Risultati della ricerca - Ronzitti, Giuseppe

Combination Therapy Is the New Gene Therapy? di Ronzitti, Giuseppe, Mingozzi, Federico

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Emerging Issues in AAV-Mediated In Vivo Gene Therapy di Colella, Pasqualina, Ronzitti, Giuseppe, Mingozzi, Federico

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Human Immune Responses to Adeno-Associated Virus (AAV) Vectors di Ronzitti, Giuseppe, Gross, David-Alexandre, Mingozzi, Federico

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Raft-partitioning of calcium channels regulates their function di Ronzitti, Giuseppe, Bucci, Giovanna, Stephens, Gary, Chieregatti, Evelina

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Progress and challenges of gene therapy for Pompe disease di Ronzitti, Giuseppe, Collaud, Fanny, Laforet, Pascal, Mingozzi, Federico

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Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients di Gross, David-Alexandre, Tedesco, Novella, Leborgne, Christian, Ronzitti, Giuseppe

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The regulation of synaptic function by α-synuclein di Bellani, Serena, Sousa, Vitor L, Ronzitti, Giuseppe, Valtorta, Flavia, Meldolesi, Jacopo, Chieregatti, Evelina

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Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction di Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight

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Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction di Han, Sang-oh, Ronzitti, Giuseppe, Arnson, Benjamin, Leborgne, Christian, Li, Songtao, Mingozzi, Federico, Koeberl, Dwight

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Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics di Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico

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Exposure to wild-type AAV drives distinct capsid immunity profiles in humans di Kuranda, Klaudia, Jean-Alphonse, Priscilla, Leborgne, Christian, Hardet, Romain, Collaud, Fanny, Marmier, Solenne, Costa Verdera, Helena, Ronzitti, Giuseppe, Veron, Philippe, Mingozzi, Federico

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Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle di Poupiot, Jérôme, Costa Verdera, Helena, Hardet, Romain, Colella, Pasqualina, Collaud, Fanny, Bartolo, Laurent, Davoust, Jean, Sanatine, Peggy, Mingozzi, Federico, Richard, Isabelle, Ronzitti, Giuseppe

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

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Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome di Parrini, Martina, Naskar, Shovan, Alberti, Micol, Colombi, Ilaria, Morelli, Giovanni, Rocchi, Anna, Nanni, Marina, Piccardi, Federica, Charles, Severine, Ronzitti, Giuseppe, Mingozzi, Federico, Contestabile, Andrea, Cancedda, Laura

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

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

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

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Parkin regulates kainate receptors by interacting with the GluK2 subunit di 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

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

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

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