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The Grousbeck Gene Therapy Center is an academic research center devoted to the broadening of therapeutic gene transfer in modern medicine. Our focus is on internal and collaborative preclinical and clinical research studies, technology development, and enabling resources for the community.

VECTOR CORE

The Gene Transfer Vector Core (GTVC) is to provide high quality research-grade viral vector preparations to support basic research and preclinical gene therapy studies for academia and industry. GTVC has its primary expertise in adeno-associated viral vector (AAV) preparation, qualification, and application.

Vandenberghe lab

The Vandenberghe Lab is the Grousbeck Gene Therapy core laboratory focused on developing and translating genetic therapies to the clinic with an emphasis on blindness and other neurosensory disorders. Researchers in the Vandenberghe lab work at the intersection of medicine, virology, engineering, immunology, synthetic and systems biology to innovate genetic medicine.

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  • PRINCIPAL INVESTIGATOR

    Luk H. Vandenberghe, PhD

    Luk H. Vandenberghe, PhD, is an Assistant Professor at Harvard Medical School and  Associate Member of the Broad Institute of Harvard and MIT in Boston, MA, USA. He directs the Grousbeck Gene Therapy Center at Massachusetts Eye and Ear Infirmary in Boston, USA, a part of the the Ocular Genomics Institute, a bench to bedside research program to study, diagnose, and develop treatments for diseases of the eye.

    He received a degree in cellular and genetic engineering from the University of Leuven, Belgium. His previous work led to the discovery of novel AAV serotypes such as AAV9, novel insights into AAV structure-function, and vector immunobiology. His laboratory aims to gain a deeper understanding of mechanisms of gene transfer, to develop technologies to overcome hurdles to gene therapy clinical applications, and to translate specific gene therapy programs in vision, hearing, and other fields. Recent studies leverage structural and evolutionary information on AAV as a starting point for the rational design of synthetic viral vector systems, a first generation of which is referred to as AncAAVs which are now progressing to the clinic for a number of indications. Dr. Vandenberghe previously co-founded GenSight Biologics and Akouos. 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 publication and more than a dozen licensed patents, mostly related to gene therapy methods, technologies, and applications.

    PubMed

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