CRISPR-Cas9-Driven Drug Screening: Customizing Cell Models for Precision Medicine

CRISPR-Cas9 technology has revolutionized the field of drug screening and toxicity testing by enabling researchers to create precisely tailored cell models that mimic specific genetic variants. Through targeted gene editing, CRISPR-Cas9 enables scientists to modify genes of interest within cell lines, mimicking the genetic background of different patient populations or disease states. This innovation has important implications for high-throughput drug screening, allowing researchers to assess how genetic variants affect drug response and toxicity profiles in different genotypes.

In CRISPR-Cas9-enabled drug screening approaches, specific genes in cell models are modified to reflect specific genetic mutations associated with disease or drug metabolism. Researchers can then expose these cells to various drug compounds and observe their responses. For example, a drug that performs well in a cell line with a specific oncogenic mutation can be identified as a potential candidate for patients with that genetic signature. Similarly, CRISPR-modified cells can be used to screen for drug toxicity, revealing how certain drugs can produce harmful side effects in cells with specific genetic variants.

This precision approach allows scientists to determine the efficacy and safety of potential drugs in a more targeted and personalized way, reducing the likelihood of adverse reactions later in development. In addition, CRISPR-Cas9 can be used for large-scale, high-throughput testing, accelerating the process of drug discovery by rapidly screening large numbers of compounds in different genetic backgrounds. By using CRISPR to create diverse, genetically customized cell models, researchers can streamline the drug development process and select only the most promising drug candidates for further development. This strategy not only improves the efficiency of drug discovery, but also supports the development of safer and more effective treatments tailored to individual genetic characteristics.

?

References

[1] Hui Xing and Ling-hua Meng et al., Acta Pharmacologica Sinica 2019 (https://doi.org/10.1038/s41401-019-0322-9)

[2] Shady Adnan Awad et al., Cell Reports Medicine 2024 (DOI: 10.1016/j.xcrm.2024.101521)