Small molecules promote CRISPR-Cpf1-mediated genome editing in human pluripotent stem cells.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 29610531.
- Also identified by DOI 10.1038/s41467-018-03760-5 and PMC identifier 5880812.
- Licence recorded as CC BY.
- The licence permits redistribution, so the abstract is shown in full and the full text is available from the publisher.
Abstract
Human pluripotent stem cells (hPSCs) have potential applications in biological studies and regenerative medicine. However, precise genome editing in hPSCs remains time-consuming and labor-intensive. Here we demonstrate that the recently identified CRISPR-Cpf1 can be used to efficiently generate knockout and knockin hPSC lines. The unique properties of CRISPR-Cpf1, including shorter crRNA length and low off-target activity, are very attractive for many applications. In particular, we develop an unbiased drug-selection-based platform feasible for high-throughput screening in hPSCs and this screening system enables us to identify small molecules VE-822 and AZD-7762 that can promote CRISPR-Cpf1-mediated precise genome editing. Significantly, the combination of CRISPR-Cpf1 and small molecules provides a simple and efficient strategy for precise genome engineering.
Medical subject headings
- CRISPR-Cas Systems
- Gene Editing
- Genome, Human
- Pluripotent Stem Cells