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1.发明申请
公开(公告)号：WO2019078916A9
公开(公告)日：2019-04-25
申请号：PCT/US2018/022853
申请日：2018-03-16
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R.
IPC: C12N15/86 , A61K48/00 , C07K14/005 , C12N15/113
Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
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2.发明申请
公开(公告)号：WO2019245973A1
公开(公告)日：2019-12-26
申请号：PCT/US2019/037489
申请日：2019-06-17
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R.
Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle s including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
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3.发明申请ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY 审中-公开微量DTR的ADENO相关病毒载体传递治疗肌萎缩
公开(公告)号：WO2017181015A1
公开(公告)日：2017-10-19
申请号：PCT/US2017/027636
申请日：2017-04-14
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R. , HELLER, Kristin, N.
Abstract: The invention provides for recombinant AAV vectors comprising a miniaturized human micro-dystrophin gene and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
Abstract translation: 本发明提供了包含微型人类微肌营养不良蛋白基因的重组AAV载体以及使用所述重组载体来减少或预防肌营养不良患者的纤维化的方法。
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公开(公告)号：WO2021257595A1
公开(公告)日：2021-12-23
申请号：PCT/US2021/037470
申请日：2021-06-15
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R. , PETERSON, Ellyn , POTTER, Rachael , GRIFFIN, Danielle
IPC: A61K35/761 , A61P21/00 , A61K45/06 , A61K48/005 , C07K16/2887 , C12N7/00
Abstract: The disclosure provides method of treating muscular dystrophy in a subject in need comprising administering a gene therapy vector, such as adeno-associated virus (AAV) vector, expressing a miniaturized human micro-dystrophin gene in combination with a step of suppressing the subject's immune system.
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5.发明申请ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRORNA-29 AND MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY 审中-公开微管相关病毒载体传递MICRNANA-29和微型DYSTROPHIN治疗肌无力
公开(公告)号：WO2017181014A1
公开(公告)日：2017-10-19
申请号：PCT/US2017/027635
申请日：2017-04-14
Inventor： RODINO-KPLAPAC, Louise , MENDELL, Jerry, R. , HELLER, Kristin, N.
IPC: A61K38/17 , A61K48/00 , A61P21/00 , C12N15/113 , C12N15/85
Abstract: The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from dystrophinopathy or muscular dystrophy. The invention also provides for combination therapies comprising expressing both miR-29 and micro-dystrophin to reduce and prevent fibrosis in patients suffering from dystrophinopathy or muscular dystrophy.
Abstract translation: 本发明提供了包含含有miR-29c的引导链的多核苷酸序列的重组AAV载体以及使用该重组载体来减少或预防患肌营养不良症或肌营养不良症的受试者纤维变性的方法。 本发明还提供了组合疗法，其包括表达miR-29和微肌营养不良蛋白以减少和预防肌营养不良症或肌营养不良症患者中的纤维变性。
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Patent Agency Ranking
公开(公告)号：WO2016057975A2
公开(公告)日：2016-04-14
申请号：PCT/US2015/055022
申请日：2015-10-09
Inventor： MENDELL, Jerry, R. , KASPAR, Brian, K. , AL-ZAIDY, Samiah
IPC: A61K38/18
CPC classification number: A61K48/0075 , A61K38/1709 , A61K48/00 , C07K14/4703 , C12N7/00 , C12N15/86 , C12N2750/14142 , C12N2750/14143 , C12N2750/14171 , C12N2799/025
Abstract: The disclosure relates to methods for treating subjects with musculoskeletal diseases or with muscle wasting not associated with a musculoskeletal disease by gene transfer with recombinant adeno-associated viruses (rAAV) encoding myostatin inhibitors such as follistatin-344. The rAAV are administered prior to development of diffuse muscle fibrosis in a subject, or the rAAV administration avoids regions of muscle fibrosis in a subject.
Abstract translation: 本公开涉及通过用编码肌肉生长抑制素（例如卵泡抑素-444）的重组腺相关病毒（rAAV）进行基因转移来治疗具有肌肉骨骼疾病的受试者或与肌肉骨骼疾病无关的肌肉消耗的方法。 在受试者发展弥漫性肌肉纤维化之前施用rAAV，或者rAAV给药避免受试者的肌肉纤维化区域。
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7.发明申请
公开(公告)号：WO2021035120A1
公开(公告)日：2021-02-25
申请号：PCT/US2020/047339
申请日：2020-08-21
Inventor： RODINO-KLAPAC, Louise , GRIFFIN, Danielle , MENDELL, Jerry, R.
Abstract: Described herein are methods of treating muscular dystrophy in a subject, comprising administration of a recombinant AAV vector AAVrh74.tMCK.hSCGA using a systemic route of administration and at a dose of about 1.0 x 1012 vg/kg to about 5.0 x 1015 vg/kg. Further disclosed are methods of expressing alpha-sarcoglycan gene in a cell or in a subject in need thereof, decreasing a serum CK level, and increasing alpha-sarcoglycan positive fibers in muscle tissue of a subject.
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8.发明申请
公开(公告)号：WO2018170408A1
公开(公告)日：2018-09-20
申请号：PCT/US2018/022881
申请日：2018-03-16
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R.
IPC: C12N15/86 , C12N15/113 , C12N15/09
Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle s including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
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9.发明申请ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRORNA-29 TO TREAT MUSCULAR DYSTROPHY 审中-公开MICRORNA-29治疗肌性肌张力障碍的腺相关病毒载体的研制
公开(公告)号：WO2017181011A1
公开(公告)日：2017-10-19
申请号：PCT/US2017/027630
申请日：2017-04-14
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R. , HELLER, Kristin, N.
IPC: A61K48/00 , C12N15/11 , C12N15/113
Abstract: The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.
Abstract translation: 本发明提供了包含含有miR-29c的引导链的多核苷酸序列的重组AAV载体以及使用该重组载体在患有肌营养不良的受试者中减少或预防纤维化的方法。
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10.发明申请ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND MICRORNA-29 AND THE TREATMENT OF MUSCULAR DYSTROPHY 审中-公开B-SARCOGLYCAN和MICRORNA-29的腺病毒相关病毒载体的输送和肌肉萎缩症的治疗
公开(公告)号：WO2017180976A1
公开(公告)日：2017-10-19
申请号：PCT/US2017/027583
申请日：2017-04-14
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R.
Abstract: Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
Abstract translation: 本文描述了包含含β-半乳聚糖的多核苷酸序列的重组AAV载体以及使用该重组载体来减少或预防肌营养不良症的哺乳动物受试者纤维化的方法。 本文还描述了组合疗法，其包括向患有肌营养不良症的哺乳动物受试者施用表达β-肌聚糖和miR-29c的AAV载体。
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