Emerging Issues in AAV-Mediated In Vivo Gene Therapy

by Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi
Published 2018-03-01

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

by Giuseppe Ronzitti, David-Alexandre Gross, Federico Mingozzi
Published 2020-04-01

Tregitope Peptides: The Active Pharmaceutical Ingredient of IVIG?

by Anne S. De Groot, Leslie Cousens, Federico Mingozzi, William Martin
Published 2013-01-01

Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia

by Paul E. Monahan, Claude Négrier, Michael Tarantino, Leonard A. Valentino, Federico Mingozzi
Published 2021-06-01

Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction

by Sang-oh Han, Giuseppe Ronzitti, Benjamin Arnson, Christian Leborgne, Songtao Li, Federico Mingozzi, Dwight Koeberl
Published 2017-03-01

Induction of hematopoietic microchimerism by gene-modified BMT elicits antigen-specific B and T cell unresponsiveness toward gene therapy products

by Jérémie Martinet, Gwladys Bourdenet, Amine Meliani, Laetitia Jean, Sahil Adriouch, José Cohen, Federico Mingozzi, Olivier Boyer
Published 2016-09-01

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

by Elena Barbon, Mattia Ferrarese, Laetitia van Wittenberghe, Peggy Sanatine, Giuseppe Ronzitti, Fanny Collaud, Pasqualina Colella, Mirko Pinotti, Federico Mingozzi
Published 2016-01-01

Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis.

by Caroline J Aalbers, Lisette Bevaart, Scott Loiler, Karin de Cortie, J Fraser Wright, Federico Mingozzi, Paul P Tak, Margriet J Vervoordeldonk
Published 2015-01-01

IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression.

by Irene Gil-Fariña, Marianna Di Scala, Lucia Vanrell, Cristina Olagüe, Africa Vales, Katherine A High, Jesus Prieto, Federico Mingozzi, Gloria Gonzalez-Aseguinolaza
Published 2013-01-01

Efficacy of adeno-associated virus gene therapy in a MNGIE murine model enhanced by chronic exposure to nucleosides

by Ferran Vila-Julià, Raquel Cabrera-Pérez, Yolanda Cámara, Miguel Molina-Berenguer, Silvia Lope-Piedrafita, Michio Hirano, Federico Mingozzi, Javier Torres-Torronteras, Ramon Martí
Published 2020-12-01

Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle

by Jérôme Poupiot, Helena Costa Verdera, Romain Hardet, Pasqualina Colella, Fanny Collaud, Laurent Bartolo, Jean Davoust, Peggy Sanatine, Federico Mingozzi, Isabelle Richard, Giuseppe Ronzitti
Published 2019-12-01

AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes

by Daniel J Hui, Shyrie C Edmonson, Gregory M Podsakoff, Gary C Pien, Lacramioara Ivanciu, Rodney M Camire, Hildegund Ertl, Federico Mingozzi, Katherine A High, Etiena Basner-Tschakarjan
Published 2015-01-01

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

by Zachary Fitzpatrick, Christian Leborgne, Elena Barbon, Elisa Masat, Giuseppe Ronzitti, Laetitia van Wittenberghe, Alban Vignaud, Fanny Collaud, Séverine Charles, Marcelo Simon Sola, Fabienne Jouen, Olivier Boyer, Federico Mingozzi
Published 2018-06-01

A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene

by Sem J. Aronson, Robert S. Bakker, Sascha Moenis, Remco van Dijk, Giulia Bortolussi, Fanny Collaud, Xiaoxia Shi, Suzanne Duijst, Lysbeth ten Bloemendaal, Giuseppe Ronzitti, Andrés F. Muro, Federico Mingozzi, Ulrich Beuers, Piter J. Bosma
Published 2020-09-01

AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice

by Pasqualina Colella, Pauline Sellier, Helena Costa Verdera, Francesco Puzzo, Laetitia van Wittenberghe, Nicolas Guerchet, Nathalie Daniele, Bernard Gjata, Solenne Marmier, Severine Charles, Marcelo Simon Sola, Isabella Ragone, Christian Leborgne, Fanny Collaud, Federico Mingozzi
Published 2019-03-01

Efficacy of AAV8-hUGT1A1 with Rapamycin in neonatal, suckling, and juvenile rats to model treatment in pediatric CNs patients

by Xiaoxia Shi, Sem J. Aronson, Lysbeth ten Bloemendaal, Suzanne Duijst, Robert S. Bakker, Dirk R. de Waart, Giulia Bortolussi, Fanny Collaud, Ronald P. Oude Elferink, Andrés F. Muro, Federico Mingozzi, Giuseppe Ronzitti, Piter J. Bosma
Published 2021-03-01

Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors

by Elena Barbon, Gabriel Ayme, Amel Mohamadi, Jean‐François Ottavi, Charlotte Kawecki, Caterina Casari, Sebastien Verhenne, Solenne Marmier, Laetitia van Wittenberghe, Severine Charles, Fanny Collaud, Cecile V Denis, Olivier D Christophe, Federico Mingozzi, Peter J Lenting
Published 2020-04-01

Th1 response and systemic treg deficiency in inclusion body myositis.

by Yves Allenbach, Wahiba Chaara, Michelle Rosenzwajg, Adrien Six, Nicolas Prevel, Federico Mingozzi, Julia Wanschitz, Lucile Musset, Jean-Luc Charuel, Bruno Eymard, Benoit Salomon, Charles Duyckaerts, Thierry Maisonobe, Odile Dubourg, Serge Herson, David Klatzmann, Olivier Benveniste
Published 2014-01-01

A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome

by Giuseppe Ronzitti, Giulia Bortolussi, Remco van Dijk, Fanny Collaud, Severine Charles, Christian Leborgne, Patrice Vidal, Samia Martin, Bernard Gjata, Marcelo Simon Sola, Laetitia van Wittenberghe, Alban Vignaud, Philippe Veron, Piter J Bosma, Andres F Muro, Federico Mingozzi
Published 2016-01-01

Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector

by Giulia De Sabbata, Florence Boisgerault, Corrado Guarnaccia, Alessandra Iaconcig, Giulia Bortolussi, Fanny Collaud, Giuseppe Ronzitti, Marcelo Simon Sola, Patrice Vidal, Jeremy Rouillon, Severine Charles, Emanuele Nicastro, Lorenzo D’Antiga, Petr Ilyinskii, Federico Mingozzi, Takashi Kei Kishimoto, Andrés F. Muro
Published 2021-03-01