Combination Therapy Is the New Gene Therapy?

od Ronzitti, Giuseppe, Mingozzi, Federico
Wydane w Mol Ther (2018)

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

od Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico
Wydane w Mol Ther Methods Clin Dev (2017)

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

od Ronzitti, Giuseppe, Gross, David-Alexandre, Mingozzi, Federico
Wydane w Front Immunol (2020)

Raft-partitioning of calcium channels regulates their function

od Ronzitti, Giuseppe, Bucci, Giovanna, Stephens, Gary, Chieregatti, Evelina
Wydane w Channels (Austin) (2015)

Progress and challenges of gene therapy for Pompe disease

od Ronzitti, Giuseppe, Collaud, Fanny, Laforet, Pascal, Mingozzi, Federico
Wydane w Ann Transl Med (2019)

The regulation of synaptic function by α-synuclein

od Bellani, Serena, Sousa, Vitor L, Ronzitti, Giuseppe, Valtorta, Flavia, Meldolesi, Jacopo, Chieregatti, Evelina
Wydane 2010

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

od Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight
Wydane w Mol Ther Methods Clin Dev (2017)

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

od Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight
Wydane w Mol Ther Methods Clin Dev (2019)

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

od Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico
Wydane w Mol Ther Nucleic Acids (2016)

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

od Kuranda, Klaudia, Jean-Alphonse, Priscilla, Leborgne, Christian, Hardet, Romain, Collaud, Fanny, Marmier, Solenne, Costa Verdera, Helena, Ronzitti, Giuseppe, Veron, Philippe, Mingozzi, Federico
Wydane w J Clin Invest (2018)

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

od Poupiot, Jérôme, Costa Verdera, Helena, Hardet, Romain, Colella, Pasqualina, Collaud, Fanny, Bartolo, Laurent, Davoust, Jean, Sanatine, Peggy, Mingozzi, Federico, Richard, Isabelle, Ronzitti, Giuseppe
Wydane w Mol Ther Methods Clin Dev (2019)

Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain

od Orefice, Nicola Salvatore, Souchet, Benoît, Braudeau, Jérôme, Alves, Sandro, Piguet, Françoise, Collaud, Fanny, Ronzitti, Giuseppe, Tada, Satoru, Hantraye, Philippe, Mingozzi, Federico, Ducongé, Frédéric, Cartier, Nathalie
Wydane w Mol Ther Methods Clin Dev (2019)

Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity

od Bartolo, Laurent, Li Chung Tong, Stéphanie, Chappert, Pascal, Urbain, Dominique, Collaud, Fanny, Colella, Pasqualina, Richard, Isabelle, Ronzitti, Giuseppe, Demengeot, Jocelyne, Gross, David A., Mingozzi, Federico, Davoust, Jean
Wydane w JCI Insight (2019)

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

od Fitzpatrick, Zachary, Leborgne, Christian, Barbon, Elena, Masat, Elisa, Ronzitti, Giuseppe, van Wittenberghe, Laetitia, Vignaud, Alban, Collaud, Fanny, Charles, Séverine, Simon Sola, Marcelo, Jouen, Fabienne, Boyer, Olivier, Mingozzi, Federico
Wydane w Mol Ther Methods Clin Dev (2018)

SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson’s Disease

od Espinoza, Stefano, Scarpato, Margherita, Damiani, Devid, Managò, Francesca, Mereu, Maddalena, Contestabile, Andrea, Peruzzo, Omar, Carninci, Piero, Santoro, Claudio, Papaleo, Francesco, Mingozzi, Federico, Ronzitti, Giuseppe, Zucchelli, Silvia, Gustincich, Stefano
Wydane w Mol Ther (2020)

Parkin regulates kainate receptors by interacting with the GluK2 subunit

od Maraschi, AnnaMaria, Ciammola, Andrea, Folci, Alessandra, Sassone, Francesca, Ronzitti, Giuseppe, Cappelletti, Graziella, Silani, Vincenzo, Sato, Shigeto, Hattori, Nobutaka, Mazzanti, Michele, Chieregatti, Evelina, Mulle, Christophe, Passafaro, Maria, Sassone, Jenny
Wydane w Nat Commun (2014)

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

od Aronson, Sem J., Bakker, Robert S., Moenis, Sascha, van Dijk, Remco, Bortolussi, Giulia, Collaud, Fanny, Shi, Xiaoxia, Duijst, Suzanne, ten Bloemendaal, Lysbeth, Ronzitti, Giuseppe, Muro, Andrés F., Mingozzi, Federico, Beuers, Ulrich, Bosma, Piter J.
Wydane w Mol Ther Methods Clin Dev (2020)

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

od Shi, Xiaoxia, Aronson, Sem J., ten Bloemendaal, Lysbeth, Duijst, Suzanne, Bakker, Robert S., de Waart, Dirk R., Bortolussi, Giulia, Collaud, Fanny, Oude Elferink, Ronald P., Muro, Andrés F., Mingozzi, Federico, Ronzitti, Giuseppe, Bosma, Piter J.
Wydane w Mol Ther Methods Clin Dev (2020)

Exogenous α-Synuclein Decreases Raft Partitioning of Ca(v)2.2 Channels Inducing Dopamine Release

od Ronzitti, Giuseppe, Bucci, Giovanna, Emanuele, Marco, Leo, Damiana, Sotnikova, Tatyana D., Mus, Liudmila V., Soubrane, Camille H., Dallas, Mark L., Thalhammer, Agnes, Cingolani, Lorenzo A., Mochida, Sumiko, Gainetdinov, Raul R., Stephens, Gary J., Chieregatti, Evelina
Wydane w J Neurosci (2014)

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

od Ronzitti, Giuseppe, Bortolussi, Giulia, van Dijk, Remco, Collaud, Fanny, Charles, Severine, Leborgne, Christian, Vidal, Patrice, Martin, Samia, Gjata, Bernard, Sola, Marcelo Simon, van Wittenberghe, Laetitia, Vignaud, Alban, Veron, Philippe, Bosma, Piter J, Muro, Andres F, Mingozzi, Federico
Wydane w Mol Ther Methods Clin Dev (2016)