Predictive power of deleterious single amino acid changes to infer on AAV2 and AAV2-13 capsids fitness
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Published in Molecular therapy. Methods & clinical development (12.09.2024)
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Published in Molecular therapy. Methods & clinical development (15.03.2019)
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A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome
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Published in Molecular therapy. Methods & clinical development (01.01.2016)
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Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector
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Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase
Puzzo, Francesco, Colella, Pasqualina, Biferi, Maria G, Bali, Deeksha, Paulk, Nicole K, Vidal, Patrice, Collaud, Fanny, Simon-Sola, Marcelo, Charles, Severine, Hardet, Romain, Leborgne, Christian, Meliani, Amine, Cohen-Tannoudji, Mathilde, Astord, Stephanie, Gjata, Bernard, Sellier, Pauline, van Wittenberghe, Laetitia, Vignaud, Alban, Boisgerault, Florence, Barkats, Martine, Laforet, Pascal, Kay, Mark A, Koeberl, Dwight D, Ronzitti, Giuseppe, Mingozzi, Federico
Published in Science translational medicine (29.11.2017)
Published in Science translational medicine (29.11.2017)
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A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III
Gardin, Antoine, Rouillon, Jérémy, Montaivo-Romera, Valle, Rossiaud, Lucille, Vidal, Patrice, Launay, Romain, Vie, Mallaury, Benchekroun, Youssef Krimi, Cosette, Jérémie, Bertin, Bérangére, La Bella, Tiziana, Dubreuil, Guillaume, Nozi, Justine, Jauze, Louisa, Fragnoud, Romain, Daniele, Nathalie, Van Wittenberghe, Laetitia, Esque, Jérémy, André, Isabelle, Nissan, Xavier, Hoch, Lucile, Ronzitti, Giuseppe
Published in The Journal of clinical investigation (01.01.2024)
Published in The Journal of clinical investigation (01.01.2024)
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A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III
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Published in The Journal of clinical investigation (16.01.2024)
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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
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감소된 간 향성을 갖는 AAV9과 AAVrh74의 하이브리드 재조합 아데노-관련 바이러스 세로타입
GICQUEL EVELYNE, MINGOZZI FREDERICO, RICHARD ISABELLE, RONZITTI GIUSEPPE, VIDAL PATRICE
Year of Publication 10.02.2021
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Year of Publication 10.02.2021
Patent
158. Cryptic ATG Removal from Synthetic Introns Increase the Therapeutic Efficacy of AAV Vector Mediated Gene Transfer
Ronzitti, Giuseppe, Collaud, Fanny, Bortolussi, Giulia, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Muro, Andres, Mingozzi, Federico
Published in Molecular therapy (01.05.2016)
Published in Molecular therapy (01.05.2016)
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Synergism of dual AAV gene therapy and rapamycin rescues GSDIII phenotype in muscle and liver
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Published in JCI insight (16.05.2024)
Published in JCI insight (16.05.2024)
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Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression
Vidal, Patrice, Pagliarani, Serena, Colella, Pasqualina, Costa Verdera, Helena, Jauze, Louisa, Gjorgjieva, Monika, Puzzo, Francesco, Marmier, Solenne, Collaud, Fanny, Simon Sola, Marcelo, Charles, Severine, Lucchiari, Sabrina, van Wittenberghe, Laetitia, Vignaud, Alban, Gjata, Bernard, Richard, Isabelle, Laforet, Pascal, Malfatti, Edoardo, Mithieux, Gilles, Rajas, Fabienne, Comi, Giacomo Pietro, Ronzitti, Giuseppe, Mingozzi, Federico
Published in Molecular therapy (01.03.2018)
Published in Molecular therapy (01.03.2018)
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Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector
de Sabbata, Giulia, Boisgerault, Florence, Guarnaccia, Corrado, Iaconcig, Alessandra, Bortolussi, Giulia, Collaud, Fanny, Ronzitti, Giuseppe, Sola, Marcelo Simon, Vidal, Patrice, Rouillon, Jeremy, Charles, Severine, Nicastro, Emanuele, D’antiga, Lorenzo, Ilyinskii, Petr, Mingozzi, Federico, Kishimoto, Takashi Kei, Muro, Andrés
Published in Molecular therapy. Methods & clinical development (01.03.2021)
Published in Molecular therapy. Methods & clinical development (01.03.2021)
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Journal Article
Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome
Collaud, Fanny, Bortolussi, Giulia, Guianvarc’h, Laurence, Aronson, Sem, Bordet, Thierry, Veron, Philippe, Charles, Severine, Vidal, Patrice, Sola, Marcelo Simon, Rundwasser, Stephanie, Dufour, Delphine, Lacoste, Florence, Luc, Cyril, Wittenberghe, Laetitia, Martin, Samia, Le Bec, Christine, Bosma, Piter, Muro, Andres, Ronzitti, Giuseppe, Hebben, Matthias, Mingozzi, Federico
Published in Molecular therapy. Methods & clinical development (01.03.2019)
Published in Molecular therapy. Methods & clinical development (01.03.2019)
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