Article
A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.
Stem cells translational medicine - 1 Jan 2018
Cai Liuhong, Bai Hao, Mahairaki Vasiliki, Gao Yongxing, He Chaoxia, Wen Yanfei, Jin You-Chuan, Wang You, Pan Rachel L, Qasba Armaan, Ye Zhaohui, Cheng Linzhao
Abstract excerpt
Beta-thalassemia is one of the most common recessive genetic diseases, caused by mutations in the HBB gene. Over 200 different types of mutations in the HBB gene containing three exons have been identified in patients with β-thalassemia (β-thal) whereas a homozygous mutation in exon 1 causes sickle cell disease (SCD). Novel therapeutic strategies to permanently correct the HBB mutation in stem cells that are able...
Topics
- Anemia, Sickle Cell
- CRISPR-Cas Systems
- Cells, Cultured
- Cellular Reprogramming Techniques
- Erythrocytes
- Female
- Gene Editing
- Genetic Therapy
- Humans
- Induced Pluripotent Stem Cells