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
- Hsu, Y, Bhattarai, S, Thompson, JM, Mahoney, A, Thomas, J, Mayer, SK et al.. Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model. Mol Ther Nucleic Acids. 2023;31 :164-181. doi: 10.1016/j.omtn.2022.12.007. PubMed PMID:36700052 PubMed Central PMC9841241.
- Florea, M, Nicolaou, F, Pacouret, S, Zinn, EM, Sanmiguel, J, Andres-Mateos, E et al.. High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography. Mol Ther Methods Clin Dev. 2023;28 :146-159. doi: 10.1016/j.omtm.2022.12.009. PubMed PMID:36654797 PubMed Central PMC9823220.
- Zinn, E, Unzu, C, Schmit, PF, Turunen, HT, Zabaleta, N, Sanmiguel, J et al.. Ancestral library identifies conserved reprogrammable liver motif on AAV capsid. Cell Rep Med. 2022;3 (11):100803. doi: 10.1016/j.xcrm.2022.100803. PubMed PMID:36327973 PubMed Central PMC9729830.
- Lu, YC, Tsai, YH, Chan, YH, Hu, CJ, Huang, CY, Xiao, R et al.. Gene therapy with a synthetic adeno-associated viral vector improves audiovestibular phenotypes in Pjvk-mutant mice. JCI Insight. 2022;7 (20):. doi: 10.1172/jci.insight.152941. PubMed PMID:36278489 PubMed Central PMC9714786.
- Zabaleta, N, Bhatt, U, Hérate, C, Maisonnasse, P, Sanmiguel, J, Diop, C et al.. Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques. Mol Ther. 2022;30 (9):2952-2967. doi: 10.1016/j.ymthe.2022.05.007. PubMed PMID:35546782 PubMed Central PMC9088091.
- Maciorowski, D, Diop, C, Bhatt, U, Estelien, R, Li, D, Chauhan, R et al.. Immunogenicity of an AAV-Based COVID-19 Vaccine in Murine Models of Obesity and Aging. Viruses. 2022;14 (4):. doi: 10.3390/v14040820. PubMed PMID:35458550 PubMed Central PMC9026898.
- Naninck, T, Kahlaoui, N, Lemaitre, J, Maisonnasse, P, De Mori, A, Pascal, Q et al.. Computed tomography and [18F]-FDG PET imaging provide additional readouts for COVID-19 pathogenesis and therapies evaluation in non-human primates. iScience. 2022;25 (4):104101. doi: 10.1016/j.isci.2022.104101. PubMed PMID:35313622 PubMed Central PMC8926429.
- Andres-Mateos, E, Landegger, LD, Unzu, C, Phillips, J, Lin, BM, Dewyer, NA et al.. Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate. Nat Commun. 2022;13 (1):1359. doi: 10.1038/s41467-022-28969-3. PubMed PMID:35292639 PubMed Central PMC8924271.
- Grotz, S, Schäfer, J, Wunderlich, KA, Ellederova, Z, Auch, H, Bähr, A et al.. Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes. EMBO Mol Med. 2022;14 (4):e14817. doi: 10.15252/emmm.202114817. PubMed PMID:35254721 PubMed Central PMC8988205.
- Wang, J, Zhao, L, Gu, X, Xue, Y, Wang, S, Xiao, R et al.. Efficient Delivery of Adeno-Associated Virus into Inner Ear In Vivo Through Trans-Stapes Route in Adult Guinea Pig. Hum Gene Ther. 2022;33 (13-14):719-728. doi: 10.1089/hum.2021.236. PubMed PMID:35156857 .
- Zolotukhin, S, Vandenberghe, LH. AAV capsid design: A Goldilocks challenge. Trends Mol Med. 2022;28 (3):183-193. doi: 10.1016/j.molmed.2022.01.003. PubMed PMID:35093287 .
- Katz, MG, Hadas, Y, Bailey, RA, Fazal, S, Vincek, A, Madjarova, SJ et al.. Efficient cardiac gene transfer and early-onset expression of a synthetic adeno-associated viral vector, Anc80L65, after intramyocardial administration. J Thorac Cardiovasc Surg. 2022;164 (6):e429-e443. doi: 10.1016/j.jtcvs.2021.05.050. PubMed PMID:34985414 PubMed Central PMC8733395.
- Chekuri, A, Logan, EM, Krauson, AJ, Salani, M, Ackerman, S, Kirchner, EG et al.. Selective retinal ganglion cell loss and optic neuropathy in a humanized mouse model of familial dysautonomia. Hum Mol Genet. 2022;31 (11):1776-1787. doi: 10.1093/hmg/ddab359. PubMed PMID:34908112 PubMed Central PMC9169455.
- Ilyinskii, PO, Michaud, AM, Rizzo, GL, Roy, CJ, Leung, SS, Elkins, SL et al.. ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia. Mol Ther Methods Clin Dev. 2021;22 :279-292. doi: 10.1016/j.omtm.2021.06.015. PubMed PMID:34485611 PubMed Central PMC8399083.
- Zabaleta, N, Dai, W, Bhatt, U, Hérate, C, Maisonnasse, P, Chichester, JA et al.. An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates. Cell Host Microbe. 2021;29 (9):1437-1453.e8. doi: 10.1016/j.chom.2021.08.002. PubMed PMID:34428428 PubMed Central PMC8346325.
- Annabi, N, Baker, M, Boettiger, A, Chakraborty, D, Chen, Y, Corbett, KS et al.. Voices of biotech research. Nat Biotechnol. 2021;39 (3):281-286. doi: 10.1038/s41587-021-00847-1. PubMed PMID:33692517 .
- Zabaleta, N, Dai, W, Bhatt, U, Chichester, JA, Sanmiguel, J, Estelien, R et al.. Immunogenicity of an AAV-based, room-temperature stable, single dose COVID-19 vaccine in mice and non-human primates. bioRxiv. 2021; :. doi: 10.1101/2021.01.05.422952. PubMed PMID:33442684 PubMed Central PMC7805441.
- Jacob, A, Brun, L, Jiménez Gil, P, Ménard, L, Bouzelha, M, Broucque, F et al.. Homologous Recombination Offers Advantages over Transposition-Based Systems to Generate Recombinant Baculovirus for Adeno-Associated Viral Vector Production. Biotechnol J. 2021;16 (1):e2000014. doi: 10.1002/biot.202000014. PubMed PMID:33067902 .
- Greenwald, SH, Brown, EE, Scandura, MJ, Hennessey, E, Farmer, R, Pawlyk, BS et al.. Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration. Mol Ther Methods Clin Dev. 2020;18 :582-594. doi: 10.1016/j.omtm.2020.07.003. PubMed PMID:32775493 PubMed Central PMC7397406.
- Hu, CJ, Lu, YC, Tsai, YH, Cheng, HY, Takeda, H, Huang, CY et al.. Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65. Mol Ther Methods Clin Dev. 2020;18 :493-500. doi: 10.1016/j.omtm.2020.06.019. PubMed PMID:32775487 PubMed Central PMC7390729.
Vandenberghe Lab Team
Luk H. Vandenberghe, PhD
Director (on leave)
Nerea Zabaleta, PhD
Investigator
Anil Chekuri, PhD
Investigator
Urja Bhatt, MS
Lead Research Lab Tech
Julio Sanmiguel
Lead Research Lab Tech
Reynette Estelien
Lead Research Lab Tech
Michael Florea
PhD Candidate
Jiten Doshi, PhD
Postdoctoral Research Fellow
Huachun Liu, PhD
Postdoctoral Research Fellow
Aruna Kumar Chelluboyina, PhD
Postdoctoral Research Fellow
Paige Osborn
Program Coordinator
Grousbeck Gene Therapy Center Alumni
2023
Jackson Lau
Maxwell Miller
2022
Dan Li
Eric Zinn, PhD
Aisling Ryan
Maya Kim
Ainsley MacLachlan, Student Intern
Cheikh Diop
Achla Gandhi, Student Intern
Abigail Jackson, Student
2021
Ruchi Chauhan, PhD
Aoyue Mao, Student Intern
Abigail Sheridan
Weiwei Wang, PhD
Caitlyn Webb
Sofia Castore, Student Intern
Dawid Maciorowski, Student Intern
Wenlong Dai, PhD
2020
Pauline Schmit, PhD
Jonathan Sands, Student Intern
Haiyan Qiu
Polina Freitas, PhD
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
Research Lab Tech I