Mingozzi, Federico

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 2,163

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Maguire, A. M., Simonelli, F., Pierce, E. A., Pugh, E. N., Mingozzi, F., Bennicelli, J., Banfi, S., Marshall, K. A., Testa, F., Surace, E. M., et al. (2008) · New England Journal of Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 2,145

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Nathwani, A. C., Tuddenham, E. G. D., Rangarajan, S., Rosales, C., McIntosh, J., Linch, D. C., Chowdary, P., Riddell, A., Pie, A. J., Harrington, C., et al. (2011) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 1,864

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Nathwani, A. C., Reiss, U. M., Tuddenham, E. G. D., Rosales, C., Chowdary, P., McIntosh, J., Della Peruta, M., Lheriteau, E., Patel, N., Raj, D., et al. (2014) · New England Journal of Medicine

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 1,273

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

Mingozzi, F., & High, K. A. (2013) · Blood

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 955

Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges

Mingozzi, F., & High, K. A. (2011) · Nature Reviews Genetics

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 893

Emerging issues in AAV-mediated in vivo gene therapy

Colella, P., Ronzitti, G., & Mingozzi, F. (2018) · Molecular Therapy - Methods & Clinical Development

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 881

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

Maguire, A. M., High, K. A., Auricchio, A., Wright, J. F., Pierce, E. A., Testa, F., Mingozzi, F., Bennicelli, J. L., Ying, G.-S., Rossi, S., et al. (2009) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 865

Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans

Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. J., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., et al. (2007) · Nature Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 748

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

Costa Verdera, H., Kuranda, K., & Mingozzi, F. (2020) · Molecular Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 641

Induction of Immune Tolerance to Coagulation Factor IX Antigen by In Vivo Hepatic Gene Transfer

Mingozzi, F., Liu, Y.-L., Dobrzynski, E., Kaufhold, A., Liu, J. H., Wang, Y., Arruda, V. R., High, K. A., & Herzog, R. W. (2003) · Journal of Clinical Investigation

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 466

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

Bennett, J., Wellman, J., Marshall, K. A., McCague, S., Ashtari, M., DiStefano-Pappas, J., Elci, O. U., Chung, D. C., Sun, J., Wright, J. F., et al. (2016) · The Lancet

Retinal Development and DisordersVirus-based gene therapy research

Cited by 461

AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness

Bennett, J., Ashtari, M., Wellman, J., Marshall, K. A., Cyckowski, L. L., Chung, D. C., McCague, S., Pierce, E. A., Chen, Y., Bennicelli, J. L., et al. (2012) · Science Translational Medicine

Retinal Development and DisordersVirus-based gene therapy research

Cited by 395

Human Immune Responses to Adeno-Associated Virus (AAV) Vectors

Ronzitti, G., Gross, D.-A., & Mingozzi, F. (2020) · Frontiers in Immunology

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 357

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

Mingozzi, F., Anguela, X. M., Pavani, G., Chen, Y., Davidson, R. J., Hui, D. J., Yazicioglu, M., Elkouby, L., Hinderer, C. J., Faella, A., et al. (2013) · Science Translational Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 355

Long-term Safety and Efficacy Following Systemic Administration of a Self-complementary AAV Vector Encoding Human FIX Pseudotyped With Serotype 5 and 8 Capsid Proteins

Nathwani, A. C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., Nawathe, S., Waddington, S. N., Bronson, R., Jackson, S., et al. (2011) · Molecular Therapy

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 345

IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Leborgne, C., Barbon, E., Alexander, J. M., Hanby, H., Delignat, S., Cohen, D. M., Collaud, F., Muraleetharan, S., Lupo, D., Silverberg, J., et al. (2020) · Nature Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 336

Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy

Jiang, H., Couto, L. B., Patarroyo-White, S., Liu, T., Nagy, D., Vargas, J. A., Zhou, S., Scallan, C. D., Sommer, J., Vijay, S., et al. (2006) · Blood

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 330

Immune Responses to AAV in Clinical Trials

Mingozzi, F., & A. High, K. (2011) · Current Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 324

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

Mingozzi, F., Meulenberg, J. J., Hui, D. J., Basner-Tschakarjan, E., Hasbrouck, N. C., Edmonson, S. A., Hutnick, N. A., Betts, M. R., Kastelein, J. J., Stroes, E. S., et al. (2009) · Blood

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 295

Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant

McIntosh, J., Lenting, P. J., Rosales, C., Lee, D., Rabbanian, S., Raj, D., Patel, N., Tuddenham, E. G. D., Christophe, O. D., McVey, J. H., et al. (2013) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

Cited by 278

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

Bennicelli, J., Wright, J. F., Komaromy, A., Jacobs, J. B., Hauck, B., Zelenaia, O., Mingozzi, F., Hui, D., Chung, D., Rex, T. S., et al. (2008) · Molecular Therapy

Retinal Development and DisordersVirus-based gene therapy research

Cited by 267

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

Meliani, A., Boisgerault, F., Hardet, R., Marmier, S., Collaud, F., Ronzitti, G., Leborgne, C., Costa Verdera, H., Simon Sola, M., Charles, S., et al. (2018) · Nature Communications

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 265

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Mingozzi, F., Hasbrouck, N. C., Basner-Tschakarjan, E., Edmonson, S. A., Hui, D. J., Sabatino, D. E., Zhou, S., Wright, J. F., Jiang, H., Pierce, G. F., et al. (2007) · Blood

Virus-based gene therapy researchHemophilia Treatment and Research

Cited by 254