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L. Vandenberghe · SciRah

Researcher profile · Semantic Scholar

L. Vandenberghe

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L. Vandenberghe — researcher profile on SciRah

h-index: 58
Works: 182
Citations: 13K

Most-cited papers

In vivo gene editing in dystrophic mouse muscle and muscle stem cells
Mohammadsharif Tabebordbar, Kexian Zhu, Jason K. W. Cheng, W. Chew +11 more
2015Science997 citations Open access
Semantic Scholar
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality
E. Hudry, L. Vandenberghe
2019Neuron321 citations Open access
Semantic Scholar
In silico reconstruction of the viral evolutionary lineage yields a potent gene therapy vector
E. Zinn, Simon Pacouret, Vadim Khaychuk, H. Turunen +8 more
2015Cell Reports300 citations Open access
Semantic Scholar
A synthetic AAV vector enables safe and efficient gene transfer to the mammalian inner ear
L. D. Landegger, Bifeng Pan, Charles Askew, Sarah J. Wassmer +7 more
2017Nature Biotechnology296 citations Open access
Semantic Scholar
Gene Therapy Restores Auditory and Vestibular Function in a Mouse Model of Usher Syndrome Type 1c
Bifeng Pan, Charles Askew, Alice Galvin, S. Heman-Ackah +8 more
2017Nature Biotechnology271 citations Open access
Semantic Scholar
Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter
T. Cronin, L. Vandenberghe, Péter Hantz, Josephine Jüttner +8 more
2014EMBO Molecular Medicine176 citations Open access
Semantic Scholar
Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction
Jun Suzuki, Ken Hashimoto, Ru Xiao, L. Vandenberghe +1 more
2017Scientific Reports166 citations Open access
Semantic Scholar
Efficient in vivo genome editing prevents hypertrophic cardiomyopathy in mice
D. Reichart, Gregory A. Newby, H. Wakimoto, M. Lun +14 more
2023Nature Medicine164 citations Open access
Semantic Scholar
Exosome-associated AAV2 vector mediates robust gene delivery into the murine retina upon intravitreal injection
Sarah J. Wassmer, Livia S. Carvalho, Bence György, L. Vandenberghe +1 more
2017Scientific Reports154 citations Open access
Semantic Scholar
Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids
Michael J. Castle, H. Turunen, L. Vandenberghe, J. H. Wolfe
2016Methods in molecular biology135 citations Open access
Semantic Scholar
Genome editing abrogates angiogenesis in vivo
Xiong-Gao Huang, Guo-Hong Zhou, Wenyi Wu, Y. Duan +7 more
2017Nature Communications134 citations Open access
Semantic Scholar
In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery.
A. Garanto, D. Chung, Lonneke Duijkers, Julio C Corral-Serrano +5 more
2016Human Molecular Genetics134 citations Open access
Semantic Scholar
GPR108 Is a Highly Conserved AAV Entry Factor.
A. Dudek, N. Zabaleta, E. Zinn, S. Pillay +7 more
2019Molecular Therapy118 citations Open access
Semantic Scholar
Adeno-associated Virus: Fit to serve
E. Zinn, L. Vandenberghe
2014Current Opinion in Virology115 citations Open access
Semantic Scholar
An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor
A. Dudek, S. Pillay, A. Puschnik, C. Nagamine +4 more
2018Journal of Virology98 citations Open access
Semantic Scholar
Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting
Livia S. Carvalho, H. Turunen, Sarah J. Wassmer, Maria V. Luna-Velez +3 more
2017Frontiers in Neuroscience77 citations Open access
Semantic Scholar
Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction
Yong Tao, Mingqian Huang, Yi-Lai Shu, A. Ruprecht +7 more
2017Human Gene Therapy76 citations Open access
Semantic Scholar
An AAV-based, room-temperature-stable, single-dose COVID-19 vaccine provides durable immunogenicity and protection in non-human primates
N. Zabaleta, Wen-Long Dai, Urja Bhatt, Cécile Herate +50 more
2021Cell Host and Microbe74 citations Open access
Semantic Scholar
Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65
E. Hudry, Eva Andrés-Mateos, Eli P Lerner, Adrienn Volak +4 more
2018Molecular Therapy: Methods & Clinical Development72 citations Open access
Semantic Scholar
FGF21 underlies a hormetic response to metabolic stress in methylmalonic acidemia.
I. Manoli, J. Sysol, M. Epping, Li-Na Li +27 more
2018JCI Insight66 citations Open access
Semantic Scholar
AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations.
Simon Pacouret, Mohammed Bouzelha, Rajani R. J. Shelke, Eva Andrés-Mateos +11 more
2017Molecular Therapy62 citations Open access
Semantic Scholar
High-efficiency purification of divergent AAV serotypes using AAVX affinity chromatography
Michael Florea, Fotini Nicolaou, Simon Pacouret, E. Zinn +5 more
2022Molecular Therapy: Methods & Clinical Development61 citations Open access
Semantic Scholar
Quantitative and Digital Droplet-Based AAV Genome Titration
Julio Sanmiguel, G. Gao, L. Vandenberghe
2019Methods in molecular biology58 citations Open access
Semantic Scholar
The Assembly-Activating Protein Promotes Stability and Interactions between AAV’s Viral Proteins to Nucleate Capsid Assembly
Anna C. Maurer, Simon Pacouret, Ana Karla Cepeda Diaz, J. Blake +2 more
2018Cell Reports58 citations Open access
Semantic Scholar
Synthetic Adeno-Associated Viral Vector Efficiently Targets Mouse and Nonhuman Primate Retina In Vivo
Livia S. Carvalho, Ru Xiao, Sarah J. Wassmer, Aliete E. Langsdorf +7 more
2018Human Gene Therapy57 citations Open access
Semantic Scholar
Efficient Gene Transfer to Kidney Mesenchymal Cells Using a Synthetic Adeno-Associated Viral Vector.
Yoichiro Ikeda, Zhao Sun, Xiao Ru, L. Vandenberghe +1 more
2018Journal of the American Society of Nephrology55 citations Open access
Semantic Scholar
Choice of vector and surgical approach enables efficient cochlear gene transfer in nonhuman primate
Eva Andrés-Mateos, L. D. Landegger, Carmen Unzu, Jeananne Phillips +11 more
2022Nature Communications51 citations Open access
Semantic Scholar
Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality.
E. Hudry, L. Vandenberghe
2019Neuron51 citations Open access
Semantic Scholar
Single stranded adeno-associated virus achieves efficient gene transfer to anterior segment in the mouse eye
Li Wang, Ru Xiao, Eva Andrés-Mateos, L. Vandenberghe
2017PLoS ONE46 citations Open access
Semantic Scholar
Hair Cell Transduction Efficiency of Single- and Dual-AAV Serotypes in Adult Murine Cochleae
Ryotaro Omichi, Hidekane Yoshimura, S. B. Shibata, L. Vandenberghe +1 more
2020Molecular Therapy: Methods & Clinical Development42 citations Open access
Semantic Scholar
Rational design of a compact CRISPR-Cas9 activator for AAV-mediated delivery
Suhani Vora, Jenny Cheng, Xiao Ru, Nathan J. VanDusen +5 more
201840 citations Open access
Semantic Scholar
The structure of AAVrh32.33, a Novel Gene Delivery Vector
Kyle Mikals, H. Nam, K. van Vliet, L. Vandenberghe +4 more
2014Journal of Structural Biology38 citations Open access
Semantic Scholar
Corrigendum: Cochlear gene therapy with ancestral AAV in adult mice: complete transduction of inner hair cells without cochlear dysfunction
Jun Suzuki, Ken Hashimoto, Ru Xiao, L. Vandenberghe +1 more
2017Scientific Reports37 citations Open access
Semantic Scholar
Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes
Sophia Grotz, Jessica Schäfer, K. Wunderlich, Z. Ellederová +36 more
2022EMBO Molecular Medicine36 citations Open access
Semantic Scholar
Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy
Suriyasri Subramanian, Anna C. Maurer, C. Bator, A. Makhov +5 more
2019Human Gene Therapy35 citations Open access
Semantic Scholar
ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia
P. Ilyinskii, Alicia M. Michaud, G. Rizzo, C. Roy +12 more
2021Molecular Therapy: Methods & Clinical Development32 citations Open access
Semantic Scholar
Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries
Pauline F. Schmit, Simon Pacouret, E. Zinn, E. Telford +10 more
2019Molecular Therapy: Methods & Clinical Development31 citations Open access
Semantic Scholar
Ancestral library identifies conserved reprogrammable liver motif on AAV capsid
E. Zinn, Carmen Unzu, Pauline F. Schmit, H. Turunen +16 more
2022Cell Reports Medicine28 citations Open access
Semantic Scholar
Gene therapy with a synthetic adeno-associated viral vector improves audiovestibular phenotypes in Pjvk-mutant mice
Ying-Chang Lu, Yi-Hsiu Tsai, Y. Chan, Chin-Ju Hu +6 more
2022JCI Insight25 citations Open access
Semantic Scholar
Efficient in Utero Gene Transfer to the Mammalian Inner Ears by the Synthetic Adeno-Associated Viral Vector Anc80L65
Chin-Ju Hu, Ying-Chang Lu, Yi-Hsiu Tsai, Haw-Yuan Cheng +8 more
2020Molecular Therapy: Methods & Clinical Development25 citations Open access
Semantic Scholar
Gene Therapy Preserves Retinal Structure and Function in a Mouse Model of NMNAT1-Associated Retinal Degeneration
Scott H. Greenwald, Emily E. Brown, Michael J. Scandura, Erin Hennessey +5 more
2020Molecular Therapy: Methods & Clinical Development20 citations Open access
Semantic Scholar
MCOLN1 gene therapy corrects neurologic dysfunction in the mouse model of mucolipidosis IV.
Samantha De Rosa, M. Salani, Sierra Smith, M. Sangster +7 more
2021Human Molecular Genetics18 citations Open access
Semantic Scholar
Subretinal gene therapy delays vision loss in a Bardet-Biedl Syndrome type 10 mouse model
Ying Hsu, Sajag Bhattarai, Jacob M. Thompson, Angela Mahoney +9 more
2022Molecular Therapy: Nucleic Acids17 citations Open access
Semantic Scholar
Novel engineered, membrane-localized variants of vascular endothelial growth factor (VEGF) protect retinal ganglion cells: a proof-of-concept study
Jun-Hui Shen, Ru Xiao, Jeffrey Bair, Fang Wang +4 more
2018Cell Death and Disease17 citations Open access
Semantic Scholar
Immunogenicity of an AAV-based, room-temperature stable, single dose COVID-19 vaccine in mouse and non-human primates
N. Zabaleta, Wen-Long Dai, Urja Bhatt, Jessica A. Chichester +36 more
2021bioRxiv14 citations Open access
Semantic Scholar
Residues on Adeno-associated Virus Capsid Lumen Dictate Interactions and Compatibility with the Assembly-Activating Protein
Anna C. Maurer, Ana Karla Cepeda Diaz, L. Vandenberghe
2019Journal of Virology14 citations Open access
Semantic Scholar
Perspectives on Best Practices for Gene Therapy Programs
Thomas R. Cheever, Dale Berkley, S. Braun, Robert H. Brown +24 more
2015Human Gene Therapy14 citations Open access
Semantic Scholar
Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.
Marina O'Reilly, H. Federoff, Y. Fong, D. Kohn +31 more
2014Human Gene Therapy14 citations Open access
Semantic Scholar
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques
N. Zabaleta, Urja Bhatt, Cécile Herate, P. Maisonnasse +19 more
2022Molecular Therapy12 citations Open access
Semantic Scholar
Homologous Recombination Offers Advantages over Transposition‐Based Systems to Generate Recombinant Baculovirus for Adeno‐Associated Viral Vector Production
Aurélien Jacob, Laurie Brun, Paloma Jiménez Gil, Lucie Ménard +10 more
2020Biotechnology Journal12 citations Open access
Semantic Scholar

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