- 专利标题: Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
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申请号: US16756988申请日: 2018-10-22
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公开(公告)号: US11723988B2公开(公告)日: 2023-08-15
- 发明人: Deniz Dalkara , Hanen Khabou , José-Alain Sahel , Thierry Leveillard , Jens Duebel
- 申请人: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) , SORBONNE UNIVERSITÉ
- 申请人地址: FR Paris
- 专利权人: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE),SORBONNE UNIVERSITE
- 当前专利权人: INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE),SORBONNE UNIVERSITE
- 当前专利权人地址: FR Paris; FR Paris
- 代理机构: Saliwanchik, Lloyd & Eisenschenk
- 优先权: EP 306430 2017.10.20
- 国际申请: PCT/EP2018/078856 2018.10.22
- 国际公布: WO2019/077159A 2019.04.25
- 进入国家日期: 2020-04-17
- 主分类号: A61K48/00
- IPC分类号: A61K48/00 ; A61P27/02 ; C12N15/86
摘要:
Several new vector-promoter combinations to overcome the limitations associated with AAV-mediated cone transduction in the fovea are provided. The delivery modality relies on a cone-specific promoter and result in high-level transgene expression compatible with optogenetic vision restoration. Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector comprising a VP1 capsid protein as set forth in SEQ ID NO: 11 and the polynucleotide of interest under the control of the pR1.7 promoter as set forth in SEQ ID NO: 12 are also provided.
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