CRISPR Cas9 Gene Editing for Target Identification and Validation



Drug Discovery Services

  • Experienced and qualified scientists functioning as project managers or study director
  • Independent quality unit assuring regulatory compliance
  • Methods validated per ICH GLP/GMP guidelines
  • Rigorous sample tracking and handling procedures to prevent mistakes
  • Controlled laboratory environment to prevent a whole new level of success

CRISPR Cas9 Gene Editing for Target Identification and Validation


CRISPR Cas9 system has rapidly developed and widely used in genomic screening because of its specificity and irreversibility. This unique technique enables to create more predictive cell-based models and reveal new drug targets, showing great potential in the early drug discovery phase from target identification to validation.

Fig.1 The picture  of gene editing technology.

Fig.1 The picture of gene editing technology.

Gene editing technology is of primary importance in the drug discovery. Scientists can develop a series of experiments to find disease-related targets and investigate their therapeutic effects through altering the sequence of genes or their expression levels.

CRISPR Cas9 gene editing system consists of a Cas9 protein (or other homologous protein) and a single guide RNA (gRNA). The function of the CRISPR system involves RNA-guided cleavage of the exogenous double-stranded DNA, followed by imprecise repair through either imprecise non-homologous end joining (NHEJ) or template-dependent homologous directed repair (HDR) pathways. CRISPR Cas9 works just like a pair of molecular scissors in which Cas9 protein is used to respond to and eliminate invading genetic material. Scientists can use Cas9 to cut into almost any gene or site, inducing cell repair and facilitating gene knockout, knock-in, or site-directed mutation by designing a short gRNA sequence.

Fig.2 The picture  of CRISPR Cas9.

Fig.2 The picture of CRISPR Cas9.

Application in Drug Discovery

Target discovery and validation

CRISPR Cas9 technology-based high-throughput screening systems can be used to knock out (CRISPR-KO), inhibit (CRISPRi) or activate (CRISPRa) of candidate genes on a large scale. Potential drug targets can be identified by observing the deterioration or remission of disease phenotypes.

High throughput compound screening

Mutant cell lines associated with disease can be constructed more accurately with the application of CRISPR Cas9, accelerating the process of cell-line-based screening. With these cell models, ineffective compounds can be accurately excluded and effective targets will be identified in the early stage of drug discovery, saving time and reducing costs.

Validation of candidate compounds

A variety of isogenotypic cell lines can be easily constructed and genetic variants associated with disease can be generated through precise genomic modification. The rapid and accurate construction of disease-related cell line models obtained by CRISPR technology greatly improves the efficiency of candidate compound validation and enables more accurate evaluation of potential compounds.

Lead compound discovery and validation

Cell line models and animal models generated by CRISPR technology can be used in the efficacy and safety assessment of lead compounds, which improve the efficiency of the lead drug validation.

CRISPR Cas9 Gene Editing Services

  • Validate the interaction between a candidate compound and the intended drug targets.
  • Discriminate between on-target and off-target effects using CRISPR Cas9.
  • Screen the related genes that regulate phenotypes, such as the genes that inhibit chemotherapy drugs or toxins, the genes that affect tumor migration, and the construction of virus screening library for a wide range of potential genes.
  • Develop pooled and arrayed high-throughput screens (pooled screening generates relatively clear-cut readouts including cell proliferation, cell death or sortable marker proteins, arrayed screens are for more complex phenotypic readouts developed from high content imaging).

Our Advantages of CRISPR Cas9 Gene Editing Services

  • With optimized plans and validated editing strategies, we have already successfully executed hundreds of projects and have a much high success rate of all projects, including knock-out and knock-ins (point mutations and large fragment insertions).
  • A multitude of high-throughput screening can be designed to simulate specific targets in disease-relevant cells combined with our CRISPR Cas9 Gene editing technology and our strong capabilities in cell-line development.
  • In target validation, we create the target of interest more precisely when developing assays using CRISPR technology, helping to deliver more lead candidates.

※ It should be noted that our service is only used for research.

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