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公开(公告)号：US11975056B2
公开(公告)日：2024-05-07
申请号：US17306376
申请日：2021-05-03
CPC分类号： A61K38/51 , A61K35/76 , A61K48/005 , A61P25/14 , C12N7/00 , C12N9/88 , C12N15/86 , C12Y401/01028 , C12N2750/14121 , C12N2750/14143 , C12N2800/22 , C12N2830/42 , C12N2830/50
摘要： The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.
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2.发明申请公开(公告)号：US20170321217A1
公开(公告)日：2017-11-09
申请号：US15585410
申请日：2017-05-03
发明人： Chi-Ren TSAI , Ching-Shiang CHI , Hsiu-Fen LEE
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12N2310/11 , C12N2320/33 , C12Y401/01028
摘要： This present invention discloses an antisense oligonucleotide for splicing adjustment of mutant dopa decarboxylase gene which is complementary to SEQ ID NO: 1. This antisense oligonucleotide can modulate alternative splicing site of mutant dopa decarboxylase gene. It is helpful to research and develop drug to treat AADC deficiency symptom. This present invention also discloses a method to use said antisense oligonucleotide in vitro.
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公开(公告)号：US08309355B2
公开(公告)日：2012-11-13
申请号：US12384935
申请日：2009-04-10
申请人： Krys Bankiewicz , Janet Cunningham
发明人： Krys Bankiewicz , Janet Cunningham
CPC分类号： A61K31/198 , A01N63/00 , A61K9/5184 , A61K35/761 , A61K38/45 , A61K38/51 , A61K45/06 , A61K47/6901 , A61K48/00 , A61K48/0025 , A61K48/0075 , A61K51/00 , A61K51/0402 , C12N7/00 , C12N15/63 , C12N15/86 , C12N2750/14142 , C12N2750/14143 , C12Y401/01028
摘要： Methods of delivering viral vectors, particularly recombinant AAV virions, to the central nervous system (CNS) are provided for the treatment of CNS disorders, particularly those disorders which involve the neurotransmitter dopamine. The methods entail providing rAAV virions that comprise a transgene encoding aromatic amino acid decarboxylase (AADC) and administering the virions to the brain of a mammal using a non-manual pump.
摘要翻译： 提供将病毒载体，特别是重组AAV病毒粒子递送至中枢神经系统（CNS）的方法用于治疗CNS疾病，特别是涉及神经递质多巴胺的那些疾病。 所述方法需要提供包含编码芳族氨基酸脱羧酶（AADC）的转基因的rAAV病毒粒子，并使用非手动泵将病毒粒子施用于哺乳动物的脑部。
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公开(公告)号：US07052913B2
公开(公告)日：2006-05-30
申请号：US10838423
申请日：2004-05-04
申请人： John W. Babich , Jon Zubieta , Grant Bonavia
发明人： John W. Babich , Jon Zubieta , Grant Bonavia
IPC分类号： A61K9/14
CPC分类号： A61K9/0024 , A61K9/0085 , A61K38/44 , A61K38/51 , A61K47/6903 , C12Q1/00 , C12Y401/01028 , G01N33/5436 , G01N33/552 , A61K2300/00
摘要： In one aspect, biocompatible matrices such as sol-gels encapsulating a reaction center may be administered to a subject for conversion of prodrugs into biologically active agents. In certain embodiments, the biocompatible matrices of the present invention are sol-gels. In one embodiment, the enzyme L-amino acid decarboxylase is encapsulated and implanted in the brain to convert L-dopa to dopamine for treatment of Parkinson's disease.
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公开(公告)号：US11931338B2
公开(公告)日：2024-03-19
申请号：US17946508
申请日：2022-09-16
IPC分类号： A61K31/4045 , A61P25/00 , C07D209/16 , C12N9/10 , C12N9/88 , C12N15/52 , C12P17/10
CPC分类号： A61K31/4045 , A61P25/00 , C07D209/16 , C12N9/1007 , C12N9/1029 , C12N9/88 , C12N15/52 , C12P17/10 , C12Y203/01087 , C12Y401/01028 , C12Y402/0102
摘要： Disclosed are novel nitrilated psilocybin derivative compounds and pharmaceutical and recreational drug formulations containing the same. The compounds may be produced by reacting a reactant psilocybin derivative with a nitrile-group containing compound.
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公开(公告)号：US20180066279A9
公开(公告)日：2018-03-08
申请号：US15306543
申请日：2015-04-24
发明人： Guangping Gao , Phillip D. Zamore , Shaoyong Li
IPC分类号： C12N15/86 , C12N15/113 , C12N7/00 , C12N15/117 , A61K48/00 , A61K38/51 , A61K38/17 , A61K38/44 , A61K38/28 , A61K38/48 , C07K16/28 , C07K16/32 , C07K16/22 , C07K16/10
CPC分类号： C12N15/86 , A61K38/1709 , A61K38/28 , A61K38/446 , A61K38/4846 , A61K38/51 , A61K48/0008 , C07K16/10 , C07K16/22 , C07K16/2875 , C07K16/2887 , C07K16/32 , C12N7/00 , C12N15/113 , C12N15/117 , C12N2310/141 , C12N2310/17 , C12N2320/30 , C12N2330/51 , C12N2710/10322 , C12N2710/10332 , C12N2750/14143 , C12N2760/00042 , C12N2830/008 , C12Y115/01001 , C12Y304/21022 , C12Y401/01028 , C12N2310/113 , C12N2310/13
摘要： The disclosure in some aspects, relates to nucleic acids, compositions and kits useful for gene therapy with reduced immune response to transgene products.
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公开(公告)号：US20170333538A1
公开(公告)日：2017-11-23
申请号：US15524986
申请日：2015-11-05
CPC分类号： A61K38/51 , A61K35/76 , A61K48/005 , A61P25/14 , C12N7/00 , C12N9/88 , C12N15/86 , C12N2750/14121 , C12N2750/14143 , C12N2800/22 , C12N2830/42 , C12N2830/50 , C12Y401/01028
摘要： The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.
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8.发明申请公开(公告)号：US20170204375A1
公开(公告)日：2017-07-20
申请号：US15321504
申请日：2015-06-26
发明人： Domenico Accili , Ryotaro Bouchi
IPC分类号： C12N5/071 , A61K35/38 , C12N15/113 , A61L27/36 , C07K14/62 , A61K38/28 , C07K16/40 , A61L27/38
CPC分类号： C12N5/0679 , A61K35/38 , A61K38/28 , A61L27/3687 , A61L27/3813 , A61L27/383 , A61L27/3882 , A61L2430/22 , C07K14/62 , C07K16/40 , C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/12 , C12N2310/14 , C12N2310/531 , C12N2506/23 , C12Y114/16004 , C12Y401/01028
摘要： Disclosed herein are methods involving the targeting of 5HT biosynthesis in gut insulin-negative cells to convert them into insulin-positive cells. Also disclosed are methods for treating a disease or disorder in a mammal, preferably a human, associated with impaired pancreatic endocrine function, by administering a therapeutically effective amount of an enumerated active agent that reduces the expression, biosynthesis, signaling or biological activity of serotonin or increases its degradation, wherein administering comprises delivering the agent to Gut Ins− cells in the mammal. Other embodiments of the method are directed to therapy wherein an agent that significantly reduces FOXO1 expression, biosynthesis, signaling or biological activity or increases its degradation is administered in addition to the agent that reduces serotonin, or alternatively an agent that reduces FOXO1 expression is targeted to serotonin-positive gut enteroendocrine cells.
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公开(公告)号：US20130101510A1
公开(公告)日：2013-04-25
申请号：US13647155
申请日：2012-10-08
申请人： Krys BANKIEWICZ , Janet Cunningham
发明人： Krys BANKIEWICZ , Janet Cunningham
IPC分类号： A61K31/198 , A61K45/06 , A61K35/76
CPC分类号： A61K31/198 , A01N63/00 , A61K9/5184 , A61K35/761 , A61K38/45 , A61K38/51 , A61K45/06 , A61K47/6901 , A61K48/00 , A61K48/0025 , A61K48/0075 , A61K51/00 , A61K51/0402 , C12N7/00 , C12N15/63 , C12N15/86 , C12N2750/14142 , C12N2750/14143 , C12Y401/01028
摘要： Methods of delivering viral vectors, particularly recombinant AAV virions, to the CNS are provided. Also provided are methods of treating Parkinson's Disease.
摘要翻译： 提供将病毒载体，特别是重组AAV病毒粒子递送至CNS的方法。 还提供治疗帕金森病的方法。
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公开(公告)号：US20100104537A1
公开(公告)日：2010-04-29
申请号：US12384935
申请日：2009-04-10
申请人： Krys Bankiewicz , Janet Cunningham
发明人： Krys Bankiewicz , Janet Cunningham
IPC分类号： A61K35/76
CPC分类号： A61K31/198 , A01N63/00 , A61K9/5184 , A61K35/761 , A61K38/45 , A61K38/51 , A61K45/06 , A61K47/6901 , A61K48/00 , A61K48/0025 , A61K48/0075 , A61K51/00 , A61K51/0402 , C12N7/00 , C12N15/63 , C12N15/86 , C12N2750/14142 , C12N2750/14143 , C12Y401/01028
摘要： Methods of delivering viral vectors, particularly recombinant AAV virions, to the CNS are provided. Also provided are methods of treating Parkinson's Disease.
摘要翻译： 提供将病毒载体，特别是重组AAV病毒粒子递送至CNS的方法。 还提供治疗帕金森病的方法。
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