Perturbomics: CRISPR-Cas screening-based functional genomics approach for drug target discovery

Area of Science:

• Genomics
• Functional Genomics
• Systems Biology

Background:

• A significant number of human genes lack functional characterization despite the Human Genome Project.
• Functional genomics seeks to understand gene roles and interactions in biological processes.
• Perturbomics systematically analyzes phenotypic changes from gene modulation to reveal gene functions.

Purpose of the Study:

• To highlight technical advances in CRISPR-based perturbomics.
• To focus on single-cell analyses for physiologically relevant insights.
• To showcase applications in discovering novel therapeutic strategies.

Main Methods:

• Utilizing CRISPR-Cas-based genome and epigenome editing for gene function modulation.
• Employing CRISPR screens as a primary method for perturbomics studies.
• Implementing single-cell-level analyses for enhanced physiological relevance.

Main Results:

• CRISPR screens identify target genes for therapeutic intervention.
• Perturbomics facilitates the discovery of functions for unannotated genes.
• Advances enable more accurate, physiologically relevant functional genomics studies.

Conclusions:

• CRISPR-based perturbomics is crucial for understanding gene function.
• This approach aids in developing targeted therapies for cancer, cardiovascular, and neurodegenerative diseases.
• Single-cell analyses in perturbomics drive innovation in gene and cell therapies.