Precise genome-editing in human diseases: mechanisms, strategies and applications.

ABSTRACT: Precise genome-editing platforms are versatile tools for generating specific, site-directed DNA insertions, deletions, and substitutions. The continuous enhancement of these tools has led to a revolution in the life sciences, which promises to deliver novel therapies for genetic disease. Precise genome-editing can be traced back to the 1950s with the discovery of DNA's double-helix and, after 70 years of development, has evolved from crude in vitro applications to a wide range of sophisticated capabilities, including in vivo applications. Nonetheless, precise genome-editing faces constraints such as modest efficiency, delivery challenges, and off-target effects. In this review, we explore precise genome-editing, with a focus on introduction of the landmark events in its history, various pl

SUBMITTER: Zheng Y 

PROVIDER: S-EPMC10897424 | biostudies-literature | 2024 Feb

REPOSITORIES: biostudies-literature