AUTHOR=Macarrón Palacios Arturo , Korus Patrick , Wilkens Bodo G. C. , Heshmatpour Najmeh , Patnaik Sarita R. 

TITLE=Revolutionizing in vivo therapy with CRISPR/Cas genome editing: breakthroughs, opportunities and challenges

JOURNAL=Frontiers in Genome Editing

VOLUME=6

YEAR=2024

URL=https://www.frontiersin.org/journals/genome-editing/articles/10.3389/fgeed.2024.1342193

DOI=10.3389/fgeed.2024.1342193

ISSN=2673-3439

ABSTRACT=<p>Genome editing using the CRISPR/Cas system has revolutionized the field of genetic engineering, offering unprecedented opportunities for therapeutic applications <italic>in vivo</italic>. Despite the numerous ongoing clinical trials focusing on <italic>ex vivo</italic> genome editing, recent studies emphasize the therapeutic promise of <italic>in vivo</italic> gene editing using CRISPR/Cas technology. However, it is worth noting that the complete attainment of the inherent capabilities of <italic>in vivo</italic> therapy in humans is yet to be accomplished. Before the full realization of <italic>in vivo</italic> therapeutic potential, it is crucial to achieve enhanced specificity in selectively targeting defective cells while minimizing harm to healthy cells. This review examines emerging studies, focusing on CRISPR/Cas-based pre-clinical and clinical trials for innovative therapeutic approaches for a wide range of diseases. Furthermore, we emphasize targeting cancer-specific sequences target in genes associated with tumors, shedding light on the diverse strategies employed in cancer treatment. We highlight the various challenges associated with <italic>in vivo</italic> CRISPR/Cas-based cancer therapy and explore their prospective clinical translatability and the strategies employed to overcome these obstacles.</p>