CRISPR Cas9 based genome editing in inherited retinal dystrophies

Background Precision genome engineering, with targeted therapy towards patient-specific mutations is predicted to be the future of personalized medicine. Ophthalmology is in the frontiers of development of targeted therapy since the eye is an accessible organ and has the ease of both delivery as well as monitoring effects of therapy. Materials and Methods We reviewed literature using keywords CRISPR, precision medicine, genomic editing, retinal dystrophies, retinitis pigmentosa, Usher syndrome, Stargardt's Disease. Further, we collated data on current clinical trials. Results There is growing evidence on the role of genomic editing in retinal dystrophies, the various methods used, and stage of development of different therapies have been summarized in this paper. Conclusions The CRISPR-Cas9 system has revolutionized genome editing, and opened avenues in drug discovery. It is important to understand the role of this system along with its applicability in the field of ophthalmology. In this review article, we briefly describe its methodology, the strategies of employing it for making genetic perturbations, and explore its applications in inherited retinal dystrophies.

Automated summary

Precision genome engineering targeting patient-specific mutations is the future of personalized medicine, with ophthalmology leading the way as an accessible organ for therapy and monitoring. Increasing evidence supports the role of genomic editing in retinal dystrophies, with CRISPR-Cas9 system revolutionizing genome editing and drug discovery in this field.