Ontology highlight
ABSTRACT:
SUBMITTER: Li P
PROVIDER: S-EPMC6319323 | biostudies-literature | 2018 Feb
REPOSITORIES: biostudies-literature
Li Pingjuan P Kleinstiver Benjamin P BP Leon Mihoko Y MY Prew Michelle S MS Navarro-Gomez Daniel D Greenwald Scott H SH Pierce Eric A EA Joung J Keith JK Liu Qin Q
The CRISPR journal 20180201
Treatment strategies for dominantly inherited disorders typically involve silencing or ablating the pathogenic allele. CRISPR-Cas nucleases have shown promise in allele-specific knockout approaches when the dominant allele creates unique protospacer adjacent motifs that can lead to allele-restricted targeting. Here, we present a spacer-mediated allele-specific knockout approach that utilizes both SpCas9 variants and truncated single-guide RNAs to achieve efficient discrimination of a single-nucl ...[more]