The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice

@inproceedings{Ou2016TheCO,
  title={The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice},
  author={Zhanhui Ou and Xiaohua Niu and Wenyin He and Yuchang Chen and Bing Song and Yexing Xian and Di Fan and Daolin Tang and Xiaofang Sun},
  booktitle={Scientific reports},
  year={2016}
}
β-thalassemia results from point mutations or small deletions in the β-globin (HBB) gene that ultimately cause anemia. The generation of induced pluripotent stem cells (iPSCs) from the somatic cells of patients in combination with subsequent homologous recombination-based gene correction provides new approaches to cure this disease. CRISPR/Cas9 is a genome editing tool that is creating a buzz in the scientific community for treating human diseases, especially genetic disorders. Here, we… CONTINUE READING
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