A study conducted jointly by the Massachusetts Eye and Ear Infirmary and Oregon Health & Science University has shown that approximately 79% of participants with hereditary retinal degeneration experienced symptom improvement after undergoing experimental CRISPR gene editing therapy. The research paper was published in the latest issue of the New England Journal of Medicine.
EDIT-101 is an experimental gene editing therapy utilizing CRISPR technology. The trial assessed the safety and efficacy of EDIT-101, which targets mutations in the centrosomal protein 290 (CEP290) gene responsible for expressing proteins crucial for vision.
The trial included 14 participants, comprising 12 adults and two children, who suffered from Leber congenital amaurosis caused by mutations in the CEP290 gene. Through surgery, the patients received a single injection of the CRISPR/Cas9 genome editing drug EDIT-101.
No severe treatment- or surgery-related adverse events were reported during the trial, and there were no dose-limiting toxicities. Regarding efficacy, researchers evaluated four indicators: best-corrected visual acuity (BCVA), full-field stimulus testing (FST), visual function navigation, and vision-related quality of life.
Signs of improvement were observed in at least one test indicator in 11 participants, with six participants showing improvement in two or more test results, and four participants experiencing clinically significant improvements in BCVA. In FST testing, six participants showed improvement in cone-mediated vision, with five participants demonstrating improvement in at least one of the other three indicators.
(Original Title: Clinical Trial Shows Gene Editing Therapy Can Improve Vision in Patients with Hereditary Blindness)
