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...

Ful tanımlama

Kaydedildi:
Detaylı Bibliyografya
Yayımlandı:Nat Med
Asıl Yazarlar: 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.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2021
Konular:
Article
Online Erişim: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
Etiketler: Etiketle
Etiket eklenmemiş, İlk siz ekleyin!

Benzer Materyaller