Luk H. Vandenberghe, Ph.D.

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.

H-index

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

Bioinformaticist
Yanhe Wen

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

Research Assistants
Trisha Barungi
Haiyan Qiu, MSc
Allison Cucalon