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公开(公告)号：US20240376498A1
公开(公告)日：2024-11-14
申请号：US18785836
申请日：2024-07-26
Inventor： Steven J. GRAY , Frances C. SHAFFO
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：US20210180085A1
公开(公告)日：2021-06-17
申请号：US17112299
申请日：2020-12-04
Inventor： Steven J. GRAY , Sarah Sinnett
IPC: C12N15/86 , C12N15/113
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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Patent Agency Ranking
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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公开(公告)号：US20250082782A1
公开(公告)日：2025-03-13
申请号：US18729389
申请日：2023-01-27
Applicant: THE BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM , THE GOVERNING COUNCIL OF THE UNIVERSITY OF TORONTO
Inventor： Steven J. GRAY , David R. HAMPSON , Hye Ri KANG , Hayes Ga-Hei WONG , Alexander W.M. HOOPER
Abstract: This technology relates to polynucleotides comprising optimized FMR1 open reading frame (ORF) sequences, viral vectors comprising the same, and methods of using the same for delivery of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of FMR1, such as Fragile X syndrome (“FXS”).
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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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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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公开(公告)号：US20210139934A1
公开(公告)日：2021-05-13
申请号：US17092240
申请日：2020-11-07
Inventor： Steven J. GRAY , Frances C. SHAFFO
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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