Advances in CRISPR/Cas9 for Pharmacogenomics and Drug Target Validation
Keywords:
CRISPR/Cas9, Pharmacogenomics, Drug Target Validation, Genome Editing, Functional Genomics, High-Throughput Screening, Personalized MedicineAbstract
The CRISPR/Cas9 genome editing system has revolutionized biomedical research and opened new frontiers in pharmacogenomics and drug discovery. As a powerful, precise, and programmable tool, CRISPR/Cas9 enables targeted manipulation of the genome to study gene function, identify drug response markers, and validate therapeutic targets. In the field of pharmacogenomics, this technology facilitates the creation of isogenic cell lines to understand gene-drug interactions and elucidate mechanisms of drug resistance. In parallel, CRISPR-based genetic screens accelerate the discovery and validation of novel drug targets, particularly for complex diseases such as cancer, neurodegeneration, and infectious diseases. This paper provides a comprehensive review of the applications, benefits, and limitations of CRISPR/Cas9 in pharmacogenomics and drug target validation. It also discusses recent developments in high-throughput screening, base editing, and CRISPR interference/activation (CRISPRi/a) technologies. The integration of CRISPR/Cas9 with other omics platforms holds the potential to usher in a new era of personalized therapeutics. However, off-target effects, delivery challenges, and ethical concerns remain critical hurdles for clinical translation.
DOI: 10.8612/37.3.2022.1
