Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 22, Issue 16, Pages -Publisher
MDPI
DOI: 10.3390/ijms22168571
Keywords
CRISPR; Cas9; genome editing; homology-directed repair; small molecules; engineered Cas9
Funding
- National Health and Medical Research Council (NHMRC) [APP1107514, APP1158164, APP1158165, APP1185002]
- NSW Ministry of Health
Ask authors/readers for more resources
Genome modification holds great potential for disease prevention or treatment, with CRISPR/Cas9 techniques showing promise in altering disease-relevant genes. Competition among DNA repair pathways can lead to undesirable editing outcomes, but small molecule modulators and engineered CRISPR/Cas proteins have been shown to enhance precision editing efficacy.
Modification of the human genome has immense potential for preventing or treating disease. Modern genome editing techniques based on CRISPR/Cas9 show great promise for altering disease-relevant genes. The efficacy of precision editing at CRISPR/Cas9-induced double-strand breaks is dependent on the relative activities of nuclear DNA repair pathways, including the homology-directed repair and error-prone non-homologous end-joining pathways. The competition between multiple DNA repair pathways generates mosaic and/or therapeutically undesirable editing outcomes. Importantly, genetic models have validated key DNA repair pathways as druggable targets for increasing editing efficacy. In this review, we highlight approaches that can be used to achieve the desired genome modification, including the latest progress using small molecule modulators and engineered CRISPR/Cas proteins to enhance precision editing.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available