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公开(公告)号：US20220185821A1
公开(公告)日：2022-06-16
申请号：US17442926
申请日：2020-03-20
发明人： Yousef AL ABED , Lingbo ZHANG
IPC分类号： C07D498/14 , C07D401/06
摘要： This disclosure generally relates to treating anemias. More specifically, the disclosure relates to use of muscarinic acetylcholine receptor subtype 4 antagonists, such as small molecule compounds, to promote self-renewal of burst forming unit erythroid (BFU-E) cells and treat anemias.
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2.发明申请公开(公告)号：US20220088058A1
公开(公告)日：2022-03-24
申请号：US17356147
申请日：2021-06-23
发明人： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC分类号： A61K31/7125 , A61K31/712 , C12Q1/6883 , C12N15/113 , C12Q1/68 , A61K31/713 , A61P43/00
摘要： Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
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公开(公告)号：US20220071981A1
公开(公告)日：2022-03-10
申请号：US17532123
申请日：2021-11-22
申请人： THE CHILDREN'S HOSPITAL OF PHILADELPHIA , THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA , COLD SPRING HARBOR LABORATORY
发明人： Gerd Blobel , Jeremy Grevet , Junwei Shi , Christopher Vakoc
IPC分类号： A61K31/454 , A61P7/06 , A61K31/517 , C12N15/113
摘要： Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed.
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4.发明授权公开(公告)号：US11083745B2
公开(公告)日：2021-08-10
申请号：US16062286
申请日：2016-12-14
发明人： Isabel Aznarez , Huw M. Nash , Adrian Krainer
IPC分类号： C07H21/04 , A61K31/7125 , A61K31/712 , C12Q1/6883 , C12N15/113 , C12Q1/68 , A61K31/713 , A61P43/00
摘要： Provided herein are methods and compositions for treating a subject in need thereof, such as a subject with deficient SYNGAP1 protein or SCN1A protein expression or a subject having AD mental retardation 5 or Dravet syndrome.
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公开(公告)号：US11058709B1
公开(公告)日：2021-07-13
申请号：US15781249
申请日：2016-12-05
发明人： David L. Spector , Sarah Daniela Diermeier , Frank Rigo , C. Frank Bennett , Susan M. Freier , Gayatri Arun , Kung-Chi Chang
IPC分类号： A61K31/713 , A61P35/00 , A61K31/175 , A61K31/712 , A61K31/7125 , C07H21/04 , C12N15/113
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a MaTAR in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate breast cancer in an individual in need.
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公开(公告)号：US20200283762A1
公开(公告)日：2020-09-10
申请号：US16839034
申请日：2020-04-02
发明人： Adrian Krainer , Isabel Aznarez , Tomoki Nomakuchi
IPC分类号： C12N15/11 , A61K31/713 , C12Q1/6883 , A61K31/4245 , A61K31/726
摘要： The present disclosure relates to compositions and methods for inhibiting nonsense-mediated mRNA decay in a gene-specific manner, for example in the treatment of diseases or disorders caused by nonsense mutations.
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公开(公告)号：US10696969B2
公开(公告)日：2020-06-30
申请号：US15949902
申请日：2018-04-10
发明人： Adrian Krainer , Isabel Aznarez
IPC分类号： C12N15/113 , C07K14/805 , C07K14/47 , C12N15/11 , C12Q1/6883
摘要： Provided herein are methods and compositions for increasing production of a target protein or functional RNA by a cell.
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公开(公告)号：US20180371457A1
公开(公告)日：2018-12-27
申请号：US15993090
申请日：2018-05-30
发明人： C. Frank Bennett , Frank Rigo , Adrian R. Krainer , Rahul Sinha
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3233 , C12N2310/3521 , C12N2310/3525 , C12N2310/3533 , C12N2320/33
摘要： The present disclosure provides compounds comprising oligonucleotides complementary to a portion of the IKBKAP gene. Certain such compounds are useful for hybridizing to a portion of the IKBKAP gene, including but not limited to a portion of the IKBKAP gene in a cell. In certain embodiments, such hybridization results in modulation of splicing of the IKBKAP gene. In certain embodiments, the IKBKAP gene includes a mutation that results in defective splicing and a truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in a decrease in the amount of defective splicing and truncated IKAP protein. In certain embodiments, hybridization of oligonucleotides complementary to a portion of the IKBKAP gene results in an increase in the amount of normal splicing and functional, full-length IKAP protein. In certain embodiments, oligonucleotides are used to treat Familial Dysautonomia.
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公开(公告)号：US20180291376A1
公开(公告)日：2018-10-11
申请号：US15818073
申请日：2017-11-20
发明人： Brenda F. Baker , Adrian R. Krainer , Yimin Hua
IPC分类号： C12N15/113 , C12N15/11
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.
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公开(公告)号：US09850495B2
公开(公告)日：2017-12-26
申请号：US14384695
申请日：2013-03-14
申请人： E I DU PONT DE NEMOURS AND COMPANY , COLD SPRING HARBOR LABORATORY , IOWA STATE UNIVERSITY RESEARCH FOUNDATION, INC.
发明人： Stephen M Allen , David Peter Jackson , Mai Komatsu , Michael Pautler , Hajime Sakai , Erik Vollbrecht , Rebecca Weeks
IPC分类号： C12N15/87 , C12N15/82 , A01H5/10 , C07K14/415 , C12Q1/68
CPC分类号： C12N15/8261 , A01H5/10 , C07K14/415 , C12N15/8216 , C12N15/8229 , C12Q1/6895 , C12Q2600/13 , C12Q2600/156 , Y02A40/146
摘要： Methods and compositions for modulating shoot apical meristem size are provided. Methods are provided for modulating the expression of Fea4 sequence in a host plant or plant cell to modulate agronomic characteristics such as altered size and number of organs, including plant seeds.
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