Scientific Publications

Multiplex Genome Engineering Using CRISPR/Cas Systems.

Publication TypeJournal Article
AuthorsCong, L., Ran FA, Cox D., Lin S., Barretto R., Habib N., Hsu PD, Wu X., Jiang W., Marraffini LA, and Zhang F.
AbstractFunctional elucidation of causal genetic variants and elements requires precise genome editing technologies. The type II prokaryotic CRISPR (clustered regularly interspaced short palindromic repeats) adaptive immune system has been shown to facilitate RNA-guided site-specific DNA cleavage. We engineered two different type II CRISPR systems and demonstrate that Cas9 nucleases can be directed by short RNAs to induce precise cleavage at endogenous genomic loci in human and mouse cells. Cas9 can also be converted into a nicking enzyme to facilitate homology-directed repair with minimal mutagenic activity. Finally, multiple guide sequences can be encoded into a single CRISPR array to enable simultaneous editing of several sites within the mammalian genome, demonstrating easy programmability and wide applicability of the CRISPR technology.
Year of Publication2013
JournalScience (New York, N.Y.)
Date Published (YYYY/MM/DD)2013/01/03
ISSN Number0036-8075
DOI10.1126/science.1231143
PubMedhttp://www.ncbi.nlm.nih.gov/pubmed/23287718?dopt=Abstract