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Neonatal Gene Therapy Achieves Sustained Disease Rescue of Maple Syrup Urine Disease in Mice

Clément Pontoizeau,Marcelo Simon-Sola,Clovis Gaborit, Vincent Nguyen,Irina Rotaru,Nolan Tual,Pasqualina Colella,Muriel Girard,Maria-Grazia Biferi,Jean-Baptiste Arnoux,Agnès Rötig,Chris Ottolenghi,Pascale de Lonlay,Federico Mingozzi,Marina Cavazzana,Manuel Schiff

Nature Communications（2022）

引用 4|浏览53
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关键词
Experimental models of disease,Gene therapy,Metabolic disorders,Molecular medicine,Science,Humanities and Social Sciences,multidisciplinary
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