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CRISPR/Cas9 in the treatment of β-thalassemia
- 1 Capital Medical University
* Author to whom correspondence should be addressed.
Abstract
β-thalassemia is a congenital hemolytic anemia, a class of hemoglobinopathies caused by a common single-gene recessive condition in which the synthesis of the chain is either totally or partially blocked. About 50% of patients have symptoms within the first three months of life. At birth, the disease is often asymptomatic, and it mostly gets worse during infancy. If left untreated, the disease usually claims its victims by the time they are five years old since its severity rises with age. Currently, there is no cure for this disease, and the main treatment is blood transfusion. With the continuous progress of medical level, gene editing technology is developing rapidly, among which CRISPR/Cas system as a gene editing technology has been applied in the research of single gene genetic disease treatment, and CRISPR/Cas9 has been widely used in the research of treating geopathic anemia because of its high efficiency, low cost, and high specificity, for example, repairing pluripotent stem cells’ HBB genes, inducing fetal hemoglobin expression and inhibition of HBA gene expression. This review methodically explains the development of CRISPR/Cas9 through its creation and mode of operation, various research hypotheses tested in animal studies and clinical trials, as well as analyses of safety concerns.
Keywords
CRISPR/Cas9, β-thalassemia, Gene Editing
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Cite this article
Li,Y. (2023). CRISPR/Cas9 in the treatment of β-thalassemia. Theoretical and Natural Science,17,174-178.
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The datasets used and/or analyzed during the current study will be available from the authors upon reasonable request.
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Volume title: Proceedings of the 2nd International Conference on Modern Medicine and Global Health
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