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公开(公告)号：US11529428B2
公开(公告)日：2022-12-20
申请号：US16910237
申请日：2020-06-24
发明人： Marija Tadin-Strapps , Tayeba Khan , Walter Richard Strapps , Laura Sepp-Lorenzino , Vasant R. Jadhav , Duncan Brown
IPC分类号： C12N15/113 , A61K48/00 , A61K31/713 , A61K47/54
摘要： The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
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公开(公告)号：US20180105812A1
公开(公告)日：2018-04-19
申请号：US15684902
申请日：2017-08-23
发明人： Brian Allen Carr , Vasant R. Jadhav , Denise M. Kenski , David M. Tellers , Aarron T. Willingham
IPC分类号： C12N15/113 , C12N15/11
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
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公开(公告)号：US20150299696A1
公开(公告)日：2015-10-22
申请号：US14398300
申请日：2013-04-26
申请人： Brian Allen Carr , Vasant R. Jadhav , Denise M. Kenski , David M. Tellers , Aarron T. Willingham , Sirna Therapeutics, Inc.
发明人： Brian Allen Carr , Vasant R. Jadhav , Denise M. Kenski , David M. Tellers , Aarron T. Willingham
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/3231 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2320/32 , C12N2320/51 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
摘要翻译： 本发明涉及用于研究，诊断和治疗响应于基因表达和/或活性的调节和/或调节基因表达途径的性状，疾病和病症的化合物，组合物和方法。 具体地说，本发明涉及包括小核酸分子的双链核酸分子，例如能够介导或介导针对靶基因表达的RNA干扰（RNAi）的短干扰核酸（siNA）分子。
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公开(公告)号：US10689648B2
公开(公告)日：2020-06-23
申请号：US15684902
申请日：2017-08-23
发明人： Brian Allen Carr , Vasant R. Jadhav , Denise M. Kenski , David M. Tellers , Aarron T. Willingham
IPC分类号： C07H21/04 , C12N15/113 , C12N15/11
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
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公开(公告)号：US10508277B2
公开(公告)日：2019-12-17
申请号：US15977487
申请日：2018-05-11
发明人： Vasant R. Jadhav
IPC分类号： C12N15/11 , C12N15/113
摘要： The present invention relates to a multifunctional short interfering nucleic acid (siNA) having a structure of Formula MF-III: wherein each X, X′, Y, and Y′ is independently an oligonucleotide of length about 15 nucleotides to about 50 nucleotides; X comprises a nucleotide sequence that is complementary to a nucleotide sequence present in region Y′; X′ comprises a nucleotide sequence that is complementary to a nucleotide sequence present in region Y; one or more of X, X′, Y, and Y′ is independently complementary to a first, second, third, or fourth target sequence, respectively, or a portion thereof; and W represents a nucleotide or non-nucleotide linker that connects sequences Y′ and Y, wherein the siNA directs cleavage of the first, second, third, and/or fourth target sequence via RNA interference.
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公开(公告)号：US20210008226A1
公开(公告)日：2021-01-14
申请号：US16910237
申请日：2020-06-24
发明人： Marija Tadin-Strapps , Tayeba Khan , Walter Richard Strapps , Laura Sepp-Lorenzino , Vasant R. Jadhav , Duncan Brown
IPC分类号： A61K48/00 , A61K31/713 , C12N15/113 , A61K47/54
摘要： The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
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7.发明申请公开(公告)号：US20190060488A1
公开(公告)日：2019-02-28
申请号：US15994450
申请日：2018-05-31
发明人： Marija Tadin-strapps , Tayeba Khan , Walter Richard Strapps , Laura Sepp-Lorenzino , Vasant R. Jadhav , Duncan Brown
IPC分类号： A61K48/00 , A61K47/54 , C12N15/113 , A61K31/713
摘要： The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
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8.发明申请公开(公告)号：US20180346913A1
公开(公告)日：2018-12-06
申请号：US15977487
申请日：2018-05-11
发明人： Vasant R. Jadhav
IPC分类号： C12N15/113 , C12N15/11
摘要： The present invention relates to a multifunctional short interfering nucleic acid (siNA) having a structure of Formula wherein each X, X′, Y, and Y′ is independently an oligonucleotide of length about 15 nucleotides to about 50 nucleotides; X comprises a nucleotide sequence that is complementary to a nucleotide sequence present in region Y′; X′ comprises a nucleotide sequence that is complementary to a nucleotide sequence present in region Y; one or more of X, X′, Y, and Y′ is independently complementary to a first, second, third, or fourth target sequence, respectively, or a portion thereof; and W represents a nucleotide or non-nucleotide linker that connects sequences Y′ and Y, wherein the siNA directs cleavage of the first, second, third, and/or fourth target sequence via RNA interference.
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9.发明公开公开(公告)号：US20230144623A1
公开(公告)日：2023-05-11
申请号：US18054264
申请日：2022-11-10
发明人： Marija Tadin-Strapps , Tayeba Khan , Walter Richard Strapps , Laura Sepp-Lorenzino , Vasant R. Jadhav , Duncan Brown
IPC分类号： A61K48/00 , A61K31/713 , C12N15/113 , A61K47/54
CPC分类号： A61K48/0058 , A61K31/713 , C12N15/1136 , A61K47/543 , A61K47/554 , C12N2310/14 , C12N2310/3515
摘要： The present invention provides methods comprising the in vivo delivery of small nucleic acid molecules capable of mediating RNA interference and reducing the expression of myostatin, wherein the small nucleic acid molecules are introduced to a subject by systemic administration. Specifically, the invention relates to methods comprising the in vivo delivery of short interfering nucleic acid (siNA) molecules that target a myostatin gene expressed by a subject, wherein the siNA molecule is conjugated to a lipophilic moiety, such as cholesterol. The myostatin siNA conjugates that are delivered as per the methods disclosed are useful to modulate the in vivo expression of myostatin, increase muscle mass and/or enhance muscle performance. Use of the disclosed methods is further indicated for treating musculoskeletal diseases or disorders and/or diseases or disorders that result in conditions in which muscle is adversely affected.
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公开(公告)号：US11530407B2
公开(公告)日：2022-12-20
申请号：US16793790
申请日：2020-02-18
发明人： Brian Allen Carr , Vasant R. Jadhav , Denise M. Kenski , David M. Tellers , Aarron T. Willingham
IPC分类号： C07H21/04 , C12N15/113 , C12N15/11
摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of gene expression and/or activity, and/or modulate a gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against target gene expression.
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