Highly efficient editing of the β-globin gene in patient-derived hematopoietic stem and progenitor cells to treat sickle cell disease

Sickle cell disease (SCD) is a monogenic disorder that affects millions worldwide. Allogeneic hematopoietic stem cell transplantation is the only available cure. Here, we demonstrate the use of CRISPR/Cas9 and a short single-stranded oligonucleotide template to correct the sickle mutation in the β-g...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Park, So Hyun, Lee, Ciaran M, Dever, Daniel P, Davis, Timothy H, Camarena, Joab, Srifa, Waracharee, Zhang, Yankai, Paikari, Alireza, Chang, Alicia K, Porteus, Matthew H, Sheehan, Vivien A, Bao, Gang
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
Assuntos:
Genome Integrity, Repair and Replication
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6735704/
https://ncbi.nlm.nih.gov/pubmed/31147717
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkz475
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