Luk H. Vandenberghe, Ph.D.



Assistant Scientist, Schepens Eye Research Institute, Massachusetts Eye and Ear

Assistant Professor, Harvard Medical School

Director, Gene Transfer Vector Core (GTVC)

Associate Director, Ocular Genomics Institute (OGI)

Investigator, Berman-Gund Laboratory for Retinal Degenerations, Howe Laboratory

Affiliate Faculty, Harvard Stem Cell Institute


Research in the Vandenberghe Lab is focused on developing and translating genetic therapies to the clinic with an emphasis on blindness and other neurosensory disorders. We aim at defining, characterizing and overcoming hurdles on the path of gene therapies to clinical applications through

  • The study of the biology of, and host response to genetic therapy.
  • The development of enabling gene transfer technologies.
  • The translation of specific therapeutic programs.

More information here


  • Postdoctoral
    • 2010-2012 : Jean Bennett, MD PhD, Perelman School of Medicine, University of Pennsylvania, PA
    • 2007-2010 : James M. Wilson, MD PhD, Perelman School of Medicine, University of Pennsylvania, PA
  • Ph.D.: 'The Structural Basis of AAV as a gene therapeutic', Molecular Medicine, Katholieke Universiteit Leuven, Belgium
    • 2002-2007 : Zeger Debyser, MD PhD, Veerle Baekelandt, PhD (KULeuven) and James M. Wilson, MD PhD (UPenn)
  • Visiting Scholar
    • 2001-2002 : James Riley, PhD and Carl June, MD
  • B.Sc & M.Sc. : Biological Engineering in Cellular and Genetic Biotechnology (KULeuven, Belgium)

Contact Information

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20 Staniford Street

Boston MA 02114

617-573-6993 (direct)

617-573-6990 (lab)

617-912-0114 (fax)

Awards & Honors

  • Laureate of the Belgian Biology Olympiad, Diepenbeek, Leuven, Belgium 1994

  • Participant International Biology Olympiad, Varna, Bulgaria 1994

  • Travel Grant,  European Society of Gene Therapy ESGT 2005

  • American Society of Gene Therapy (ASGT) 2006, 2007

  • Late Breaking Abstract 2006 Annual meeting of the American Society of Gene Therapy (ASGT)

  • 1st Prize Basic Science Session, 2011, Sylvan M. Cohen Annual Retreat, Institute on Aging, University of Pennsylvania


Selected Publications

Click here for a PubMed list of abstracts formatted by BioMed Central

  1. Gao, G., Alvira, M. R., Somanathan, S., Lu, Y., Vandenberghe, L. H., Rux, J. J., Calcedo, R., Sanmiguel, J., Abbas, Z. and Wilson, J. M. (2003). "Adeno-associated viruses undergo substantial evolution in primates during natural infections." Proc Natl Acad Sci U S A 100(10): 6081-6.

  2. Parry, R. V., Rumbley, C. A., Vandenberghe, L. H., June, C. H. and Riley, J. L. (2003). "CD28 and inducible costimulatory protein Src homology 2 binding domains show distinct regulation of phosphatidylinositol 3-kinase, Bcl-xL, and IL-2 expression in primary human CD4 T lymphocytes." J Immunol 171(1): 166-74.

  3. Simmons, G., Rennekamp, A. J., Chai, N., Vandenberghe, L. H., Riley, J. L. and Bates, P. (2003). "Folate receptor alpha and caveolae are not required for Ebola virus glycoprotein-mediated viral infection." J Virol 77(24): 13433-8.

  4. Vandenberghe, L. H.*, Gao, G.*, Alvira, M. R., Lu, Y., Calcedo, R., Zhou, X. and Wilson, J. M. (2004). "Clades of Adeno-associated viruses are widely disseminated in human tissues." J Virol 78(12): 6381-8.

  5. Vandenberghe, L. H.*, Wang, L.*, Somanathan, S., Zhi, Y., Figueredo, J., Calcedo, R., Sanmiguel, J., Desai, R. A., Chen, C. S., Johnston, J., Grant, R. L., Gao, G. and Wilson, J. M. (2006). "Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid." Nat Med 12(8): 967-71.

  6. Allocca, M., Mussolino, C., Garcia-Hoyos, M., Sanges, D., Iodice, C., Petrillo, M., Vandenberghe, L. H., Wilson, J. M., Marigo, V., Surace, E. M. and Auricchio, A. (2007). "Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors." J Virol 81(20): 11372-80.

  7. Taymans, J. M., Vandenberghe, L. H., Haute, C. V., Thiry, I., Deroose, C. M., Mortelmans, L., Wilson, J. M., Debyser, Z. and Baekelandt, V. (2007). "Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain." Hum Gene Ther 18(3): 195-206.

  8. Cearley, C. N., Vandenberghe, L. H., Parente, M. K., Carnish, E. R., Wilson, J. M. and Wolfe, J. H. (2008). "Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain." Mol Ther. 16(10): 1710-8.

  9. Breous, E., Somanathan, S., Vandenberghe, L. H. and Wilson, J. M. (2009). "Hepatic regulatory T cells and Kupffer cells are crucial mediators of systemic T cell tolerance to antigens targeting murine liver." Hepatology 50(2): 612-21.

  10. Vandenberghe, L. H.*, Calcedo, R.*, Gao, G., Lin, J. and Wilson, J. M. (2009). "Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses." J Infect Dis 199(3): 381-90.

  11. Calcedo, R., Vandenberghe, L. H., Roy, S., Somanathan, S., Wang, L. and Wilson, J. M. (2009). "Host immune responses to chronic adenovirus infections in human and nonhuman primates." J Virol 83(6): 2623-31.

  12. Lin, J., Calcedo, R., Vandenberghe, L. H., Bell, P., Somanathan, S. and Wilson, J. M. (2009). "A new genetic vaccine platform based on an adeno-associated virus isolated from a rhesus macaque." J Virol In Press

  13. Mays, L. E., Vandenberghe, L. H., Xiao, R., Bell, P., Nam, H. J., Agbandje-McKenna, M. and Wilson, J. M. (2009). "Adeno-associated virus capsid structure drives CD4-dependent CD8+ T cell response to vector encoded proteins." J Immunol 182(10): 6051-60.

  14. Vandenberghe, L. H.*, Roy, S.*, Kryazhimskiy, S., Grant, R., Calcedo, R., Yuan, X., Keough, M., Sandhu, A., Wang, Q., Medina-Jaszek, C. A., Plotkin, J. B. and Wilson, J. M. (2009). "Isolation and characterization of adenoviruses persistently shed from the gastrointestinal tract of non-human primates." PLoS Pathog 5(7): e1000503.

  15. Vandenberghe, L. H., Breous, E., Nam, H. J., Gao, G., Xiao, R., Sandhu, A., Johnston, J., Debyser, Z., Agbandje-McKenna, M. and Wilson, J. M. (2009). "Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints." Gene Ther Dec;16(12):1416-28.

  16. Wang, L., Calcedo, R., Wang, H., Bell, P., Grant, R., Vandenberghe, L. H., Sanmiguel, J., Morizono, H., Batshaw, M. L. and Wilson, J. W. (2010). "The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques. Mol Ther Jan;18(1):126-34.

  17. Lock M., Alvira M., Vandenberghe L. H., Samanta A., Toelen J., Debyser Z. and Wilson JM. Rapid, Simple and Versatile Manufacturing of Recombinant Adeno-Associated Virus Vectors at Scale. Hum Gene Ther 2010 Jul 22. [Epub ahead of print]

  18. Vandenberghe L. H., Xiao, R, Lock, M., Lin, J, Korn, M, and Wilson, JM. Efficient serotype-dependent release of functional vector into the culture medium during AAV manufacturing. Hum Gene Ther 2010 Jul 22. [Epub ahead of print]

  19. Kassim S, Li H, Vandenberghe L. H., Hinderer C, Bell P, Marchadier D, Wilson A, Cromley D, Yu H, Wilson J. M., Rader DJ. Gene therapy in a humanized mouse model of familial hypercholesterolemia leads to marked regression of atherosclerosis (2011). Plos One 5(10); e13424

  20. Bell C.B., Vandenberghe L.H., Bell P, Limberis M.P., Gao G.P., Van Vliet K, Agbandje-McKenna M and Wilson J.M. The receptor for AAV9 and its modification to improve in vivo lung gene therapy. Journal of Clinical Investigation. June 2011

  21. Vandenberghe LH, Bell P, Maguire AM, Cearley C.N., Xiao R., Calcedo R., Wang L., Castle M.J., Maguire A., Grant R., Wolfe J.H., Wilson JM, Bennett J. Dosage thresholds of AAV2 and AAV8 photoreceptor targeted gene therapies in a non-human primate model. Sci Transl Med. 2011 Jun 22;3(88)

  22. Aleman TS, Cideciyan AV, Aguirre GK, Huang WC, Mullins CL, Roman AJ, Sumaroka A, Olivares MB, Tsai FF, Schwartz SB, Vandenberghe LH, Limberis MP, Stone EM, Bell P, Wilson JM and Jacobson SG. Human CRB1-associated retinal degeneration: comparison with the rd8 Crb1-mutant mouse model. IOVS (accepted for publication)

  23. Mason JB, Vandenberghe LH, Xiao R., Wilson JM and Richardson DW. Transduction Efficiency of Recombinant Adeno-Associated Viral Vectors is Differentially Influenced by Vector Serotype and Tissue Composition in Equine Synovial Joint Tissues. American Journal of Veterinary Research (accepted for publication)

* shared first authorship

Review Publications

  1. Gao, G., Vandenberghe, L. H. and Wilson, J. M. (2005). "New recombinant serotypes of AAV vectors." Curr Gene Ther 5(3): 285-97.

  2. Vandenberghe, L. H. and Wilson, J. M. (2007). "AAV as an immunogen." Curr Gene Ther 7(5): 325-33.

  3. Vandenberghe, L. H., Wilson, J. M. and Gao, G. (2009). "Tailoring the AAV vector capsid for gene therapy." Gene Ther 16(3): 311-9.

  4. Vandenberghe LH and Auricchio A. Novel adeno-associated viral vectors for retinal gene therapy. Gene Therapy

Published & Pending Patents

    1. WO/2005/033321 - Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor - PCT/US2004/028817, EP1668143, EP2292779, EP2292780, EP2345731, EP2298926., 2005.

    2. WO/2006/110689 - Method of increasing the function of an AAV vector - PCT/US2006/013375, EP1866422, EP2357010, 2006.

    3. WO/2007/127264 - Scalable production method for AAV - PCT/US2007/010055, EP2018421. USA , 2007.

    4. WO/2008/027084 - Modified AAV vectors having reduced capsid immunogenicity and use thereof - PCT/US2007/010056 EP2016174, 2008.

    5. WO/2009/073103 - Simian subfamily B adenoviruses SADV-28,27,-29,-32,-33, and -35, and uses thereof - PCT/US2008/013065, SG161579, EP2220242, 2009.

    6. WO/2009/073104 - Simian E adenoviruses SADV-39, -25. 2, -26, -30, -37, and -38 - PCT/US2008/013066, SG161586, EP2220241, 2009.

    7. WO/2009/105084 - Simian subfamily C adenoviruses SADV-40, -31, and -34 and uses thereof - PCT/US2008/013067, SG161584, EP2220217, 2009.

    8. WO/2009/134681 - Adeno associated viral vectors for targeted transduction of retinal pigment epithetial cells - PCT/US2009/041606, 2009.

    9. WO/2009/136977 - Simian adenoviruses SADV-36,-42.1, -42.2, and -44, and uses thereof - PCT/US2009/001344, EP2250255, EP2325298, 2009.

    10. WO/2010/051367 - Simian adenovirus SADV-43, -45,-48,-49, AND -50 and uses thereof - PCT/US2009/062548, EP2350269, 2010.

    11. WO/2010/138675 - Simian adenovirus 41 and uses thereof - PCT/US2010/036332, 2010.

    12. WO/2011/038187 - Controlled adeno-associated virus (AAV) diversification and libraries prepared therefrom - PCT/US2010/050135, 2010.

Vandenberghe Laboratory Opportunities

Research in this laboratory focuses on developing gene therapy for blinding and hearing disorders. The aim is to further the technology for therapeutic gene transfer making use of viral vectors, to develop new animal models and to translate our findings and technology into the clinic.

Current opportunities in the Vandenberghe laboratory exist at one level: