Article
Development of Methodology and Study Protocol: Safety and Efficacy of a Single Subretinal Injection of rAAV.hCNGA3 in Patients with CNGA3-Linked Achromatopsia Investigated in an Exploratory Dose-Escalation Trial.
Human gene therapy. Clinical development - 1 Sept 2018
Kahle Nadine A, Peters Tobias, Zobor Ditta, Kuehlewein Laura, Kohl Susanne, Zhour Ahmad, Werner Annette, Seitz Immanuel P, Sothilingam Vithiyanjali, Michalakis Stylianos, Biel Martin, Ueffing Marius, Zrenner Eberhart, Bartz-Schmidt Karl U, Fischer M Dominik, Wilhelm Barbara J C
Abstract excerpt
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, achromatopsia patients were treated with a single subretinal injection of rAAV.hCNGA3 to restore cone function. The focus of this trial was on the safety of the treatment. After surgery, patients were monitored in eight extensive...
Topics
- Adult
- Aged
- Color Vision Defects
- Cyclic Nucleotide-Gated Cation Channels
- Dependovirus
- Dose-Response Relationship, Drug
- Female
- Genetic Therapy