Revvity Horizon Discovery Pin-point Base Editing Platform

The Horizon Discovery Pin-point Base Editing platform from Revvity, with its novel three-part design and strong safety profile, is designed to enhance base editing’s capabilities by enabling simultaneous modification of several genes at once, thereby expanding its application in human disease modelling.

Base editing is a CRISPR-Cas9-based technology that allows researchers to make precision base changes in genomic DNA. Editing with such precision can be used to silence disease-causing genes, correct disease-associated mutations and optimise cell therapies.

Pin-point base editing requires three key components: guide RNA with patented and proprietary aptamer; modified Cas9 (nickase Cas9, or nCas9); and deaminase fused to an aptamer binding protein. The gRNA guides the modified Cas9 to a specific location in the genome where a single strand ‘nick’ is made in the DNA. Meanwhile, the deaminase enzyme is recruited to the site and modifies bases on the non-nicked strand, for example, C>T conversions. The modified DNA strand is then used as a repair template for the nicked strand to complete the base pair conversion.

Compared to traditional CRISPR technologies that create double-stranded breaks in the DNA, base editing only nicks one strand of the DNA making it safer for therapeutic use. Key applications include: the creation of gene knock-out/SNP correction; proprietary concurrent knock-out and knock-in demonstrated; multiplexed gene editing for complex cell models; the ability to edit cells sensitive to the DNA damage response pathway; mutagenesis for functional gene characterisation; and orthogonal validation (RNAi, CRISPRi, traditional CRISPR/Cas9).