Applications of CRISPR Technology in Gene Editing and Genetic Diseases

The advent of CRISPR-Cas genome editing technology has revolutionized biological research and holds immense promise for the treatment of genetic diseases. This review provides a comprehensive overview of CRISPR applications in gene editing, focusing on its mechanisms, diverse systems, and applications in various biomedical fields. We delve into the molecular underpinnings of CRISPR-Cas۹, Cas۱۲, and Cas۱۳ systems, elucidating their distinct modes of action in targeting and modifying DNA or RNA. The review details key gene editing strategies, including gene knockout, gene correction, and base editing, and explores their implementation in correcting disease-causing mutations. Significant attention is dedicated to therapeutic applications for inherited disorders such as sickle cell disease, beta-thalassemia, cystic fibrosis, Duchenne muscular dystrophy, and inherited blindness, highlighting the potential to restore normal gene function. Furthermore, we examine CRISPR's role in cancer research and therapy, as well as its application in controlling infectious diseases. Crucial aspects of CRISPR delivery methods, including viral vectors, lipid nanoparticles, and ribonucleoprotein complexes, are discussed in detail. The review also addresses the critical safety challenges, particularly off-target effects, and explores the complex ethical, legal, and social considerations surrounding germline editing and regulatory frameworks. Finally, we present a detailed analysis of recent clinical trials and advances in CRISPR technology published between ۲۰۲۲ and ۲۰۲۵, alongside a forward-looking perspective on the future of CRISPR medicine.

کلیدواژه ها:

CRISPR ، Gene Editing ، Genetic Diseases ، Genome Engineering ، Therapeutic Applications ، Molecular Mechanisms ، Clinical Trials