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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：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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3.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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公开(公告)号：US10004814B2
公开(公告)日：2018-06-26
申请号：US15035950
申请日：2014-11-10
发明人： Marija Tadin-Strapps , Tayeba Khan , Walter Richard Strapps , Laura Sepp-Lorenzino , Vasant Jadhav , Duncan Brown
IPC分类号： C12N15/11 , A61K48/00 , A61K31/713 , C12N15/113 , A61K47/54
CPC分类号： A61K48/0058 , A61K31/713 , A61K47/543 , A61K47/554 , C12N15/1136 , 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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5.发明申请SYSTEMIC DELIVERY OF MYOSTATIN SHORT INTERFERING NUCLEIC ACIDS (siNA) CONJUGATED TO A LIPOPHILIC MOIETY 有权将肌松素干细胞干细胞（siNA）系统传递给一个亲水的小鼠公开(公告)号：US20160256570A1
公开(公告)日：2016-09-08
申请号：US15035950
申请日：2014-11-10
发明人： Marija Tadin-Strapps , Tayeba Khan , Walter Richard Strapps , Laura Sepp-Lorenzino , Vasant Jadhav , Duncan Brown
IPC分类号： A61K48/00 , C12N15/113 , A61K47/48
CPC分类号： A61K48/0058 , A61K31/713 , A61K47/543 , A61K47/554 , C12N15/1136 , 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.
摘要翻译： 本发明提供了包括体内递送能够介导RNA干扰并降低肌生成抑制素表达的小核酸分子的方法，其中通过全身给药将小核酸分子引入受试者。 具体而言，本发明涉及包括体内递送针对受试者表达的肌生长抑制素基因的短干扰核酸（siNA）分子的方法，其中所述siNA分子与亲脂部分如胆固醇结合。 根据所公开的方法递送的肌生成抑制素siNA缀合物可用于调节肌生成抑制素的体内表达，增加肌肉质量和/或增强肌肉性能。 还公开了所公开的方法的使用用于治疗导致肌肉受到不利影响的病症的肌肉骨骼疾病或病症和/或疾病或病症。
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6.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：US10729787B2
公开(公告)日：2020-08-04
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 , C12N15/113 , 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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