Establishing gene therapy treatments for Macular Degeneration in large-eyed animal models

Neovascular Age-related macular degeneration (nAMD) is a blinding disease that is very common in Sweden. This disease is caused by abnormal blood vessels growth and it is a therapeutic challenge and economical burden to the health care system. Today treatment for nAMD is based mostly on repeated intravitreal injections of anti-Vascular Endothelial Growth Factor (VEGF) drugs. These therapies require repeated injections that can cause vision threatening complications. Gene therapy can help overcome these problems by offering a sustainable and long lasting treatment with only one injection. The establishment of a large eyed animal model of nAMD will facilitate the transition of sustainable therapeutical gene therapy approaches into the clinic. 

Filippo Locri
Filippo Locri
Karolinska Institutet