A preliminary clinical trial using an experimental gene therapy to treat wet age-related macular degeneration (AMD) has recorded promising results.
Results from the trial, published in The Lancet, suggest that introducing a therapeutic gene into the eye using a viral vector is safe and may be effective in preserving the vision of wet AMD patients.
The study involved 19 men and women with advanced wet AMD who received increasing doses of viral particles containing the therapeutic gene.
Participants were monitored for adverse reactions before the next, higher dose was administered.
The researchers found that even at the highest dose, there were no serious side effects.
The gene therapy works by using a viral vector to deposit a gene into retinal cells. The gene prompts the cells to start producing a therapeutic protein, sFLT01, which binds to VEGF and stops it from stimulating leakage.