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Using a “one-and-done” gene therapy to treat wet AMD

Six patients with wet age-related macular degeneration have gone for six months without the need for regular injections

28 Oct 2019 by Selina Powell

US researchers have highlighted the potential of using gene therapy to treat wet age-related macular degeneration (AMD) at the annual meeting of the American Academy of Ophthalmology (12–15 October, San Francisco).

Dr Szilard Kiss, from Weill Cornell Medical College, shared his belief that a gene therapy for wet AMD could be available within the next three to five years.

Dr Kiss and his team developed a vector that inserts genetic material producing a molecule similar to anti-VEGF medicine aflibercept within the cells of the eye.

Once inserted, the DNA sequence begins making aflibercept protein.

“Instead of taking a vile of aflibercept and injecting it into the eye, your eye makes the aflibercept,” Dr Kiss highlighted.

“The goal is a potentially one-and-done treatment. You may need a booster once in a while, but this gene therapy could theoretically last a lifetime,” he added.

Image credit: Pixabay/PublicDomainPictures


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