Pharmaceutical Applications of CRISPR Technology in Drug Development: Precision Genome Engineering Strategies for Target Identification, Therapeutic Design, and Translational Nanomedicine
Abstract
The advent of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) technology has revolutionized pharmaceutical sciences by enabling precise genome manipulation for drug discovery and therapeutic development. Traditional drug development paradigms face limitations in target validation, therapeutic specificity, and translational efficiency. CRISPR-Cas systems, particularly CRISPR-Cas9, Cas12, and Cas13, offer unprecedented capabilities for functional genomics, disease modeling, and direct therapeutic intervention. This review examines the integration of CRISPR technology across pharmaceutical drug development pipelines, encompassing target identification through high-throughput screening, validation via loss-of-function studies, and therapeutic design including ex vivo and in vivo gene editing approaches. Critical emphasis is placed on delivery strategies, including viral vectors, lipid nanoparticles, and polymer-based carriers that enable clinical translation. Current applications span oncology, rare genetic disorders, infectious diseases, and metabolic conditions, with multiple candidates advancing through clinical trials. Challenges including off-target effects, immunogenicity, and regulatory frameworks are discussed alongside emerging solutions. The pharmaceutical significance of CRISPR extends beyond gene correction to encompass epigenome editing, transcriptional modulation, and precision medicine paradigms, positioning this technology as a cornerstone of next-generation drug development strategies.
How to Cite This Article
Jonathan Michael Whitaker, Alyssa Renee Harrington (2024). Pharmaceutical Applications of CRISPR Technology in Drug Development: Precision Genome Engineering Strategies for Target Identification, Therapeutic Design, and Translational Nanomedicine . International Journal of Pharma Insight Studies (IJPIS), 1(4), 45-53.