Gene therapy for age-related macular degeneration

Journal article

Age-related macular degeneration is the most common cause of untreatable blindness in the world, and its prevalence is increasing. Current therapies for neovascular age-related macular degeneration aim to prevent growth of the abnormal retinal blood vessels that could leak and cause rapid visual loss in the later stages of the disease. This growth inhibition is achieved by regular injections into the eye of molecules that block the activity of vascular-endothelial growth factor (VEGF). These drugs are expensive, and the requirement for regular and indefinite intraocular injections puts a substantial strain on health-care resources. In The Lancet, Elizabeth Rakoczy and colleagues 3 present 1 year results from a phase 1 clinical trial to assess a single treatment, with gene therapy, as an alternative to regular injections for achieving long-term VEGF blockade.

Authors: MacLaren, RE

• Publication status: Published
• Peer review status: Peer reviewed
• Publisher: Elsevier
• Journal: Lancet
• Volume: 386
• Issue: 10011
• Pages: 2369-2370
• Publication date: 2015-09-30
• DOI: 10.1016/S0140-6736(15)00346-3
• EISSN: 1474-547X
• ISSN: 0140-6736