AAV-mediated multiple gene therapy combining VEGFA-targeting miR-agshRNAs and PEDF for the suppression of choroidal neovascularization

Molecular Therapy — Nucleic Acids 2026 Open access

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Authors: Bjørn K. Fabian-Jessing, Anne Louise Askou, Thomas Stax Jakobsen, Rikke L. Adsersen, Anna Bøgh Lindholm, Anne Kirstine Køllner Bjerre, Sidsel Alsing, Bek T, Lars Aagaard, Thomas J. Corydon
DOI: 10.1016/j.omtn.2026.102833
Cited by: 3
References: 62

Abstract

suppression and strong PEDF expression were observed. Notably, laser-induced choroidal neovascularization (CNV) was significantly reduced in the therapeutic groups, with the multi-targeting vector achieving the highest level of CNV suppression. Collectively, our data demonstrated robust anti-angiogenic effects of multiple gene therapies, suggesting a "one-and-done" AAV-based delivery of cross-species anti-VEGFA RNAi therapeutics together with PEDF as a valuable tool for the management of neovascular AMD (nAMD) and other complex neovascular ocular diseases.

Fields of Study & Subjects

Retinal Development and Disorders Retinal Diseases and Treatments RNA Interference and Gene Delivery

Publication details

Journal / source
Molecular Therapy — Nucleic Acids
Publisher
Elsevier BV
Volume
37
Issue
1
Pages
102833–102833
Publication date
10 January 2026
Language
EN