Subretinal gene therapy not associated with substantial safety concerns in achromatopsia

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Gene therapy applying an adeno-associated viral vector encoding CNGA3 appears safe for the treatment of patients with achromatopsia, according to a study published in JAMA Ophthalmology.

In this nonrandomized controlled trial, 9 patients with CNGA3-linked achromatopsia received gene therapy with adeno-associated virus (AAV) encoding CNGA3 (AAV8.CNGA3) by subretinal injection.

During the 12-month follow-up period, no substantial safety problems were observed and all treated eyes demonstrated improvement in cone function including a change in visual acuity and contrast sensitivity.


Fischer MD, Michalakis S, Wilhelm B, et al. Safety and vision outcomes of subretinal gene therapy targeting cone photoreceptors in achromatopsia. JAMA Ophthalmol. 2020;138(6):643-651. doi:10.1001/jamaophthalmol.2020.1032

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