“CRISPR” refers to clustered regularly interspaced short palindromic repeats. The CRISPR platform technology utilizes a molecular complex comprised of the protein Cas9 together with one or more guide RNAs which binds to the genome in living cells at any desired locations. By this method, researchers are able to specifically alter the DNA sequence at the targeted sites (DNA deletion, insertion or replacement), alter regulation of the target locus or tag the locus with markers.

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(Left panel) CRISPR-Cas9 nuclease complex cleaves target genes to stimulate (i) gene disruption resulting from imperfect DNA repair by non-homologous end joining (NHEJ) or (ii) precise gene insertion or modification by homology directed repair (HDR) of DNA.

(Right panel) Fusions of cleavage-deficient CRISPR-Cas9 with other factors (transcription regulators, fluorescent proteins, other functional moieties) allow site-specific tethering of these functions to chromosomal DNA.