Luk H. Vandenberghe, Ph.D.

Harvard Medical School

Assistant Professor of Ophthalmology
Associate Director, Ocular Genomics Institute
Member, PhD Program in Biological and Biomedical Sciences,

Massachusetts Eye and Ear

Director, Grousbeck Gene Therapy Center
Assistant Scientist

Research Summary

Center/Research Area Affiliations


Through the study and development of technologies that overcome current translational hurdles, Dr. Vandenberghe is helping to position gene therapy as a broadly applicable clinical modality in ophthalmology. Previously, he developed a novel adeno-associated viral vector (AAV) platform, and in translational studies, he characterized those for safety and efficacy parameters in large animal models. His current research focuses on gene therapy for neurosensory disorders, especially retinal blindness. He is on the editorial board of Molecular Therapy and is an active member of the American Society of Cell and Gene Therapy, for which he chairs the educational committee. In addition, he co-founded GenSight Biologics, a biotech entity that pursues innovative ocular gene therapy strategies.


    MS.c., Ph.D., Biological Engineering in Cellular and Genetic Biotechnology, Katholieke Universiteit Leuven, Belgium (2008)

    Postgraduate Training

    Postdoctoral fellowship (James M. Wilson, MD PhD), Perelman School of Medicine, University of Pennsylvania (2007-2010)
    Postdoctoral fellowship (Jean Bennett, MD PhD), Perelman School of Medicine, University of Pennsylvania (2010-2012)


    2016: Mass. Eye and Ear Curing Kids Award
    2011: 1st Prize Basic Science Session, Sylvan M. Cohen Annual Retreat, Institute on Aging, University of Pennsylvania
    2006: Late Breaking Abstract, American Society of Gene Therapy Annual MEeting
    2005: Travel Grant, European Society of Gene Therapy


    Research Interests

    • Gene therapy for inherited disorders, with an emphasis on blindness and other neurosensory disorders

    Biology of Adeno-Associated Virus (AAV)

    Dr. Vandenberghe is interested in studying the biology of AAV transduction, including binding, entry, post-entry, replication, viral assembly, and release, as well as AAV epidemiology and immunology.

    Viral Evolution

    Dr. Vandenberghe studies the evolution of viruses currently in use for gene transfer applications. He hypothesizes that the study of the natural history of the viruses on which vectors are based highlight underlying biology relevant to their application as vectors and fundamental findings in how viruses assemble, package DNA, and transduce a particular cell target.

    Delivering Large Therapeutic Expression Cassettes to the Retina

    Current vector systems are limited in terms of how much genetic cargo they can effectively shuttle. Dr. Vandenberghe is interested in developing technologies to overcome this limitation.

    Immunology of Retinal Gene Transfer

    The eye is generally seen as an immune-privileged organ due to passive and active means of tolerance. Many gene therapy approaches rely on the delivery of a protein or vector system that is foreign to the host and may elicit an immune-response. Dr. Vandenberghe's laboratory studies and develops assays to monitor these processes in an effort to maximize the safety of the therapeutic strategy.

    Genome Editing Therapy

    While many ideas use genetic information to treat disease are built on adding, augmenting or silencing genes to the host cell, tissue or organ, Dr. Vandenberghe is also exploring ways that novel methods to somatically alter cellular genomic information could be used therapeutically in blindness.

    Vector Development

    Delivery of a gene to a cell is cornerstone of genetic therapy. Its efficiency, safety, specificity, longevity and many of the other parameters essential for a treatment are determined by the vector the gene is shuttled into the cell. Dr. Vandenberghe's laboratory continuously pursues the optimization of current vectors and the discovery and generation of new vector technologies.

    Translational Gene Therapy Programs

    Dr. Vandenberghe's laboratory is dedicated to using existing technologies in preclinical and clinical programs for indications with an unmet clinical needs.



    39 (Google Scholar, as of October 2017)

    Selected Publications

    Dr. Vandenberghe has published more than 60 peer-reviewed articles. Below is a list of selected publications. View his publications on PubMed, Google Scholar.

    1. Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC. Corrigendum: Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Sci Rep. 2017 May 22;7:46827.
    2. Pacouret S, Bouzelha M, Shelke R, Andres-Mateos E, Xiao R, Maurer A, Mevel M, Turunen H, Barungi T, Penaud-Budloo M, Broucque F, Blouin V, Moullier P, Ayuso E, Vandenberghe LH. AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations. Mol Ther. 2017 Jun 7;25(6):1375-1386.
    3. Suzuki J, Hashimoto K, Xiao R, Vandenberghe LH, Liberman MC. Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction. Sci Rep. 2017 Apr 3;7:45524.
    4. Wassmer SJ, Carvalho LS, György B, Vandenberghe LH, Maguire CA. Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection. Sci Rep. 2017 Mar 31;7:45329.
    5. Pan B, Askew C, Galvin A, Heman-Ackah S, Asai Y, Indzhykulian AA, Jodelka FM, Hastings ML, Lentz JJ, Vandenberghe LH, Holt JR, Géléoc GS. Gene therapy restores auditory and vestibular function in a mouse model of Usher syndrome type 1c. Nat Biotechnol. 2017 Mar;35(3):264-272.


    Current Members of Dr. Vandenberghe’s Name’s Laboratory

    Project Manager and Associate Director of Quality Assurance
    Eva Andres-Mateos, Ph.D.

    Associate Director, Gene Transfer Vector Core
    Ru Xiao, M.D., M.S.

    Senior Research Technician
    Julio Sanmiguel

    Haiyan Qiu, MSc

    Senior Scientific Associates
    Khan Maola, Ph.D.
    Carmen Unzu, Ph.D.

    Postdoctoral Research Fellows
    Mohammadsharif Tabebordbar, Ph.D.
    Sarah Wasserman, Ph.D.

    PhD Candidates
    Amanda Dudek
    Anna C. Maurer
    Pauline Schmidt

    Graduate Students
    Simon Pacouret
    Eric Zinn, B.A.

    Research Specialist
    Fotini Nicolaou, M.A.

    Research Assistants
    Trisha Barungi
    Jessica Blake
    Yang Lin, M.D., M.S.
    Jennifer Santos-Franceschini