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公开(公告)号：US20240191254A1
公开(公告)日：2024-06-13
申请号：US18411647
申请日：2024-01-12
Inventor： Steven J. GRAY , Sarah SINNETT
IPC: C12N15/86 , C12N15/113
CPC classification number: C12N15/86 , C12N15/113 , C12N2310/141 , C12N2310/315 , C12N2310/321
Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to MeCP2 loss and/or misfunction, including RETT syndrome. The methods and compositions of the present disclosure comprise rAAV vectors and rAAV viral vectors comprising transgene nucleic acid molecules comprising nucleic acid sequences encoding for a MeCP2 polypeptide.
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