Luk Vandenberghe is an inventor on Anc80 and other gene transfer technologies, for which he receives royalties.He is director of the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear and assistant professor of ophthalmology at Harvard Medical School.He completed his doctorate on the structural basis of AAVs in 2007 at the Catholic University of Leuven in Belgium, and later went on to become an associate professor at Harvard University in Cambridge, Massachusetts.His work on developing and improving numerous vector delivery systems has already proved innovative and useful for efficacy and safety in a number of therapeutic applications.He also is a founder, board member, and advisor to Odylia Therapeutics, a non-profit catalyzing translation for gene therapies within the challenging field of ultra-rare disorders.Dr. Vandenberghe has over 50 peer reviewed publications and more than a dozen licensed patents, mostly related to gene therapy methods, technologies, and applications.
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Grousbeck Gene Therapy Center
Researchers at Massachusetts Eye and Ear/Schepens Eye Research Institute have reconstructed an ancient virus that is highly effective at delivering gene therapies to the liver, muscle, and retina. "We believe our findings will teach us how complex biological structures, such as AAVs, are built," said senior study author Luk H. Vandenberghe, of Mass. Researchers have taken advantage of this natural property to develop viral vectors, or carriers, capable of shuttling therapeutic genes to the appropriate cells or tissues. The solution is to engineer new, benign AAVs that patients' immune system will not recognize, giving them time to insert the therapeutic genes into the target cells.
Now the researchers report restoring a much higher level of hearing—down to 25 decibels, the equivalent of a whisper—using an improved gene therapy vector developed at Massachusetts Eye and Ear. Eye and Ear; and Luk Vandenberghe, HMS assistant professor of ophthalmology at Mass. Eye and Ear who led the development of Anc80 in 2015. Since patients—and mice—with Usher 1C also have balance problems caused by hair-cell damage in the vestibular organs, the researchers also tested whether gene therapy restored balance.