Seamless gene correction of β-thalassemia mutations in patient-specific iPSCs using CRISPR/Cas9 and piggyBac

β-thalassemia, one of the most common genetic diseases worldwide, is caused by mutations in the human hemoglobin beta (HBB) gene. Creation of human induced pluripotent stem cells (iPSCs) from β-thalassemia patients could offer an approach to cure this disease. Correction of the disease-causing mutat...

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Main Authors: Xie, Fei, Ye, Lin, Chang, Judy C., Beyer, Ashley I., Wang, Jiaming, Muench, Marcus O., Kan, Yuet Wai
Formato: Artigo
Idioma:Inglês
Publicado em: Cold Spring Harbor Laboratory Press 2014
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Method
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4158758/
https://ncbi.nlm.nih.gov/pubmed/25096406
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1101/gr.173427.114
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