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Composition for treatment of Crigler-Najjar syndrome
专利权人:
The Trustees of the University of Pennsylvania
发明人:
Tretiakova, Anna P.,Sidrane, Jenny Agnes,Wilson, James M.
申请号:
AU2016370590
公开号:
AU2016370590A1
申请日:
2016.12.14
申请国别(地区):
AU
年份:
2018
代理人:
摘要:
The invention provides a polynucleotide sequence (e.g., a gene, e.g., DNA or RNA) encoding UGT1A1 (e.g., expressing human UGT1A1). The invention further provides a vector, such as an adeno-associated virus (AAV) vector (e.g., AAV8) having a vector genome including inverted terminal repeat sequences and a UGT1 Al coding sequence operably linked to one or more expression control sequences (e.g., expression control sequences including a liver-specific promoter). Also provided are compositions containing these AAV vectors and methods of treating Crigler-Nijjar syndrome type I, Crigler-Nijjar syndrome type II, and Gilbert syndrome.
来源网站:
中国工程科技知识中心
来源网址:
http://www.ckcest.cn/home/

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