Achromatopsia is an autosomal recessively inherited congenital defect characterized by a
lack of cone photoreceptor function, leading to severely impaired vision. In this clinical study …

Purpose: The aim of this clinical interventional study (NCT02610582) was to test safety
aspects of AAV8 based supplementation gene therapy in patients with CNGA3 …

Importance Achromatopsia linked to variations in theCNGA3gene is associated with day
blindness, poor visual acuity, photophobia, and involuntary eye movements owing to lack of …

Aims To determine long-term safety and efficacy outcomes of a subretinal gene therapy for
CNGA3-associated achromatopsia. We present data from an open-label, nonrandomised …

Achromatopsia causes severely reduced visual acuity, photoaversion, and inability to
discern colors due to cone photoreceptor dysfunction. In 2010, we reported on day …

Purpose: The purpose of this study was to clinically characterize patients with CNGA3-linked
achromatopsia (CNGA3-ACHM) in preparation of a gene therapy trial. Methods: Thirty-six …

Achromatopsia is a rare autosomal recessive disorder which shows color blindness,
severely impaired visual acuity, and extreme sensitivity to bright light. Mutations in the alpha …

Achromatopsia is an inherited retinal disorder of cone photoreceptors characterized by
markedly reduced visual acuity, extreme light sensitivity, and absence of color …

Applied Genetic Technologies Corporation (AGTC) is developing a recombinant adeno-
associated virus (rAAV) vector expressing the human CNGA3 gene designated AGTC-402 …

Achromatopsia is a rare congenital cause of vision loss due to isolated cone photoreceptor
dysfunction. The most common underlying genetic mutations are autosomal recessive …