CRISPR-Based Gene Editing in Clinical Therapeutics: Challenges and Breakthroughs

Abstract

CRISPR-Cas systems have rapidly transformed the landscape of gene editing, offering unprecedented precision and efficiency in modifying the genome. Over the past decade, CRISPR technology has evolved from a molecular biology tool into a promising platform for clinical therapeutics, with applications spanning monogenic disorders, cancer, infectious diseases, and beyond. This review provides a comprehensive overview of the current state of CRISPR-based therapies in clinical development, highlighting key breakthroughs such as ex vivo edited T cells for cancer immunotherapy and in vivo editing for conditions like transthyretin amyloidosis and sickle cell disease. Despite these advances, significant challenges remain, including delivery efficiency, off-target effects, immunogenicity, and ethical considerations. We discuss innovative strategies to overcome these hurdles, including base and prime editing, novel delivery vectors, and transient CRISPR systems. Additionally, regulatory, manufacturing, and access-related aspects of CRISPR therapeutics are examined. As the field moves closer to widespread clinical application, understanding both the scientific and translational barriers is critical to harnessing the full potential of gene editing in medicine.