- 专利标题: Compositions and methods for treating phenylketonuria
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申请号: US16617462申请日: 2018-05-31
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公开(公告)号: US11939600B2公开(公告)日: 2024-03-26
- 发明人: Kiyoshi Tachikawa , Carlos Gustavo Perez-Garcia , Padmanabh Chivukula , Hari Bhaskaran , Sean Christopher Daugherty , Christian W. Cobaugh
- 申请人: Arcturus Therapeutics, Inc.
- 申请人地址: US CA San Diego
- 专利权人: Arcturus Therapeutics, Inc.
- 当前专利权人: Arcturus Therapeutics, Inc.
- 当前专利权人地址: US CA San Diego
- 代理机构: MINTZ, LEVIN, COHN, FERRIS, GLOVSKY AND POPEO, P.C.
- 国际申请: PCT/US2018/035476 2018.05.31
- 国际公布: WO2018/222925A 2018.12.06
- 进入国家日期: 2019-11-26
- 主分类号: C12N9/02
- IPC分类号: C12N9/02 ; A61K31/7115 ; A61K48/00
摘要:
This invention provides a range of translatable polynucleotide and oligomer molecules for expressing a human phenylalanine hydroxylase (PAH), or a fragment thereof having PAH activity. The polynucleotide and oligomer molecules are expressible to provide the human PAH or a fragment thereof having PAH activity. The molecules can be used as active agents to express an active polypeptide or protein in cells or subjects. The agents can be used in methods for ameliorating, preventing, delaying onset, or treating a disease or condition associated with phenylketonuria, decreased metabolism of phenylalanine, or increased levels of phenylalanine in a subject.
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