RT Journal Article
SR Electronic
T1 Gene therapy for neovascular age-related macular degeneration: rationale, clinical trials and future directions
JF British Journal of Ophthalmology
JO Br J Ophthalmol
FD BMJ Publishing Group Ltd.
SP bjophthalmol-2020-316195
DO 10.1136/bjophthalmol-2020-316195
A1 Guimaraes, Thales Antonio Cabral de
A1 Georgiou, Michalis
A1 Bainbridge, James W B
A1 Michaelides, Michel
YR 2020
UL http://bjo.bmj.com/content/early/2020/04/08/bjophthalmol-2020-316195.abstract
AB Age-related macular degeneration (AMD) is one of the leading causes of irreversible blindness in the developed world. Antivascular endothelial growth factor therapy has transformed the management and outcome of neovascular AMD (nAMD), although the need for repeated intravitreal injections—even lifelong—and the related complications, high drug costs, frequent clinic visits and repeated imaging have resulted in an enormous burden both to healthcare systems and patients. The application of gene therapy approaches for sustained delivery of a range of antiangiogenic proteins has the promise of helping to address these aforementioned challenges. A number of early phase clinical trials of gene therapy in nAMD have provided encouraging results, with many more ongoing or anticipated. There remain significant areas of controversy, including regarding the optimal treatment targets, routes of administration and potential safety concerns. In this review we aim to provide an update of the current status of gene therapy for nAMD and briefly discuss future prospects.