Koodamvetty Abhijith, Thangavel Saravanabhavan
Centre for Stem Cell Research (CSCR), A unit of InStem Bengaluru, Christian Medical College campus, Vellore, Tamil Nadu, 632002, India.
Manipal Academy of Higher Education, Manipal, Karnataka, 576104, India.
Adv Sci (Weinh). 2025 Apr;12(14):e2410237. doi: 10.1002/advs.202410237. Epub 2025 Mar 2.
The advent of gene editing has significantly advanced the field of medicine, opening new frontiers in the treatment of genetic disorders, cancer, and infectious diseases. Gene editing technology remains a dynamic and promising area of research and development. Recent advancements in protein and RNA engineering within this field have addressed critical issues such as imprecise edits, poor editing efficiency, and off-target effects. Advancements in delivery methods have allowed the achievement of therapeutic or even selection-free gene editing efficiency with reduced toxicity in primary cells, thereby enhancing the safety and efficacy of gene manipulation. This progress paves the way for transformative changes in molecular biology, medicine, and other fields. This review provides a comprehensive overview of the advancements in gene editing techniques, focusing on prime editor proteins and their engineered variants. It also explores alternative systems that expand the toolkit for precise genomic modifications and highlights the potential of these innovations in treating hematological disorders, while also discussing the limitations and challenges that remain.
基因编辑的出现极大地推动了医学领域的发展,为遗传疾病、癌症和传染病的治疗开辟了新的前沿领域。基因编辑技术仍然是一个充满活力且前景广阔的研发领域。该领域内蛋白质和RNA工程的最新进展已经解决了诸如编辑不精确、编辑效率低和脱靶效应等关键问题。递送方法的进步使得在原代细胞中能够实现治疗性甚至无选择的基因编辑效率,同时降低毒性,从而提高了基因操作的安全性和有效性。这一进展为分子生物学、医学和其他领域的变革性变化铺平了道路。本综述全面概述了基因编辑技术的进展,重点关注引导编辑器蛋白及其工程变体。它还探讨了扩展精确基因组修饰工具包的替代系统,并强调了这些创新在治疗血液疾病方面的潜力,同时也讨论了仍然存在的局限性和挑战。