发明授权
- 专利标题: Methods and compositions for treatment of ocular disorders and blinding diseases
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申请号: US17099421申请日: 2020-11-16
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公开(公告)号: US12097267B2公开(公告)日: 2024-09-24
- 发明人: Jeannette Bennicelli , Jean Bennett , Junwei Sun
- 申请人: The Trustees of the University of Pennsylvania
- 申请人地址: US PA Philadelphia
- 专利权人: The Trustees of the University of Pennsylvania
- 当前专利权人: The Trustees of the University of Pennsylvania
- 当前专利权人地址: US PA Philadelphia
- 代理机构: Howson & Howson LLP
- 代理商 Colleen M. Schaller; Francis J. Coffey
- 分案原申请号: US16066970
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; A61K38/44 ; A61P27/02 ; C12N9/02 ; C12N15/86
摘要:
Codon optimized nucleic acid sequences for the long form and short form of RdCVF are provided, as well as recombinant viral vectors, such as AAV, expression cassettes, proviral plasmids or other plasmids containing the codon optimized sequences. Recombinant vectors are provided that express the codon optimized RdCVFL and RdCVF individually, or express two copies of a codon optimized RdCVF or RdCVFL nucleic acid sequence, or both RdCVFL and RdCVF in a single vector or virus. Compositions containing these codon optimized sequences are useful in methods for treating, retarding or halting certain blinding diseases resulting from the absence or inappropriate expression of RdCVF and RdCVFL.
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