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The Making of Medicine

An Important Step Toward Preventing Vision Loss

Our Jayakrishna Ambati, MD, and Shoo-bin Wang, PhD, have discovered a previously unknown contributor to harmful blood vessel growth in the eye that helps us better understand blinding macular degeneration and other causes of vision loss. That understanding could lead to new and better treatments for proliferative diabetic retinopathy, neovascular age-related macular degeneration and other sight-stealing diseases.

The discovery identifies a key protein that regulates a substance called “vascular endothelial growth factor-A,” or VEGF. VEGF is important for the formation of blood vessels in the healthy eye, but too much of it causes the vessels to overgrow into tangled knots that contribute to vision loss.

There are treatments available that target VEGF to prevent vessel overgrowth, but the effectiveness of the treatments can dwindle over time. So we need new options, and that's where Dr. Ambati and Professor Wang's discovery appears most promising.

By targeting the protein they identified, the researchers were able to reduce VEGF levels in lab mice significantly. This prevented blood vessel overgrowth, and it did so without unwanted side effects. For example, there were no harmful effects on the retina, the light-sensing portion inside the eye.

The researchers are hopeful that their approach could, eventually, offer a new option for preventing and treating blood-vessel overgrowth. More research will be needed, but they say the approach could offer benefits such as avoiding the drug resistance that plagues existing treatments that target VEGF.

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