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公开(公告)号:US12290574B2
公开(公告)日:2025-05-06
申请号:US17250712
申请日:2019-08-23
Applicant: SPARK THERAPEUTICS, INC.
Inventor: Xavier Anguela , Liron Elkouby
IPC: A61K48/00 , A61K9/00 , A61P7/04 , C07K14/755 , C07K14/775 , C07K14/81 , C12N7/00 , C12N15/86 , A61K38/00
Abstract: The disclosure includes CpG reduced regulatory elements, polynucleotides comprising such CpG reduced regulatory elements, expression cassettes comprising such CpG reduced regulatory elements, and recombinant AAV vectors comprising such CpG reduced regulatory elements. The regulatory elements can be, for example, operably coupled to a transgene.