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公开(公告)号：US20240084303A1
公开(公告)日：2024-03-14
申请号：US18451147
申请日：2023-08-17
发明人： Christian Mueller , Neil Aronin , Edith L. Pfister
IPC分类号： C12N15/113 , A61P25/28 , C12N7/00 , C12N15/86
CPC分类号： C12N15/113 , A61P25/28 , C12N7/00 , C12N15/86 , C12N2310/141 , C12N2310/3519 , C12N2330/51 , C12N2750/14121 , C12N2750/14141 , C12N2750/14143 , C12N2750/14171
摘要： Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (111 1) and methods of treating Huntington's disease using the same.
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公开(公告)号：US20240076668A1
公开(公告)日：2024-03-07
申请号：US18362190
申请日：2023-07-31
IPC分类号： C12N15/113 , C12N15/11 , C12N15/86
CPC分类号： C12N15/113 , C12N15/111 , C12N15/1137 , C12N15/86 , C12Y115/01001 , C12N2310/14 , C12N2310/141 , C12N2320/32 , C12N2330/51 , C12N2750/14143
摘要： The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.
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公开(公告)号：US20230416757A1
公开(公告)日：2023-12-28
申请号：US18344926
申请日：2023-06-30
IPC分类号： C12N15/113 , A61K35/76 , C12N7/00 , C12N15/86
CPC分类号： C12N15/1137 , A61K35/76 , C12N7/00 , C12N15/86 , A61K48/00
摘要： In some aspects, the disclosure relates to compositions and methods useful for inhibiting SOD1 expression in cells (e.g., cells of a subject). In some embodiments, the disclosure describes isolated nucleic acids engineered to express an inhibitory nucleic acid targeting endogenous SOD1 and an mRNA encoding a hardened SOD1 protein. In some embodiments, compositions and methods described by the disclosure are useful for treating Amyotrophic Lateral Sclerosis (ALS) in a subject.
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公开(公告)号：US20220033824A1
公开(公告)日：2022-02-03
申请号：US17297521
申请日：2019-11-27
IPC分类号： C12N15/113 , C12N15/86 , C12N15/10 , A61K35/761
摘要： The disclosure provides, in some aspects, compositions (e.g., isolated nucleic acids and rAAVs) comprising a transgene which encodes at least one inhibitory nucleic acid which decreases expression of a gene encoding a serine palmitoyltransferase protein (e.g., SPTLC1). In some aspects, the disclosure relates to methods of treating hereditary sensory and autonomic neuropathy 1 (HSAN1) by administering the compositions described by the disclosure to a cell or subject.
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公开(公告)号：US20220010312A1
公开(公告)日：2022-01-13
申请号：US17326400
申请日：2021-05-21
发明人： Christian Mueller , Neil Aronin , Edith L. Pfister
IPC分类号： C12N15/113 , A61P25/28 , C12N7/00 , C12N15/86
摘要： Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.
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公开(公告)号：US20210246450A1
公开(公告)日：2021-08-12
申请号：US17174452
申请日：2021-02-12
IPC分类号： C12N15/113 , C12N15/11 , C12N15/86
摘要： The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.
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公开(公告)号：US10711274B2
公开(公告)日：2020-07-14
申请号：US16364126
申请日：2019-03-25
IPC分类号： C07H21/04 , C12N15/113 , C12N15/11 , C12N15/86
摘要： The invention relates to inhibitory nucleic acids and rAAV-based compositions, methods and kits useful for treating Amyotrophic Lateral Sclerosis.
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公开(公告)号：US10457940B2
公开(公告)日：2019-10-29
申请号：US15705909
申请日：2017-09-15
发明人： Christian Mueller , Neil Aronin , Edith L. Pfister
IPC分类号： C12N7/00 , C12N15/113 , C12N15/86 , A61P25/28
摘要： Aspects of the disclosure relate to compositions and methods useful for treating Huntington's disease. In some embodiments, the disclosure provides interfering nucleic acids (e.g., artificial miRNAs) targeting the huntingtin gene (HTT) and methods of treating Huntington's disease using the same.
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公开(公告)号：US09885057B2
公开(公告)日：2018-02-06
申请号：US14952217
申请日：2015-11-25
IPC分类号： C07H21/02 , C07H21/04 , A61K48/00 , A61K35/00 , C12N15/86 , C12N7/00 , C12N15/113 , A61K35/12
CPC分类号： C12N15/86 , A61K31/713 , A61K35/12 , A61K38/57 , A61K48/005 , A61K48/0058 , C07K14/8125 , C12N7/00 , C12N15/111 , C12N15/113 , C12N15/67 , C12N2310/141 , C12N2310/3519 , C12N2320/31 , C12N2750/14121 , C12N2750/14132 , C12N2750/14143
摘要： The invention relates to isolated nucleic acids and rAAV-based compositions, methods and kits useful for treating genetic diseases (e.g., alpha-1 antitrypsin deficiency).
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公开(公告)号：US20170327843A1
公开(公告)日：2017-11-16
申请号：US15595554
申请日：2017-05-15
发明人： Christian Mueller , Florie Borel
CPC分类号： C12N15/8509 , A01K67/0276 , A01K2227/105 , A01K2267/0306 , C07K14/8125 , C12N15/00 , C12N15/09 , C12N2015/8527
摘要： Transgenic non-human animals, e.g., rodents, e.g., mice comprising genomic mutations that inactive all of the serpinlA genes and thus lack any functional serpinA1 genes. As a result of the genomic mutations, the animals express no hepatic or circulatory AAT protein. Also provided herein are cells and tissues derived from the transgenic mice.
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