Vandenberghe Lab at Grousbeck Gene Therapy Center
About the Lab
Broadening Therapeutic Gene Transfer Through Mechanistic Studies, Technology Development, and Translation to the Clinic
Gene therapy is a conceptually naïve approach that has proven difficult in execution. It is the bringing together of a molecular intervention to a disease target by the delivery of a gene. Over 40 years in the making, clinical studies over the past years have illustrated the transformative potential of gene therapy in inherited blindness, hemophilia, and motor neuron disease.
Research in the Vandenberghe laboratory is focused on broadening applications of gene therapy generally. Research is centered on three overlapping areas of focus: Mechanism, Technology, and Translation.
Our Research
Publications
- Goldstein, JM, Tabebordbar, M, Zhu, K, Wang, LD, Messemer, KA, Peacker, B et al.. In Situ Modification of Tissue Stem and Progenitor Cell Genomes. Cell Rep. 2019;27 (4):1254-1264.e7. doi: 10.1016/j.celrep.2019.03.105. PubMed PMID:31018138 .
- Vandenberghe, LH. AAV Engineering Identifies a Species Barrier That Highlights a Portal to the Brain. Mol. Ther. 2019;27 (5):901-903. doi: 10.1016/j.ymthe.2019.04.006. PubMed PMID:31000409 PubMed Central PMC6520466.
- Hudry, E, Vandenberghe, LH. Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality. Neuron. 2019;102 (1):263. doi: 10.1016/j.neuron.2019.03.020. PubMed PMID:30946822 .
- Hudry, E, Vandenberghe, LH. Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality. Neuron. 2019;101 (5):839-862. doi: 10.1016/j.neuron.2019.02.017. PubMed PMID:30844402 .
- Sanmiguel, J, Gao, G, Vandenberghe, LH. Quantitative and Digital Droplet-Based AAV Genome Titration. Methods Mol. Biol. 2019;1950 :51-83. doi: 10.1007/978-1-4939-9139-6_4. PubMed PMID:30783968 .
- Maurer, AC, Cepeda Diaz, AK, Vandenberghe, LH. Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly-Activating Protein. J. Virol. 2019;93 (7):. doi: 10.1128/JVI.02013-18. PubMed PMID:30651367 PubMed Central PMC6430561.
- Manoli, I, Sysol, JR, Epping, MW, Li, L, Wang, C, Sloan, JL et al.. FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia. JCI Insight. 2018;3 (23):. doi: 10.1172/jci.insight.124351. PubMed PMID:30518688 PubMed Central PMC6328030.
- Shen, J, Xiao, R, Bair, J, Wang, F, Vandenberghe, LH, Dartt, D et al.. Novel engineered, membrane-localized variants of vascular endothelial growth factor (VEGF) protect retinal ganglion cells: a proof-of-concept study. Cell Death Dis. 2018;9 (10):1018. doi: 10.1038/s41419-018-1049-0. PubMed PMID:30282966 PubMed Central PMC6170416.
- Hudry, E, Andres-Mateos, E, Lerner, EP, Volak, A, Cohen, O, Hyman, BT et al.. Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65. Mol Ther Methods Clin Dev. 2018;10 :197-209. doi: 10.1016/j.omtm.2018.07.006. PubMed PMID:30109242 PubMed Central PMC6083902.
- Miller, JW, Vandenberghe, LH. Breaking and Sealing Barriers in Retinal Gene Therapy. Mol. Ther. 2018;26 (9):2081-2082. doi: 10.1016/j.ymthe.2018.08.003. PubMed PMID:30107998 PubMed Central PMC6127874.
- Ikeda, Y, Sun, Z, Ru, X, Vandenberghe, LH, Humphreys, BD. Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector. J. Am. Soc. Nephrol. 2018;29 (9):2287-2297. doi: 10.1681/ASN.2018040426. PubMed PMID:29976586 PubMed Central PMC6115653.
- Maurer, AC, Pacouret, S, Cepeda Diaz, AK, Blake, J, Andres-Mateos, E, Vandenberghe, LH et al.. The Assembly-Activating Protein Promotes Stability and Interactions between AAV's Viral Proteins to Nucleate Capsid Assembly. Cell Rep. 2018;23 (6):1817-1830. doi: 10.1016/j.celrep.2018.04.026. PubMed PMID:29742436 PubMed Central PMC5983388.
- Dudek, AM, Pillay, S, Puschnik, AS, Nagamine, CM, Cheng, F, Qiu, J et al.. An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor. J. Virol. 2018;92 (7):. doi: 10.1128/JVI.02213-17. PubMed PMID:29343568 PubMed Central PMC5972900.
- Carvalho, LS, Xiao, R, Wassmer, SJ, Langsdorf, A, Zinn, E, Pacouret, S et al.. Synthetic Adeno-Associated Viral Vector Efficiently Targets Mouse and Nonhuman Primate Retina In Vivo. Hum. Gene Ther. 2018;29 (7):771-784. doi: 10.1089/hum.2017.154. PubMed PMID:29325457 PubMed Central PMC6066192.
- Tao, Y, Huang, M, Shu, Y, Ruprecht, A, Wang, H, Tang, Y et al.. Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction. Hum. Gene Ther. 2018;29 (4):492-506. doi: 10.1089/hum.2017.120. PubMed PMID:29130354 PubMed Central PMC5909114.
- Carvalho, LS, Turunen, HT, Wassmer, SJ, Luna-Velez, MV, Xiao, R, Bennett, J et al.. Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting. Front Neurosci. 2017;11 :503. doi: 10.3389/fnins.2017.00503. PubMed PMID:28943836 PubMed Central PMC5596095.
- Wang, L, Xiao, R, Andres-Mateos, E, Vandenberghe, LH. Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye. PLoS ONE. 2017;12 (8):e0182473. doi: 10.1371/journal.pone.0182473. PubMed PMID:28763501 PubMed Central PMC5538712.
- 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;7 :46827. doi: 10.1038/srep46827. PubMed PMID:28530226 PubMed Central PMC5439157.
- Pacouret, S, Bouzelha, M, Shelke, R, Andres-Mateos, E, Xiao, R, Maurer, A et al.. AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations. Mol. Ther. 2017;25 (6):1375-1386. doi: 10.1016/j.ymthe.2017.04.001. PubMed PMID:28427840 PubMed Central PMC5475248.
- 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;7 :45524. doi: 10.1038/srep45524. PubMed PMID:28367981 PubMed Central PMC5377419.
Vandenberghe Lab Team
Luk H. Vandenberghe, PhD
Director
Christopher Tipper, Ph.D.
Director of Discovery Research
Eric Zinn, BA
Systems Biology Graduate Student
Pauline Schmit
PhD Candidate, BBS Program, Harvard
Weiwei Wang, Ph.D.
Postdoctoral Research Fellow
Xia Wang, Ph.D.
Postdoctoral Research Fellow
Michael Florea
PhD Candidate
Julio Sanmiguel
Senior Research Technologist
Allegra Fieldsend
Senior Research Technologist
Reynette Estelien
Research Assistant
Cheikh Diop
Research Assistant
Yanhe (Crane) Wen
Bioinformatician (MS)
Erin Merkel
Research Assistant
Urja Bhatt
Research Assistant
Nerea Zabaleta Lasarte PhD
Postdoctoral Research Fellow
Polina Freitas, Ph.D
Laboratory Manager
Aisling Ryan
Program Coordinator
Maya Kim
Research Assistant
Anil Chekuri, PhD
Postdoctoral Research Fellow
Grousbeck Gene Therapy Center Alumni
2019
Boqiao Song, Undergraduate Co-op student (2019), currently at Northeastern University, Boston, MA
Rakesh Gurrala, Research Assistant II (2018-2019), currently at Tulane University School of Medicine
Carmen Unzu, Senior Scientific Associate & Associate Director of Translational Studies (2017-2019), currently Principal Scientist at Apic Bio, Cambridge, MA
Jennifer Santos-Franceschini, Research Assistant II (2016-2019), currently at Akouos, Boston, MA
Ana Karla Cepeda Diaz, Undergraduate Student Research Assistant at Harvard University (2016-2019), currently graduate student at Harvard Medical School
Anna Maurer, Harvard Medical School Graduate Student (2015-2018), currently Postdoctoral Fellow at UC Berkeley
2018
Amanda Dudek, Harvard Medical School Graduate Student (2015-2018), currently Postdoctoral Fellow at Stanford University, Stanford, CA
Simran Tanna, Undergraduate Co-op student (2018), currently at Northeastern University, Boston, MA
Shibangi Saha, Undergraduate Co-op student (2018), currently at Northeastern University, Boston, MA
Wajd Alsharif, Undergraduate Co-op student (2018), currently at Northeastern University, Boston, MA
Eva Andres-Mateos, PhD, Associate Director of Project Management (2014-2018), currently Associate Director of Translational Sciences at Akouos, Boston, MA
Fotini Nicolaou, Research Specialist (2017-2018), currently at Eikonizo Therapeutics, Cambridge, MA
Alex Mellott, Summer High School Intern (2018), currently at Westwood High School
Henry Ren, Undergraduate Co-op student (2018), currently at Northeastern University, Boston, MA
Dante Del Priore, Undergraduate Co-op student (2018), currently at Northeastern University, Boston, MA
Jessica Blake, Research Assistant (2016-2018), currently a Research Biologist at the U.S. Department of Veterans Affairs, San Diego, CA
Sarah Wassmer, Postdoctoral Fellow (2015-2018), currently Venture Associate II at Centre for Commercialization of Regenerative Medicine (CCRM), Ottawa, Canada
2017
Lizzie Telford, Undergraduate Summer Intern (2017), currently at Georgetown University, Washington D.C.
Simon Pacouret, Graduate Student (2014-2017), currently at Université de Nantes, Nantes, France
2016
Li Wang, MD, PhD, Postdoctoral Fellow (2015-2016), currently at Eye & ENT Hospital, Shanghai Medical College, Fudan University, Key Laboratory of Myopia of State Health Ministry and Key Laboratory of Visual Impairment and Restoration of Shanghai, Shanghai, China
Rajani Shelke, Scientific Associate (2012-2016), currently a Scientist at MassBiologics, Fall River, MA
Heikki Turunen, Postdoctoral Fellow (2012-2016), currently a Research Scientist at Applied Genetic Technologies Corporation
Suzanne De Bruijn, Visiting Research Scholar (2016), currently a Ph.D. student at Radboud University Medical Center, Nijmegen, Netherlands
Positions
We are always interested in talented and passionate researchers who work well in a team and compelled by gene therapy and virology. See below to learn more about our open positions and how to apply.
Graduate Student Positions
Postdoctoral Positions
Scientific Staff Positions