Researchers from the Casey Eye Institute at the Oregon Health & Science University are reporting the successful use of CRISPR gene-editing to restore vision for patients with a rare eye disease. Leber congenital amaurosis, or LCA, is an uncommon genetic form of vision impairment, but seven patients are seeing clearer following an experimental DNA modification study.
CRISPR has been used successfully to treat patients with sickle cell disease, but this is the first time it has been used in this type of application. Presenting the results at an International Symposium on Retinal Degeneration, Dr. Mark Pennesi explained how the team used a harmless virus to ferry the CRISPR gene editor and billions of the modified viruses into patients’ retinas. The procedure was done in one eye of each patient.
Although the procedure was not successful in all patients, researchers are not sure if this is due to too low of a dosage or because of something else. More research is needed, but for those whom it did work, they are reporting the ability to see shapes and colors for the first time in years.
LCA is a degenerative disease, slowly reducing vision over time, but participant Michael Kalberer celebrated being able to see the strobe lights change color at his cousin’s wedding. Another participant, Carlene Knight, reports that she can more safely navigate her home and workplace.