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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Veröffentlicht in:	Genome research 2014-09, Vol.24 (9), p.1526-1533
Hauptverfasser:	Xie, Fei, Ye, Lin, Chang, Judy C, Beyer, Ashley I, Wang, Jiaming, Muench, Marcus O, Kan, Yuet Wai
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals beta-Globins - genetics beta-Thalassemia - genetics CRISPR-Cas Systems DNA Transposable Elements - genetics Erythroblasts - cytology Erythroblasts - metabolism Erythropoiesis HEK293 Cells Humans Induced Pluripotent Stem Cells - cytology Induced Pluripotent Stem Cells - metabolism Method Mice Mutation Targeted Gene Repair - methods
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