CRISPR is an RNA-guided gene-editing platform that makes use of a bacterially derived protein (Cas9) and a synthetic guide RNA to introduce a double strand break at a specific location within the genome. Editing is achieved by transfecting a cell with the Cas9 protein along with a specially designed guide RNA (gRNA) that directs the cut through hybridization with its matching genomic sequence.   
   
Use of wild-type Cas9 has been shown to lead to off-target cleavage, but a modified version introduces only single strand “nicks” to the DNA, which can be exploited to introduce subtle genetic changes while avoiding off-target effects common to double strand breaks.   
   
Horizon has licensed CRISPR IP from Harvard University to ensure that we will be able to offer uninterrupted use of this technology to customers.