Luk H. Vandenberghe, Ph.D.

Harvard Medical School

Associate Professor of Ophthalmology
Associate Director, Ocular Genomics Institute
Member, Biological and Biomedical Sciences, Virology, Therapeutics Graduate Programs

Massachusetts Eye and Ear

Grousbeck Family Chair in Gene Therapy
Director, Grousbeck Gene Therapy Center
Associate 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.

For more information please download his CV [PDF].


    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: RPB Nelson Trust Awardee for Retinitis Pigmentosa

    2017: Ed Gollob Board of Directors' Award, Foundation Fighting Blindness

    2018: Outstanding New Investigator Award, American Society of Gene and Cell Therapy


    Research Interests

    • Therapeutic Gene Transfer: mechanistic studies, technology development, translational programs 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.



    42 (Google Scholar, as of October 2018)

    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. Maurer, AC, Pacouret, S, Cepeda Diaz, AK, Blake, J, Andres-Mateos, E, and Vandenberghe, LH (2018). The Assembly-Activating Protein Promotes Stability and Interactions between AAV's Viral Proteins to Nucleate Capsid Assembly. Cell reports 23: 1817-1830.
    2. Hudry, E, Andres-Mateos, E, Lerner, EP, Volak, A, Cohen, O, Hyman, BT, Maguire, CA, and Vandenberghe, LH (2018). Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65. Molecular therapy Methods & clinical development 10: 197-209.
    3.  Dudek, AM, Pillay, S, Puschnik, AS, Nagamine, CM, Cheng, F, Qiu, J, Carette, JE, and Vandenberghe, LH (2018). An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor. Journal of virology 92.
    4. 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, and Vandenberghe, LH (2017). AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations. Molecular therapy : the journal of the American Society of Gene Therapy 25: 1375-1386.
    5. Landegger, LD, Pan, B, Askew, C, Wassmer, SJ, Gluck, SD, Galvin, A, Taylor, R, Forge, A, Stankovic, KM, Holt, JR, and Vandenberghe, LH (2017). A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear. Nature biotechnology 35: 280-284.
    6. Zinn, E, Pacouret, S, Khaychuk, V, Turunen, HT, Carvalho, LS, Andres-Mateos, E, Shah, S, Shelke, R, Maurer, AC, Plovie, E, Xiao, R, and Vandenberghe, LH (2015). In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector. Cell reports 12: 1056-1068.


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

    Senior Research Technologist
    Julio Sanmiguel
    Allegra Fieldsend

    Senior Scientists
    Christopher Tipper, Ph.D.
    Carmen Unzu, Ph.D.

    Postdoctoral Research Fellows
    Amanda Dudek, Ph.D.
    Weiwei Wang, Ph.D.
    Xia Wang, Ph.D.

    PhD Candidates
    Michael Florea
    Anna C. Maurer
    Pauline Schmidt
    Eric Zinn

    Research Specialist
    Fotini Nicolaou, M.A.

    Animal Care Technician
    Jennifer Santos-Franceschini

    Research Assistants
    Reynette Estelien
    Ana Karla Cepeda Diaz
    Cheikh Diop
    Erin Merkel
    Urja Bhatt
    Rakesh Gurrala

    Yanhe Wen

    Members of the Gene Transfer Vector Core

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

    Research Assistants
    Trisha Barungi
    Haiyan Qiu, MSc
    Allison Cucalon