Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells.
basic_science · Level V
Where this comes from
- Record sourced from PubMed, PMID 26121415.
- Also identified by DOI 10.1038/nbt.3290 and PMC identifier 4729442.
- No licence information is recorded for this record.
- Because redistribution is not established, this page shows the abstract only. Follow the links below for the full text.
Abstract
CRISPR-Cas-mediated genome editing relies on guide RNAs that direct site-specific DNA cleavage facilitated by the Cas endonuclease. Here we report that chemical alterations to synthesized single guide RNAs (sgRNAs) enhance genome editing efficiency in human primary T cells and CD34(+) hematopoietic stem and progenitor cells. Co-delivering chemically modified sgRNAs with Cas9 mRNA or protein is an efficient RNA- or ribonucleoprotein (RNP)-based delivery method for the CRISPR-Cas system, without the toxicity associated with DNA delivery. This approach is a simple and effective way to streamline the development of genome editing with the potential to accelerate a wide array of biotechnological and therapeutic applications of the CRISPR-Cas technology.
Medical subject headings
- CRISPR-Cas Systems
- Clustered Regularly Interspaced Short Palindromic Repeats
- Genetic Engineering
- Genome, Human
- RNA, Guide, CRISPR-Cas Systems