Inherited retinal dystrophies (IRDs) are a leading cause of visual impairment in the developing world. These conditions present an\nirreversible dysfunction or loss of neural retinal cells, which significantly impacts quality of life. Due to the anatomical accessibility\nand immunoprivileged status of the eye, ophthalmological research has been at the forefront of innovative and advanced gene- and\ncell-based therapies, both of which represent great potential as therapeutic treatments for IRD patients. However, due to a genetic\nand clinical heterogeneity, certain IRDs are not candidates for these approaches. New advances in the field of genome editing using\nClustered Regularly Interspaced Short Palindromic Repeats (CRISPR) and CRISPR-associated protein (Cas) have provided an\naccurate and efficient way to edit the human genome and represent an appealing alternative for treating IRDs. We provide a\nbrief update on current gene augmentation therapies for retinal dystrophies. Furthermore, we discuss recent advances in the\nfield of genome editing and stem cell technologies, which together enable precise and personalized therapies for patients. Lastly,\nwe highlight current technological limitations and barriers that need to be overcome before this technology can become a viable\ntreatment option for patients.
Loading....