CRISPR Drug Discovery (CRISPR)

The application of CRISPR-Cas gene editing technology to drug development, encompassing target identification through genome-wide knockout screens, target validation, and direct therapeutic gene editing. CRISPR screens can systematically identify gene dependencies in disease models, revealing druggable targets. Also enables creation of isogenic cell lines for precise pharmacological studies.

Örnekler

• Casgevy (exagamglogene autotemcel) for sickle cell disease
• Genome-wide CRISPR screens for cancer drug targets
• CRISPR base editing for hypercholesterolemia (Verve Therapeutics)