Park, Byung-Sun Lee, Mieun Kim, Jaeyeol Kim, Tackhoon 2025-07-18T06:00:28Z 2025-07-18T06:00:28Z 2025-07-18 2025-07 1226-3613 https://pubs.kist.re.kr/handle/201004/152769 Despite more than two decades since the completion of the first draft of the Human Genome Project, a substantial proportion of human genes remain poorly characterized in terms of their functions. Functional genomics aims to elucidate the roles and interactions of genes and genetic elements, providing insights into their involvement in various biological processes. In this context, the perturbomics approach-a systematic analysis of phenotypic changes resulting from gene function modulation-offers valuable insights into the function of unannotated genes. With the advent of CRISPR-Cas-based genome and epigenome editing, CRISPR screens have become the method of choice for perturbomics studies, enabling the identification of target genes whose modulation may hold therapeutic potential for diseases such as cancer, cardiovascular disorders and neurodegeneration. These findings contribute to the development of targeted drug therapies and the design of gene and cell therapies for regenerative medicine. Here we highlight recent technical advances in CRISPR-based perturbomics, focusing on more physiologically relevant, single-cell-level analyses and their successful applications in discovering novel therapeutic strategies. English Springer Nature Perturbomics: CRISPR-Cas screening-based functional genomics approach for drug target discovery Article 10.1038/s12276-025-01487-0 1 Experimental & Molecular Medicine, v.57, no.7, pp.1443 - 1454 Experimental & Molecular Medicine 57 7 1443 1454 Y scie scopus kci 001520398200001 Biochemistry & Molecular Biology Medicine, Research & Experimental Biochemistry & Molecular Biology Research & Experimental Medicine Review PANCREATIC-CANCER CELLS GENES DEPENDENCY MECHANISMS RESISTANCE REGULATORS CIRCUITS