Expert view: CRISPR screening: a powerful approach to drug discovery and development

It is also used to explore the effect of genetic mutations on drug activity, patient responsiveness and resistance.

This screening method uses CRISPR-Cas9 technology to modify and assess the function of thousands of genes in a single experiment, allowing researchers to quickly identify the genes relevant to a specific biological pathway. The most common approach, CRISPR KO (knockout) delivers complete gene inactivation, while CRISPRi (inactivate) and CRISPRa (activate) are used to target the endogenous gene at the transcriptional initiation site. These mechanisms can be combined to deliver dual loss-of-function or dual direction screening, conducted in either a pooled or arrayed format.

The speed with which CRISPR has enhanced therapeutic development is remarkable. This highly specific screening approach has enabled researchers to not only identify the compounds more likely to be successful in clinical trials but also stratify a patient population to determine which therapeutics will be most effective, helping to reduce the risk of trial failure. This technology can also be used to find the genes responsible for a particular cell behaviour, such as differentiation or required to engineer effective T cells for immunotherapies.

Horizon Discovery has more than 10 years’ of experience working in gene engineering and leads the way in offering a broad range of CRISPR screening services – from initial design and cell-line selection to bioinformatic analysis of the screen results. The breadth of our platforms enables us to design custom solutions for almost any screening requirement and cellular background, including primary cancer lines and primary immune cells. We are actively engaged with biopharmaceutical companies, providing services that save time and resources while they navigate towards a successful therapeutic.