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公开(公告)号:US20250073314A1
公开(公告)日:2025-03-06
申请号:US18719466
申请日:2022-12-14
Applicant: UNIVERSITÄTSKLINIKUM HAMBURG-EPPENDORF
Inventor: Thomas ESCHENHAGEN , Charlotta BEHRENS , Bärbel ULMER , Julia FREESE , Ingke BRAREN , Lucie CARRIER
Abstract: The present invention relates to nucleic acid molecules encoding Lamin A, vectors, AAV and pharmaceutical compositions comprising said nucleic acid molecules for use in treatment of laminopathies.