Vandenberghe, Luk H.

AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations

Pacouret, S., Bouzelha, M., Shelke, R., Andres-Mateos, E., Xiao, R., Maurer, A., Mevel, M., Turunen, H., Barungi, T., Penaud-Budloo, M., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 69

AAV capsid design: A Goldilocks challenge

Zolotukhin, S., & Vandenberghe, L. H. (2022) · Trends in Molecular Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 64

Synthetic Adeno-Associated Viral Vector Efficiently Targets Mouse and Nonhuman Primate Retina In Vivo

Carvalho, L. S., Xiao, R., Wassmer, S. J., Langsdorf, A., Zinn, E., Pacouret, S., Shah, S., Comander, J. I., Kim, L. A., Lim, L., et al. (2018) · Human Gene Therapy

Virus-based gene therapy researchRetinal Development and Disorders

Cited by 63

Impact of Preexisting Vector Immunity on the Efficacy of Adeno-Associated Virus-Based HIV-1 Gag Vaccines

Lin, J., Calcedo, R., Vandenberghe, L. H., Figueredo, J. M., & Wilson, J. M. (2008) · Human Gene Therapy

Virus-based gene therapy researchHIV Research and Treatment

Cited by 53

Rational design of a compact CRISPR-Cas9 activator for AAV-mediated delivery

Vora, S., Cheng, J., Xiao, R., VanDusen, N. J., Quintino, L., Pu, W. T., Vandenberghe, L. H., Chavez, A., & Church, G. (2018) · bioRxiv (Cold Spring Harbor Laboratory)

CRISPR and Genetic EngineeringVirus-based gene therapy research

Cited by 42

Filling Adeno-Associated Virus Capsids: Estimating Success by Cryo-Electron Microscopy

Subramanian, S., Maurer, A. C., Bator, C. M., Makhov, A. M., Conway, J. F., Turner, K. B., Marden, J. H., Vandenberghe, L. H., & Hafenstein, S. L. (2019) · Human Gene Therapy

Virus-based gene therapy researchAdvanced Electron Microscopy Techniques and Applications

Cited by 40

ImmTOR nanoparticles enhance AAV transgene expression after initial and repeat dosing in a mouse model of methylmalonic acidemia

Ilyinskii, P. O., Michaud, A. M., Rizzo, G. L., Roy, C. J., Leung, S. S., Elkins, S. L., Capela, T., Chowdhury, A., Li, L., Chandler, R. J., et al. (2021) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 39

Correction: AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina

Vandenberghe, L. H., Bell, P., Maguire, A. M., Xiao, R., Hopkins, T. B., Grant, R., Bennett, J., & Wilson, J. M. (2013) · PLoS ONE

Retinal Development and DisordersVirus-based gene therapy research

Cited by 16