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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
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Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome by Fanny Collaud, Giulia Bortolussi, Laurence Guianvarc’h, Sem J. Aronson, Thierry Bordet, Philippe Veron, Severine Charles, Patrice Vidal, Marcelo Simon Sola, Stephanie Rundwasser, Delphine G. Dufour, Florence Lacoste, Cyril Luc, Laetitia v. Wittenberghe, Samia Martin, Christine Le Bec, Piter J. Bosma, Andres F. Muro, Giuseppe Ronzitti, Matthias Hebben, Federico Mingozzi
Published 2019-03-01
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