CRISPR-Based Therapeutics: Pharmacological Mechanisms and Pharmaceutical Delivery Challenges

Abstract

CRISPR-Cas systems have revolutionized gene editing, offering unprecedented potential for therapeutic intervention in genetic, infectious, and neoplastic diseases. While the pharmacological mechanisms of CRISPR therapeutics involve precise DNA or RNA targeting, pharmaceutical delivery remains a critical challenge, limiting clinical translation. This review provides a comprehensive overview of CRISPR-based therapeutics, including molecular mechanisms, target specificity, off-target considerations, and current delivery strategies such as viral vectors, lipid nanoparticles, and polymeric carriers. Key challenges in stability, immunogenicity, tissue-specific targeting, and regulatory considerations are discussed. Emerging innovations, including stimuli-responsive and targeted delivery systems, are highlighted, emphasizing the integration of pharmacology and pharmaceutics for safe and effective CRISPR therapy.