Precision medicine for the Inherited retinal diseases (IRDS) has brought a new perspective that has led to development of therapeutic tools to target patient-specific genetic mutations. The results of clinical trials that use in vivo gene editing to treat IRDs can prove to be essential for the future treatment for IRDs.
Inherited retinal diseases, or IRDS, are a group of diseases that can cause severe vision loss or even blindness. Each IRD is caused by at least one gene that is not working as it should. The disease onset ranges from congenital to late adulthood. The disease’s progress rate differs from individual to individual. This disease is rare and affects 1 in 3,000 individuals worldwide and greatly impacts the patients’ quality of life.
Genome editing technologies have evolved to enable precise genome editing with fewer side effects and risks, making precision genome editing possible.
More CRISPR-based treatment approaches are being tested in clinical trials for targeting IRDs, to treat any mutation that causes them to be responsive to this approach. Recently, precise genome editing agents are being developed to enable efficient and precise target gene correction, rather than gene disruption, in therapeutics applications including mouse models of IRDs.
Currently there are no effective treatments available, however over the past two decades major advances in gene therapy combined with improvement in technology and delivery techniques have birthed new hope for successful treatment of IRDs and other genetic disorders of vision.
Susie Suh, Elliot H. Choi, Aditya Raguram, et. al. Precision genome editing in the eye. Proceedings of the National Academy of Sciences, 2022 September 23.