AUG 21, 2014 08:45 AM PDT

From Genetics to Genetic Medicine: Gene Therapy for vision restoration as a case study

  • Assistant Professor, Harvard Medical School, Assistant Scientist, Schepens Eye Research Institute, Massachusetts Eye and Ear
      Assistant Professor, Harvard Medical School
      <br />Assistant Scientist, Schepens Eye Research Institute, Massachusetts Eye and Ear
      <br />Director, Gene Transfer Vector Core (GTVC)
      <br />Associate Director, Ocular Genomics Institute (OGI)
      <br />Investigator, Berman-Gund Laboratory for Retinal Degenerations, Howe Laboratory
      <br />Affiliate Faculty, Harvard Stem Cell Institute
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      <br />Luk H. Vandenberghe, PhD, trained as a biochemical engineer through his training at the University in Leuven, Belgium where he later also obtained his doctoral degree in Medical Sciences. Most of his graduate and postdoctoral work was performed at the University of Pennsylvania in Philadelphia in the leading gene therapy laboratories of Carl June, James Wilson, and Jean Bennett. In 2012, Dr. Vandenberghe set up a lab at the Schepens Eye Research Institute and Mass Eye and Ear in Boston where he is Assistant Professor at Harvard Medical School. Currently he is Director of the Grousbeck Gene Therapy Center and Associate Director of the Ocular Genomics Institute, both primarily focused at bringing gene therapies for vision restoration to the clinic.
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      <br />In previous work Dr. Vandenberghe has co-discovered, developed, and characterized novel serotypes of the adeno-associated virus (including AAV8,9), several of which are now in wide use at preclinical and clinical experimental stages for a host of target tissues and indications. His work has elucidated key insights into the host response of AAV in humans in the context of pre-existing immunity of this virus endemic in humans. Dr. Vandenberghe is a co-founder of a biotechnology company called GenSight Biologics (Paris, France) and named inventor in over a dozen published and pending patents of gene therapy related technologies.
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      <br />The Vandenberghe lab at Harvard studies gene therapy approaches for neurosensory disorders with a focus on clinical programs for retinal dystrophies. Its broader ambition is to make gene therapy a widely applicable clinical modality in ophthalmology and beyond. Toward this goal his program builds innovative vector technologies to overcome current translational hurdles.&nbsp; His research brings together molecular virology and immunology, synthetic biology, engineering, and medicine in attempt to understand and improve the challenges of the therapeutic potential of in vivo gene transfer.
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    Gene therapy for two forms of inherited retinal degeneration have met promising safety and efficacy endpoints in early stage clinical trials. These approaches made use of a replication defective virus or vector based on the adeno-associated virus type 2 (AAV2), a small non enveloped ssDNA virus endemic in human population. AAV2 targets the retinal pigment epithelium (RPE) at high efficiency in small and large animal models, which is the primary target in these studies. Retinal disease however has a range of etiologies and cell types at the basis of its pathogenesis, and a broader set of tools to deliver therapeutic genes to the retina is required. Here, we describe our past and current efforts to develop novel vectors and therapeutic strategies to unlock genetic treatment for retinal blinding disorders.

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