Fischer MD, Michalakis S, Wilhelm B, et al. - Researchers conducted this nonrandomized controlled trial to evaluate safety and vision outcomes of supplemental gene therapy with adeno-associated virus (AAV) encoding CNGA3 (AAV8.CNGA3) in patients with CNGA3-linked achromatopsia. The sample consisted of 9 patients (mean [SD] age, 39.6 [11.9] years; age range, 24-59 years; 8 [89%] male) with confirmed CNGA3-linked achromatopsia. During the 12-month follow-up period, no substantial safety problems were observed. According to the findings, subretinal gene therapy with AAV8.CNGA3 was not correlated with substantial safety problems and was related to cone photoreceptor activation in adult patients, as demonstrated by visual acuity and contrast sensitivity gains.