Translational and Clinical Progress in Genome Editing

Genome editing stands at the forefront of modern biomedical research, offering transformative potential for understanding, preventing, and treating human diseases. In recent years, technologies such as CRISPR-Cas systems, base editing, and prime editing have rapidly evolved, showcasing unprecedented accuracy and versatility in modifying genetic material. While these breakthroughs have revolutionized preclinical models and disease research, significant challenges persist in bringing these innovations safely and effectively to clinical practice. Central questions remain about optimal delivery methods, long-term effects, immunogenicity, safety, and ethical considerations for genome editing therapies. Despite remarkable progress in laboratory settings and proof-of-concept studies in animal models, translating these advances into robust, standardized, and equitable therapies for patients continues to be a complex and urgent endeavor.This Research Topic aims to accelerate the translation of genome editing technologies from the laboratory to the clinic by fostering innovative research and dialogue across disciplines. The main objectives are to explore and address the key scientific, technical, regulatory, and ethical barriers to clinical implementation; to highlight progress in preclinical and clinical trial design; and to deepen our understanding of safety, manufacturing, and societal impacts. By promoting studies that tackle pivotal questions—such as how to maximize efficacy while minimizing off-target effects, how to select and monitor patients, and how to ensure equitable access—this Research Topic seeks to shape the evolving landscape of genome editing for therapeutic benefit.Focusing on the pathway from discovery to clinical application, this Research Topic welcomes original research, review articles, and perspectives spanning a wide range of diseases, delivery methods, and implementation frameworks. Submissions should primarily address developments and challenges associated with translational and clinical genome editing. To gather further insights into advancing genome editing toward clinical practice, we welcome articles addressing, but not limited to, the following themes:o Optimization and innovation in genome editing platformso Preclinical models and studies for therapeutic applicationso Advances in in vivo and ex vivo delivery technologieso Clinical trial design, early-phase studies, and regulatory considerationso Assessment and mitigation of safety and off-target effectso Ethical, legal, and societal implications of genome editingo Manufacturing, scalability, and standardization of editing reagents and therapiesWe especially encourage the submission of original research articles, systematic reviews and FAIR2 Data articles. Topic editors: Rasmani Hazra, Disha Aggarwal Source: https://www.frontiersin.org/research-topics/80592/translational-and-clinical-progress-in-genome-editing