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公开(公告)号：US12077772B2
公开(公告)日：2024-09-03
申请号：US17092240
申请日：2020-11-07
Inventor： Steven J. Gray , Frances C. Shaffo
CPC classification number: C12N15/86 , A61K9/0085 , A61K38/1787 , A61K48/005 , A61K48/0083 , C07K14/70571 , C12N7/00 , C12N2750/14143 , C12N2800/22 , C12N2830/50
Abstract: The present disclosure provides methods and compositions for the treatment of diseases and genetic disorders linked to SLC6A1 loss and/or misfunction. 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 GAT1 polypeptide.
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公开(公告)号：US11891616B2
公开(公告)日：2024-02-06
申请号：US17112299
申请日：2020-12-04
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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公开(公告)号：US11753655B2
公开(公告)日：2023-09-12
申请号：US17230484
申请日：2021-04-14
Inventor： Rachel M. Bailey , Steven J. Gray
CPC classification number: C12N15/86 , A61K48/005 , A61P25/02 , C07K14/01 , C07K16/081
Abstract: The present disclosure provides methods and compositions for the treatment of diseases and/or disorders in a subject, including, but not limited to neurological disorders such as giant axonal neuropathy. The methods described herein include direct administration of a gene therapy (e.g. an rAAV viral vector) to a subject via injection into a vagus nerve (e.g. the left vagus nerve) of the subject.
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