CRISPR/Cas: from adaptive immune system in prokaryotes to therapeutic weapon against immune-related diseases

Abstract

CRISPR/Cas evolved as an adaptive immune system in bacteria and archaea to inactivate foreign viral and plasmid DNA. However, the capacities of various CRISPR/Cas systems for precise genome editing based on sequence homology also allow their use as tools for genomic and epigenomic modification in eukaryotes. Indeed, these genetic characteristics have proven useful for disease modeling and testing the specific functions of target genes under pathological conditions. Moreover, recent studies provide compelling evidence that CRISPR/Cas systems could be useful therapeutic tools against human diseases, including cancer, monogenic disorders, and autoimmune disorders.

Highlights

• CRISPR/Cas evolved as an adaptive immune system in bacteria and archaea.

• CRISPR/Cas systems are nowadays used as tools for genomic modification.

• CRISPR/Cas systems could be useful therapeutic tools against human disease, including autoimmune conditions.

Keywords:

• CRISPR/Cas
• evolution
• gene editing
• monogenic disease
• autoimmune disease

Disclosure statement

The authors declare no conflict of interest

Acknowledgments

Dr. García extends special appreciation to the Rheumatology team of Fundación Valle del Lili in Cali, Colombia, for support during medical training as well as to the research group on rheumatology, autoimmunity and translational medicine (GIRAT) at Universidad Icesi (Gabriel J. Tobón MD PhD, Carlos Cañas MD, David Aguirre MD, Erika Navarro MD, Alex Echeverri MD and Anilza Bonelo PhD). Dr. Garcia also wants to thank Natalia Arango Mesa MD for the support. All authors thank Lady J. Rios-Serna BSc from GIRAT for assistance in graphic design, and Adolfo Abadía (Universidad Icesi) for Editorial Support.