
Cells, Journal Year: 2024, Volume and Issue: 13(23), P. 1963 - 1963
Published: Nov. 27, 2024
Gene therapy is a promising approach to the treatment of various inherited diseases, but its development complicated by number limitations natural promoters used. The currently used strong ubiquitous do not allow for specificity expression, while tissue-specific have lowactivity. These can be addressed creating new synthetic that achieve high levels target gene expression. This review discusses recent advances in provide more precise regulation Approaches design are reviewed, including manual and bioinformatic methods using machine learning. Examples successful applications hereditary diseases cancer presented, as well prospects their clinical use.
Language: Английский