Mingozzi, Federico

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Le Guiner, C., Servais, L., Montus, M., Larcher, T., Fraysse, B., Moullec, S., Allais, M., François, V., Dutilleul, M., Malerba, A., et al. (2017) · Nature Communications

Virus-based gene therapy researchMuscle Physiology and Disorders

Cited by 246

Unraveling the complex story of immune responses to AAV vectors trial after trial.

Vandamme, C., Adjali, O., & Mingozzi, F. (2017) · Human Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 234

Engineering adeno-associated viral vectors to evade innate immune and inflammatory responses

Chan, Y. K., Wang, S. K., Chu, C. J., Copland, D. A., Letizia, A. J., Costa Verdera, H., Chiang, J. J., Sethi, M., Wang, M. K., Neidermyer, W. J., et al. (2021) · Science Translational Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 215

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

Haurigot, V., Marcó, S., Ribera, A., Garcia, M., Ruzo, A., Villacampa, P., Ayuso, E., Añor, S., Andaluz, A., Pineda, M., et al. (2013) · Journal of Clinical Investigation

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

Cited by 215

Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia b

George, L. A., Ragni, M. V., Rasko, J. E. J., Raffini, L. J., Samelson-Jones, B. J., Ozelo, M., Hazbon, M., Runowski, A. R., Wellman, J. A., Wachtel, K., et al. (2020) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 213

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

Mingozzi, F., & High, K. A. (2017) · Annual Review of Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 206

Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction

Fitzpatrick, Z., Leborgne, C., Barbon, E., Masat, E., Ronzitti, G., van Wittenberghe, L., Vignaud, A., Collaud, F., Charles, S., Simon Sola, M., et al. (2018) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 192

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

Pien, G. C., Basner-Tschakarjan, E., Hui, D. J., Mentlik, A. N., Finn, J. D., Hasbrouck, N. C., Zhou, S., Murphy, S. L., Maus, M. V., Mingozzi, F., et al. (2009) · Journal of Clinical Investigation

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 192

Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells

Martino, A. T., Basner-Tschakarjan, E., Markusic, D. M., Finn, J. D., Hinderer, C., Zhou, S., Ostrov, D. A., Srivastava, A., Ertl, H. C. J., Terhorst, C., et al. (2013) · Blood

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 162

Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity

Paulk, N. K., Pekrun, K., Zhu, E., Nygaard, S., Li, B., Xu, J., Chu, K., Leborgne, C., Dane, A. P., Haft, A., et al. (2018) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 161

AAV liver expression of FIX-padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia b dogs and mice

Crudele, J. M., Finn, J. D., Siner, J. I., Martin, N. B., Niemeyer, G. P., Zhou, S., Mingozzi, F., Lothrop, C. D., & Arruda, V. R. (2015) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 153

Production, purification and characterization of adeno-associated vectors

Ayuso, E., Mingozzi, F., & Bosch, F. (2010) · Current Gene Therapy

Virus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects

Cited by 151

Exosome-associated AAV vector as a robust and convenient neuroscience tool

Hudry, E., Martin, C., Gandhi, S., György, B., Scheffer, D. I., Mu, D., Merkel, S. F., Mingozzi, F., Fitzpatrick, Z., Dimant, H., et al. (2016) · Gene Therapy

Virus-based gene therapy researchExtracellular vesicles in disease

Cited by 146

Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness

Amado, D., Mingozzi, F., Hui, D., Bennicelli, J. L., Wei, Z., Chen, Y., Bote, E., Grant, R. L., Golden, J. A., Narfstrom, K., et al. (2010) · Science Translational Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 140

Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue

Mingozzi, F., Chen, Y., Edmonson, S. C., Zhou, S., Thurlings, R. M., Tak, P. P., High, K. A., & Vervoordeldonk, M. J. (2013) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 138

Induction of antigen-specific CD4+ T-cell anergy and deletion by in vivo viral gene transfer

Dobrzynski, E., Mingozzi, F., Liu, Y.-L., Bendo, E., Cao, O., Wang, L., & Herzog, R. W. (2004) · Blood

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 135

Risk and Prevention of Anti-factor IX Formation in AAV-Mediated Gene Transfer in the Context of a Large Deletion of F9

Fields, P. A., Arruda, V. R., Armstrong, E., Chu, K., Mingozzi, F., Hagstrom, J. N., Herzog, R. W., & High, K. A. (2001) · Molecular Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 127

Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors

Meliani, A., Boisgerault, F., Fitzpatrick, Z., Marmier, S., Leborgne, C., Collaud, F., Simon Sola, M., Charles, S., Ronzitti, G., Vignaud, A., et al. (2017) · Blood Advances

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 127

Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction

Finn, J. D., Hui, D., Downey, H. D., Dunn, D., Pien, G. C., Mingozzi, F., Zhou, S., & High, K. A. (2010) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 115

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

Mack, D. L., Poulard, K., Goddard, M. A., Latournerie, V., Snyder, J. M., Grange, R. W., Elverman, M. R., Denard, J., Veron, P., Buscara, L., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 114

Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia b

Mingozzi, F., Chen, Y., Murphy, S. L., Edmonson, S. C., Tai, A., Price, S. D., Metzger, M. E., Zhou, S., Wright, J. F., Donahue, R. E., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 112

Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells

Dobrzynski, E., Fitzgerald, J. C., Cao, O., Mingozzi, F., Wang, L., & Herzog, R. W. (2006) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 105

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

Kuranda, K., Jean-Alphonse, P., Leborgne, C., Hardet, R., Collaud, F., Marmier, S., Costa Verdera, H., Ronzitti, G., Veron, P., & Mingozzi, F. (2018) · Journal of Clinical Investigation

Virus-based gene therapy researchImmunotherapy and Immune Responses

Cited by 104

Diverse IgG subclass responses to adeno‐associated virus infection and vector administration

Murphy, S. L., Li, H., Mingozzi, F., Sabatino, D. E., Hui, D. J., Edmonson, S. A., & High, K. A. (2009) · Journal of Medical Virology

Virus-based gene therapy researchMonoclonal and Polyclonal Antibodies Research

Cited by 101