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Gene Therapy: First It Cured Blindness. Next Stop: Coronavirus

There’s a not-so-new genetic technology – in fact, ophthalmologists already use it to reverse an incurable type of blindness called Leber congenital amaurosis (LCA) – that may well prove the SARS-CoV-2 magic bullet the world so desperately seeks. It leverages a unique vaccine strategy, called adeno-associated viral (AAV) vector, using an innocuous virus scientists have been studying for 25 years, to deliver a powerful knock-out punch to COVID-19 before it can establish a foothold in the human body.

Three researchers, two from Harvard and one from UPenn, have teamed up to develop the vaccine, which they call AAVCOVID, and they’re making progress in record time. The lead scientist, Luk H. Vandenberghe, PhD, is director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear, a vision research lab.

That shouldn’t be surprising. Vision labs are often ahead of the pack when it comes to developing breakthrough medications. Molecular geneticist Jean Bennett, MD, PhD, with her husband, ophthalmologist Albert Maguire, MD lead the development of the first gene therapy cure for LCA, Luxturna, in 2017. LCA is an inherited, early onset form of childhood blindness that affects up to 100,000 Americans.

AAVCOVID works by exposing genetic sequences of the SARS-CoV-2 Spike antigen, administered through a single injection, to the body’s immune system. The body then quickly learns to recognize and defend against it. The Spike antigen is simply a protein, by itself harmless, taken from the coronavirus. But it’s the delivery system, the AAV, that makes this vaccine unique. It’s the only one in the race already approved by the FDA for clinical use, so we know it’s safe, effective, and quickly adaptable for use in a wide variety of indications.

Luk H. Vandenberghe, PhD, director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear and Associate Professor of Ophthalmology at Harvard Medical School.

Mason Freeman, MD, director and founder of the MGH Translational Research Center

James M. Wilson, MD, PhD, Perelman School of Medicine at the University of Pennsylvania.

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