RNAi Treatment for Macular Degeneration


Experimental treatment of age-related macular degeneration (AMD), a debilitating eye disease, with RNAi (RNA interference). AMD is triggered by a protein called VEGF that promotes blood vessel growth. In patients with macular degeneration, too much of this protein leads to the sprouting of excess blood vessels behind the retina. The blood vessels leak, clouding and often entirely destroying vision. The new RNAi drugs shut down genes that produce VEGF and allow it to make the leaky vessels. The first clinical trial, launched in the fall of 2004, shows promising results. Two months after being injected with the drug, a quarter of the patients had significantly clearer vision, and the other patients' vision had at least stabilized. If subsequent trials prove they are effective, RNAi drugs for this condition could hit the market by 2009.

Credit: BSIP / Science Source

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