cas9/AAV6-mediated genome editing strategy that can replace the entire HBA1 gene with a full-length HBB tran...

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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Nat Med
Prif Awduron: Cromer, M. Kyle, Camarena, Joab, Martin, Renata M., Lesch, Benjamin J., Vakulskas, Christopher A., Bode, Nicole M., Kurgan, Gavin, Collingwood, Michael A., Rettig, Garrett R., Behlke, Mark A., Lemgart, Viktor T., Zhang, Yankai, Goyal, Ankush, Zhao, Feifei, Ponce, Ezequiel, Srifa, Waracharee, Bak, Rasmus O., Uchida, Naoya, Majeti, Ravindra, Sheehan, Vivien A., Tisdale, John F., Dever, Daniel P., Porteus, Matthew H.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: 2021
Pynciau:
Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC8265212/
https://ncbi.nlm.nih.gov/pubmed/33737751
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41591-021-01284-y
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