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公开(公告)号:US20240269161A1
公开(公告)日:2024-08-15
申请号:US18569019
申请日:2022-06-27
Inventor: Rachel M. BAILEY
IPC: A61K31/713 , A61P25/28 , C12N15/113 , C12N15/86
CPC classification number: A61K31/713 , A61P25/28 , C12N15/113 , C12N15/86 , C12N2310/141 , C12N2750/14143
Abstract: The present disclosure provides methods and compositions for the treatment of tauopathies, such as, for example, Alzheimer's Disease or FTDP-17. The methods and compositions of the present disclosure comprise isolated nucleic acid molecules, rAAV vectors and rAAV viral vectors comprising polynucleotide sequences encoding for artificial micro RNAs (amiRNAs) directed against MAPT.
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公开(公告)号:US20240102050A1
公开(公告)日:2024-03-28
申请号:US18355380
申请日:2023-07-19
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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公开(公告)号:US20210324411A1
公开(公告)日:2021-10-21
申请号:US17230484
申请日:2021-04-14
Inventor: Rachel M. BAILEY , Steven J. GRAY
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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