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Successful Treatment of Severe MSUD in Bckdhb-/- Mice with Neonatal AAV Gene Therapy

Clement Pontoizeau,Clovis Gaborit,Nolan Tual,Marcelo Simon-Sola,Irina Rotaru, Marion Benoist,Pasqualina Colella,Antonin Lamaziere,Anais Brassier,Jean-Baptiste Arnoux,Agnes Rotig,Chris Ottolenghi,Pascale de Lonlay,Federico Mingozzi,Marina Cavazzana,Manuel Schiff

JOURNAL OF INHERITED METABOLIC DISEASE（2024）

引用 2|浏览43
关键词
AAV8,adeno associated virus,BCKDH,BCKDHB,branched-chain 2-keto acid dehydrogenase,EF1 alpha,gene therapy,maple syrup urine disease,mouse model,MSUD
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