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公开(公告)号：US20240318198A1
公开(公告)日：2024-09-26
申请号：US18274485
申请日：2022-02-02
Inventor： Ryan BUTLER , Steven J. GRAY , Hye Ri KANG , Berge A. MINASSIAN , Emrah GUMUSGOZ
IPC: C12N15/86 , C12N15/113
CPC classification number: C12N15/86 , C12N15/113 , C12N2310/531 , C12N2750/14143
Abstract: The present disclosure provides methods and compositions for the treatment of Angelman Syndrome The methods and compositions of the present disclosure comprise isolated nucleic acid molecules, rAAV vectors and rAAV viral vectors comprising polynucleotide sequences encoding for short hairpin RNA (shRNA) molecules directed against UBE3A-ATS.
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