- 专利标题: MOLECULES FOR ORGANELLE-SPECIFIC PROTEIN DELIVERY
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申请号: US17214757申请日: 2021-03-26
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公开(公告)号: US20210355177A1公开(公告)日: 2021-11-18
- 发明人: Joan David Bettoun , Rebecca Wissner
- 申请人: Larimar Therapeutics, Inc.
- 申请人地址: US PA Bala Cynwyd
- 专利权人: Larimar Therapeutics, Inc.
- 当前专利权人: Larimar Therapeutics, Inc.
- 当前专利权人地址: US PA Bala Cynwyd
- 主分类号: C07K14/47
- IPC分类号: C07K14/47 ; C07K14/005 ; A61K47/64 ; C12N9/10
摘要:
The present disclosure provides a fusion protein useful for treating a non-nuclear organelle associated disorder, such as a genetic disorder, e.g., Friedrich's Ataxia. The fusion protein may comprise a protein of interest to be delivered to a non-nuclear organelle; an organelle targeting sequence (OTS); a cell penetrating peptide (CPP); and a target enhancing sequence (TES); wherein the CPP is capable of interference with delivery of the protein of interest to the non-nuclear organelle; and wherein the TES prevents said interference by the CPP. The fusion protein may also comprise a protein of interest to be delivered to a non-nuclear organelle; a CPP and a TES. The present disclosure also provides methods for treating a non-nuclear organelle associated disorder by administering the fusion protein to a subject in need thereof.
公开/授权文献
- US11891420B2 Molecules for organelle-specific protein delivery 公开/授权日:2024-02-06
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