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公开(公告)号：US20150184165A1
公开(公告)日：2015-07-02
申请号：US14581485
申请日：2014-12-23
IPC分类号： C12N15/113
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/3513
摘要： A method and compound for suppressing an immune response in a mammalian subject, for the treatment or prevention of an autoimmune condition or transplantation rejection are disclosed. The compound is an antisense oligonucleotide analog compound having a targeting sequence complementary to a preprocessed CTLA-4 mRNA region identified by SEQ ID NO: 22 in SEQ ID NO: 1, spanning the splice junction between intron 1 and exon 2 of the preprocessed mRNA of the subject. The compound is effective, when administered to a subject, to form within host cells, a heteroduplex structure (i) composed of the preprocessed CTLA-4 mRNA and the oligonucleotide compound, (ii) characterized by a Tm of dissociation of at least 45° C., and (iii) resulting in an increased ratio of processed mRNA encoding ligand-independent CTLA-4 to processed mRNA encoding full-length CTLA-4.
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公开(公告)号：US08933216B2
公开(公告)日：2015-01-13
申请号：US13940098
申请日：2013-07-11
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/3513
摘要： A method and compound for suppressing an immune response in a mammalian subject, for the treatment or prevention of an autoimmune condition or transplantation rejection are disclosed. The compound is an antisense oligonucleotide analog compound having a targeting sequence complementary to a preprocessed CTLA-4 mRNA region identified by SEQ ID NO: 22 in SEQ ID NO: 1, spanning the splice junction between intron 1 and exon 2 of the preprocessed mRNA of the subject. The compound is effective, when administered to a subject, to form within host cells, a heteroduplex structure (i) composed of the preprocessed CTLA-4 mRNA and the oligonucleotide compound, (ii) characterized by a Tm of dissociation of at least 45° C., and (iii) resulting in an increased ratio of processed mRNA encoding ligand-independent CTLA-4 to processed mRNA encoding full-length CTLA-4.
摘要翻译： 公开了用于抑制哺乳动物受试者的免疫应答的方法和化合物，用于治疗或预防自身免疫病症或移植排斥反应。 该化合物是具有与SEQ ID NO：1中SEQ ID NO：22鉴定的预处理的CTLA-4mRNA区域互补的靶序列的反义寡核苷酸类似物化合物，其跨越了预处理mRNA的内含子1和外显子2之间的剪接连接 主题。 该化合物在给受试者施用时在宿主细胞内形成，由预处理的CTLA-4mRNA和寡核苷酸化合物组成的异源双链体结构（i），（ii）特征在于解离的Tm至少为45° （iii）导致编码与配体无关的CTLA-4的加工的mRNA与编码全长CTLA-4的加工的mRNA的比例增加。
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3.发明申请ANTISENSE COMPOSITIONS AND METHODS FOR MODULATING CONTACT HYPERSENSITIVITY OR CONTACT DERMATITIS 审中-公开反应组合物和调节接触高度或接触剂量的方法公开(公告)号：US20140287983A1
公开(公告)日：2014-09-25
申请号：US14063844
申请日：2013-10-25
IPC分类号： C12N15/113 , A61K45/06 , A61K38/19 , A61K31/7125
CPC分类号： C12N15/1136 , A61K31/7125 , A61K38/19 , A61K45/06 , C12N15/1135 , C12N2310/11 , C12N2310/3513 , C12N2320/32
摘要： Provided are methods and compositions, including topical compositions, for inducing tolerance to a sensitizing agent known to provoke contact hypersensitivity in a subject. Included are methods of topically applying to the subject an effective amount of an antisense composition targeting the start site or splice site of a CFLAR mRNA.
摘要翻译： 提供了用于诱导对已知引起受试者的接触超敏反应的敏化剂的耐受性的方法和组合物，包括局部组合物。 包括对局部施用有效量的靶向CFLAR mRNA的起始位点或剪接位点的反义组合物的方法。
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公开(公告)号：US11020417B2
公开(公告)日：2021-06-01
申请号：US15579497
申请日：2016-06-03
IPC分类号： C12N15/113 , A61K31/7125 , A61P35/00 , A61P37/06 , A61K31/675
摘要： Methods for treatment of lymphocyte-related diseases and conditions, such as cancer and automimmune diseases, are provided. The methods comprise administration of an effective amount of an oligomer to a patient in need thereof, wherein the oligomer comprises, inter alia, at least one intersubunit linkage having the following structure: wherein R1, L1, X, Y and Z are as defined herein.
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公开(公告)号：US10849917B2
公开(公告)日：2020-12-01
申请号：US15578612
申请日：2016-06-01
发明人： Dan V. Mourich
摘要： The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.
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公开(公告)号：US20180161359A1
公开(公告)日：2018-06-14
申请号：US15579497
申请日：2016-06-03
IPC分类号： A61K31/7125 , A61K31/675 , C12N15/113
CPC分类号： A61K31/7125 , A61K31/675 , A61P35/00 , A61P37/06 , C12N15/113 , C12N2310/11 , C12N2310/3233
摘要： Methods for treatment of lymphocyte-related diseases and conditions, such as cancer and automimmune diseases, are provided. The methods comprise administration of an effective amount of an oligomer to a patient in need thereof, wherein the oligomer comprises, inter alia, at least one intersubunit linkage having the following structure: wherein R1, L1, X, Y and Z are as defined herein.
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公开(公告)号：US09487786B2
公开(公告)日：2016-11-08
申请号：US14581485
申请日：2014-12-23
IPC分类号： C12N15/11 , A61K48/00 , C07H21/02 , C07H21/04 , C12N15/113
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/3513
摘要： A method and compound for suppressing an immune response in a mammalian subject, for the treatment or prevention of an autoimmune condition or transplantation rejection are disclosed. The compound is an antisense oligonucleotide analog compound having a targeting sequence complementary to a preprocessed CTLA-4 mRNA region identified by SEQ ID NO: 22 in SEQ ID NO: 1, spanning the splice junction between intron 1 and exon 2 of the preprocessed mRNA of the subject. The compound is effective, when administered to a subject, to form within host cells, a heteroduplex structure (i) composed of the preprocessed CTLA-4 mRNA and the oligonucleotide compound, (ii) characterized by a Tm of dissociation of at least 45° C., and (iii) resulting in an increased ratio of processed mRNA encoding ligand-independent CTLA-4 to processed mRNA encoding full-length CTLA-4.
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公开(公告)号：US11911403B2
公开(公告)日：2024-02-27
申请号：US17080363
申请日：2020-10-26
发明人： Dan V. Mourich
IPC分类号： C07H21/02 , A61K31/70 , C12N15/113 , C07H21/04 , A61P17/00
CPC分类号： A61K31/70 , A61P17/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N2310/11 , C12N2310/3145 , C12N2310/3181 , C12N2310/3231 , C12N2310/3233 , C12N2320/33 , C12N2310/321 , C12N2310/3521 , C12N2310/321 , C12N2310/3525 , C12N2310/322 , C12N2310/3533 , C12N2310/321 , C12N2310/3527
摘要： The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.
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公开(公告)号：US20210283170A1
公开(公告)日：2021-09-16
申请号：US17245456
申请日：2021-04-30
IPC分类号： A61K31/7125 , C12N15/113 , A61P35/00 , A61P37/06 , A61K31/675
摘要： Methods for treatment of lymphocyte-related diseases and conditions, such as cancer and automimmune diseases, are provided. The methods comprise administration of an effective amount of an oligomer to a patient in need thereof, wherein the oligomer comprises, inter alia, at least one intersubunit linkage having the following structure: wherein R1, L1, X, Y and Z are as defined herein.
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公开(公告)号：US20210161922A1
公开(公告)日：2021-06-03
申请号：US17080363
申请日：2020-10-26
发明人： Dan V. Mourich
IPC分类号： A61K31/70 , C07H21/02 , C12N15/113 , C07H21/04 , A61P17/00
摘要： The present disclosure relates to antisense oligomers and related compositions and methods for increasing the expression of functional human type VII collagen and methods for treating dystrophic epidermolysis bullosa and related disorders and relates to inducing exclusion of exon 80 in human type VII collagen mRNA.
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