Article
Transcription activator-like effector nuclease (TALEN)-mediated gene correction in integration-free β-thalassemia induced pluripotent stem cells.
The Journal of biological chemistry - 29 Nov 2013
Ma Ning, Liao Baojian, Zhang Hui, Wang Linli, Shan Yongli, Xue Yanting, Huang Ke, Chen Shubin, Zhou Xiaoxiao, Chen Yang, Pei Duanqing, Pan Guangjin
Abstract excerpt
β-Thalassemia (β-Thal) is a group of life-threatening blood disorders caused by either point mutations or deletions of nucleotides in β-globin gene (HBB). It is estimated that 4.5% of the population in the world carry β-Thal mutants (1), posing a persistent threat to public health. The generation of patient-specific induced pluripotent stem cells (iPSCs) and subsequent correction of the disease-causing mutations...
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