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SaveGene Therapy in Patients with the Crigler-Najjar Syndrome
D'Antiga, Lorenzo; Beuers, Ulrich; Ronzitti, Giuseppe; Brunetti-Pierri, Nicola; Baumann, Ulrich; Di Giorgio, Angelo; Aronson, Sem; Hubert, Aurelie; Romano, Roberta; Junge, Norman; Bosma, Piter; Bortolussi, Giulia; Muro, Andres F.; Soumoudronga, Ravaka F.; Veron, Philippe; Collaud, Fanny; Knuchel-Legendre, Nathalie; Labrune, Philippe; Mingozzi, Federico
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SaveEarly Phase Clinical Immunogenicity of Valoctocogene Roxaparvovec, an AAV5-Mediated Gene Therapy for Hemophilia A
Long, Brian R.; Veron, Philippe; Kuranda, Klaudia; Hardet, Romain; Mitchell, Nina; Hayes, Gregory M.; Wong, Wing Yen; Lau, Kelly; Li, Mingjin; Hock, M. Benjamin; Zoog, Stephen J.; Vettermann, Christian; Mingozzi, Federico; Schweighardt, Becky
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SaveCapsid-specific removal of circulating antibodies to adeno-associated virus vectors
Bertin, Berangere; Veron, Philippe; Leborgne, Christian; Deschamps, Jack-Yves; Moullec, Sophie; Fromes, Yves; Collaud, Fanny; Boutin, Sylvie; Latournerie, Virginie; van Wittenberghe, Laetitia; Delache, Benoit; Le Grand, Roger; Dereuddre-Bosquet, Nathalie; Benveniste, Olivier; Moullier, Philippe; Masurier, Carole; Merten, Otto; Mingozzi, Federico
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