Article
Recent advance on genome editing for therapy of β-hemoglobinopathies.
Yi chuan = Hereditas - 20 Feb 2018
Liu Jia-Wei, Hong Tao, Qin Xin, Liang Ying-Min, Zhang Ping
Abstract excerpt
β-hemoglobinopathies are one of six groups of common illnesses affecting human health. Although the genetic mechanisms have been elucidated for several decades, curable treatment options, other than allogeneic bone marrow transplantation, are still lacking. In recent years, rapid development in genome editing technologies and their clinical applications have opened up new directions for treatment of...
Topics
- CRISPR-Cas Systems
- Fetal Hemoglobin
- Gene Editing
- Genetic Therapy
- Hemoglobinopathies
- Humans
- Mutation
- Translational Research, Biomedical
- beta-Globins
