Molecular mechanisms, off-target activities, and clinical potentials of genome editing systems.

ABSTRACT: Methodologies of genome editing are rapidly developing with the improvement of gene science and technology, mechanism-based understanding, and urgent needs. In addition to the specificity and efficiency of on-target sites, one of the most important issues is to find and avoid off-targets before clinical application of gene editing as a therapy. Various algorithms, modified nucleases, and delivery vectors are developed to localize and minimize off-target sites. The present review aimed to clarify off-targets of various genome editing and explore potentials of clinical application by understanding structures, mechanisms, clinical applications, and off-target activities of genome editing systems, including CRISPR/Cas9, CRISPR/Cas12a, zinc finger nucleases, transcription activator-like effecto

SUBMITTER: Zheng N 

PROVIDER: S-EPMC7240848 | biostudies-literature | 2020 Jan

REPOSITORIES: biostudies-literature