From Bench to Bedside: Crispr Gene Editing in Therapeutic Development

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)–based gene editing technologies have recently emerged as game-changing tools in biomedical research, new drug development, and therapeutic intervention.1,3,6 Evolved from bacterial adaptive immune systems, CRISPR-associated Cas nucleases offer the ability to modify DNA and RNA sequences.1,6 with unprecedented programmability and accuracy. Compared to previous generations of gene editing tools, CRISPR delivers superior efficacy, scalability, and versatility, thereby accelerating the translation of basic genomic knowledge toward therapeutic concepts.4,7 In new drug development, CRISPR technologies empower genome-scale functional screening, accelerated target identification, and validation, investigation of drug resistance mechanisms, and establishment of physiologically relevant disease models.4,5,7 The rapeutically, CRISPR-based approaches have been clinically effective in monogenic disorders, oncology, cardiovascular and metabolic diseases, neurological conditions, and infectious diseases, resulting in regulatory approval for the first CRISPR-edited cell therapies.14,17,19 Despite excellent progress, obstacles associated with delivery efficiency, off-target effects, immunogenicity, ethical concerns, and regulatory oversight remain major hurdles to broad clinical adoption.12,13,19 In this review, we provide a brief but comprehensive overview of CRISPR and gene editing technologies, focusing on their applications in drug discovery and therapy, current clinical progress, considerations on safety, and future perspectives.7,19 Collectively, CRISPR-based gene editing points to a paradigm shift toward precision medicine and next-generation therapeutics.6,7