Advancements in CRISPR-Cas9 Gene Editing: A Comprehensive Review of Therapeutic Applications and Ethical Considerations
Authors: ZHANG Wei, LI Ming, WANG Fang
CRISPR-Cas9 technology has revolutionized the field of genetic engineering, offering unprecedented precision and efficiency in genome editing. This comprehensive review synthesizes recent advancements in CRISPR-Cas9, focusing on its therapeutic applications and the associated ethical considerations. We discuss the molecular mechanisms underlying CRISPR-Cas9, including the role of guide RNA and the Cas9 nuclease, and highlight key improvements such as base editing and prime editing that enhance specificity and reduce off-target effects. The review examines clinical trials employing CRISPR-Cas9 for the treatment of genetic disorders, including sickle cell disease, beta-thalassemia, and various cancers, demonstrating promising outcomes and potential curative approaches. Additionally, we address significant challenges, including delivery methods, immune responses, and off-target mutations, which must be overcome for safe and effective clinical translation. Ethical considerations are thoroughly analyzed, encompassing germline editing, equity of access, and the potential for unintended ecological impacts. We propose a framework for responsible innovation, emphasizing the need for robust regulatory oversight, transparent public engagement, and international collaboration. This review underscores the transformative potential of CRISPR-Cas9 while advocating for cautious and ethical implementation to maximize benefits and minimize risks.
