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Prime editing and its application in beta-hemoglobinopathies.

Authors :
Das, Sankha Subhra
Source :
Nucleus (0029568X); Apr2024, Vol. 67 Issue 1, p227-235, 9p
Publication Year :
2024

Abstract

Genome-editing technologies have provided researchers with the ability to edit DNA mutations associated with various genetic diseases. Prime editing, a 'search-and-replace' gene-editing technology, can correct any base-to-base substitutions, small insertions, and small deletions without requiring double-stranded breaks. Beta-hemoglobinopathies, such as beta-thalassemia and sickle cell anemia, are common monogenic disorders caused by mutations in the beta-globin gene, which alter adult hemoglobin production. Prime editing, the latest genome editing technology, has shown the potential to cure beta-thalassemia and sickle cell anemia by correcting pathogenic mutations. This review presents a basic principle, innovations, delivery strategies, advantages, limitations, and improvements of prime editing, as well as the potential of prime editing to cure beta-thalassemia and sickle cell anemia. [ABSTRACT FROM AUTHOR]

Details

Language :
English
ISSN :
0029568X
Volume :
67
Issue :
1
Database :
Complementary Index
Journal :
Nucleus (0029568X)
Publication Type :
Academic Journal
Accession number :
177350459
Full Text :
https://doi.org/10.1007/s13237-024-00470-w