Prime editing and its application in beta-hemoglobinopathies

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.