Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation.

@article{Huang2015ProductionOG,
  title={Production of Gene-Corrected Adult Beta Globin Protein in Human Erythrocytes Differentiated from Patient iPSCs After Genome Editing of the Sickle Point Mutation.},
  author={Xiaosong Huang and Ying Wang and Wei Yan and Cory D Smith and Zhaohui Ye and Jing Wang and Yongxing Gao and Laurel G Mendelsohn and Linzhao Cheng},
  journal={Stem cells},
  year={2015},
  volume={33 5},
  pages={1470-9}
}
Human induced pluripotent stem cells (iPSCs) and genome editing provide a precise way to generate gene-corrected cells for disease modeling and cell therapies. Human iPSCs generated from sickle cell disease (SCD) patients have a homozygous missense point mutation in the HBB gene encoding adult β-globin proteins, and are used as a model system to improve strategies of human gene therapy. We demonstrate that the CRISPR/Cas9 system designer nuclease is much more efficient in stimulating gene… CONTINUE READING
Recent Discussions
This paper has been referenced on Twitter 55 times over the past 90 days. VIEW TWEETS

Citations

Publications citing this paper.
Showing 1-10 of 60 extracted citations

Similar Papers

Loading similar papers…