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31.发明申请RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTI-TRYPSIN DEFICIENCIES 审中-公开基于RAAV的组合物和治疗ALPHA-1抗血小板缺乏症的方法公开(公告)号：US20160326524A1
公开(公告)日：2016-11-10
申请号：US15098833
申请日：2016-04-14
发明人： Terence Flotte , Christian Mueller
IPC分类号： C12N15/113 , A61K31/713 , A61K48/00 , A61K38/57 , C12N7/00 , C07K14/81
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).
摘要翻译： 本发明涉及分离的核酸和基于rAAV的组合物，用于治疗遗传疾病（例如α-1抗胰蛋白酶缺乏症）的方法和试剂盒。
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公开(公告)号：US20160186211A1
公开(公告)日：2016-06-30
申请号：US14952217
申请日：2015-11-25
发明人： Terence Flotte , Christian Mueller
IPC分类号： C12N15/86 , A61K48/00 , A61K35/12 , C12N7/00 , C12N15/113
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：US11859179B2
公开(公告)日：2024-01-02
申请号：US16611581
申请日：2018-05-09
发明人： Christian Mueller , Abbas Abdallah
CPC分类号： C12N15/11 , A61K35/76 , C12N9/22 , C12N15/86 , C12N15/8616 , C12N15/907 , C12N2310/20 , C12N2750/14143 , C12N2750/14171 , C12N2800/80
摘要： Aspects of the disclosure relate to recombinant gene editing complexes comprising a recombinant gene editing protein and guide RNA (gRNA) that specifically hybridizes to a region of a C90RF72 gene (e.g., a region flanking a G4C2 repeat or within a exonic region of the gene).
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公开(公告)号：US20220204974A1
公开(公告)日：2022-06-30
申请号：US17576130
申请日：2022-01-14
IPC分类号： C12N15/113 , C12N15/67 , A61K48/00 , A61K35/12 , C12N7/00 , C12N15/86 , A61K31/713 , A61K38/57 , C07K14/81
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：US20210230568A1
公开(公告)日：2021-07-29
申请号：US17051632
申请日：2019-05-03
发明人： Scot A. Wolfe , Charles P. Emerson, JR. , Sukanya Iyer , Sneha Suresh , Christian Mueller , Jennifer Chen , Dongsheng Guo , Oliver King
摘要： The present invention is directed to the filed of gene therapy. In particular, compositions and methods are disclosed that repair gene microduplication mutations by reversion to a wild type sequence. For example, the creation of a double stranded break by a programmable nuclease protein within a microduplication induces the microhomology mediated end joining DNA repair pathway that in the process of DNA repair removes the microduplication mutation and restores the wild type sequence.
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公开(公告)号：US10851375B2
公开(公告)日：2020-12-01
申请号：US16403948
申请日：2019-05-06
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：US20200339988A1
公开(公告)日：2020-10-29
申请号：US16795757
申请日：2020-02-20
IPC分类号： C12N15/113 , C12N15/67 , A61K48/00 , A61K35/12 , C12N7/00 , C12N15/86 , A61K31/713 , A61K38/57 , C07K14/81
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：US20190316126A1
公开(公告)日：2019-10-17
申请号：US16403948
申请日：2019-05-06
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：US20190276826A1
公开(公告)日：2019-09-12
申请号：US16364126
申请日：2019-03-25
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：US20190169635A1
公开(公告)日：2019-06-06
申请号：US16216324
申请日：2018-12-11
发明人： Christian Mueller , Florie Borel
IPC分类号： C12N15/85 , C12N15/09 , A01K67/027 , C07K14/81
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 serpin1A 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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