Article
Development of gene editing strategies for human β-globin (HBB) gene mutations.
Gene - 15 Apr 2020
Kalkan Batuhan Mert, Kala Ezgi Yagmur, Yuce Melek, Karadag Alpaslan Medine, Kocabas Fatih
Abstract excerpt
Recent developments in gene editing technology have enabled scientists to modify DNA sequence by using engineered endonucleases. These gene editing tools are promising candidates for clinical applications, especially for treatment of inherited disorders like sickle cell disease (SCD). SCD is caused by a point mutation in human β-globin gene (HBB). Clinical strategies have demonstrated substantial success, however...
Topics
- Anemia, Sickle Cell
- CRISPR-Cas Systems
- DNA Breaks, Double-Stranded
- DNA Repair
- Gene Editing
- Genetic Vectors
- HEK293 Cells
- Humans
- Mutation
- RNA, Guide, CRISPR-Cas Systems
- beta-Globins