换一批摘要In some aspects the disclosure provides compositions and methods for promoting expression of functional BCKDHA protein, which is the E1-alpha subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex, and/or BCKDHB, which is the E1-beta subunit of the branched-chain alpha-keto acid (BCAA) dehydrogenase complex in a subject. In some embodiments, the disclosure provides methods of treating a subject having Maple Syrup Urine Disease (MSUD).
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申请/专利号
US202218288211A
申请日期
2022-04-26
公开/公告号
公开/公告日
2024-06-27
主分类号
A61K38/44（含肽的医药配制品）
分类号
A61K38/44 A61K48/0033 A61K48/0066 A61P3/00 C12N15/86 C12Y102/04004 C12N2750/14143
发明/设计人
学术成果认领
国别省市代码
美国;US
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