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公开(公告)号：US20120252879A1
公开(公告)日：2012-10-04
申请号：US13395188
申请日：2010-09-10
申请人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
发明人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
IPC分类号： A61K31/711 , A61P25/14
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/345 , C12N2310/346 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
摘要翻译： 本文提供了用于降低动物中亨廷丁蛋白mRNA和蛋白质表达的方法，化合物和组合物。 这些方法，化合物和组合物可用于治疗，预防，延迟或改善亨廷顿病或其症状。
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公开(公告)号：US08906873B2
公开(公告)日：2014-12-09
申请号：US13395188
申请日：2010-09-10
申请人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
发明人： Gene Hung , C. Frank Bennett , Susan M. Freier , Holly Kordasiewicz , Lisa Stanek , Don W. Cleveland , Seng H. Cheng , Lamya Shihabuddin
IPC分类号： A61K48/00 , C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/341 , C12N2310/345 , C12N2310/346 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of huntingtin mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate Huntington's disease, or a symptom thereof.
摘要翻译： 本文提供了用于降低动物中亨廷丁蛋白mRNA和蛋白质表达的方法，化合物和组合物。 这些方法，化合物和组合物可用于治疗，预防，延迟或改善亨廷顿病或其症状。
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3.发明授权公开(公告)号：US08980853B2
公开(公告)日：2015-03-17
申请号：US13380021
申请日：2010-06-17
申请人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： A61K31/70 , C07H21/02 , C07H21/04 , A61K31/7088 , C12N15/113 , C12Q1/68 , A61K31/712
CPC分类号： A61K31/7088 , A61K31/712 , C12N15/113 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. 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.
摘要翻译： 本文公开了用于调节受试者中SMN2 mRNA的剪接的化合物，组合物和方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症（包括脊髓性肌肉萎缩）的药物中的用途。
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4.发明申请公开(公告)号：US20120190728A1
公开(公告)日：2012-07-26
申请号：US13380021
申请日：2010-06-17
申请人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
发明人： C. Frank Bennett , Gene Hung , Frank Rigo , Adrian R. Krainer , Yimin Hua , Marco A. Passini , Lamya Shihabuddin , Seng H. Cheng , Katherine W. Klinger
IPC分类号： A61K31/713 , A61P25/28
CPC分类号： A61K31/7088 , A61K31/712 , C12N15/113 , C12N2310/11 , C12N2320/33
摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. 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.
摘要翻译： 本文公开了用于调节受试者中SMN2 mRNA的剪接的化合物，组合物和方法。 还提供了所公开的化合物和组合物在制备用于治疗疾病和病症（包括脊髓性肌肉萎缩）的药物中的用途。
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5.发明申请MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION 审中-公开DYSTROPHIA MYOTONICA - 蛋白激酶（DMPK）表达的调控公开(公告)号：US20130237585A1
公开(公告)日：2013-09-12
申请号：US13811181
申请日：2011-07-19
申请人： C. Frank Bennett , Susan M. Freier , Robert A. MacLeod , Sanjay K. Pandey , Charles A. Thornton , Thurman Wheeler , Seng H. Cheng , Andrew Leger , Bruce M. Wentworth
发明人： C. Frank Bennett , Susan M. Freier , Robert A. MacLeod , Sanjay K. Pandey , Charles A. Thornton , Thurman Wheeler , Seng H. Cheng , Andrew Leger , Bruce M. Wentworth
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/3181 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525 , C12Y207/11 , C12N2310/322 , C12N2310/3533 , C12N2310/3527
摘要： Provided herein are methods, compounds, and compositions for reducing expression of a DMPK mRNA and protein in an animal. Also provided herein are methods, compounds, and compositions for preferentially reducing CUGexp DMPK RNA, reducing myotonia or reducing spliceopathy in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate type 1 myotonic dystrophy, or a symptom thereof.
摘要翻译： 本文提供了用于减少动物体内DMPK mRNA和蛋白质表达的方法，化合物和组合物。 本文还提供了用于优先减少CUGexp DMPK RNA，减少肌动蛋白或减少动物中的拼接病变的方法，化合物和组合物。 这些方法，化合物和组合物可用于治疗，预防，延迟或改善1型肌强直营养不良症或其症状。
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公开(公告)号：US20110038851A1
公开(公告)日：2011-02-17
申请号：US12600141
申请日：2008-05-16
IPC分类号： A61K38/47 , A61K31/7088 , A61K38/48 , A61K39/395 , A61K38/17 , A61P25/28 , A61P25/16 , A61P25/14 , A61P25/00 , A61P43/00 , C12N9/24 , C12N9/48 , C07H21/00 , C07K14/47
CPC分类号： A61K38/4873 , A61K31/00 , A61K31/047 , A61K31/4353 , A61K31/436 , A61K31/7088 , A61K38/13 , A61K38/1709 , A61K38/47 , A61K38/488 , A61K45/06 , A61K48/005 , A61K2300/00 , C12Y302/01045 , C12Y304/22041 , C12Y304/23005
摘要： This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.
摘要翻译： 本发明一般涉及治疗未临床诊断患有溶酶体贮积病的受试者的突触核蛋白病，以及制备药物和筛选方法的相关方法。
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公开(公告)号：US08454954B2
公开(公告)日：2013-06-04
申请号：US12600141
申请日：2008-05-16
IPC分类号： A61K38/47
CPC分类号： A61K38/4873 , A61K31/00 , A61K31/047 , A61K31/4353 , A61K31/436 , A61K31/7088 , A61K38/13 , A61K38/1709 , A61K38/47 , A61K38/488 , A61K45/06 , A61K48/005 , A61K2300/00 , C12Y302/01045 , C12Y304/22041 , C12Y304/23005
摘要： This invention relates generally to treating synucleinopathies in subjects that are not clinically diagnosed with a lysosomal storage disease, as well as associated methods of making medicaments and screening methods.
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公开(公告)号：US20090123451A1
公开(公告)日：2009-05-14
申请号：US12187896
申请日：2008-08-07
CPC分类号： A61K31/00 , A61K9/0024
摘要： Neurological diseases, including lysosomal storage diseases, can be successfully treated using intraventricular delivery of the therapeutic agents to bypass the blood-brain barrier. Similarly, diagnostic agents and anesthetic agents can be delivered to the brain in this manner. The administration can be performed slowly to achieve maximum effect. Such administration permits greater penetration of distal portions of the brain.
摘要翻译： 可以使用治疗剂的脑室内递送绕过血脑屏障来成功地治疗包括溶酶体贮积病的神经疾病。 类似地，诊断剂和麻醉剂可以以这种方式递送到大脑。 可以缓慢执行给药以达到最大效果。 这样的施用允许脑的远端部分更大的穿透。
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公开(公告)号：US08926967B2
公开(公告)日：2015-01-06
申请号：US12175610
申请日：2008-07-18
IPC分类号： A61K38/46
CPC分类号： A61K38/465 , A61K9/0019 , A61K38/47 , C12Y301/04012 , C12Y301/06013 , C12Y302/0102 , C12Y302/01045 , C12Y302/01076 , C12Y304/14
摘要： Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
摘要翻译： 可以使用致病性疾病缺陷的酶的心室内递送来成功地治疗溶酶体贮积病。 可以缓慢执行给药以达到最大效果。 令人惊讶的是，在血脑屏障的两侧看到效果，使其成为影响脑和内脏器官的溶酶体储存疾病的理想递送手段。
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公开(公告)号：US20090130079A1
公开(公告)日：2009-05-21
申请号：US12175610
申请日：2008-07-18
申请人： James Dodge , Marco Passini , Lamya Shihabuddin , Seng H. Cheng
发明人： James Dodge , Marco Passini , Lamya Shihabuddin , Seng H. Cheng
IPC分类号： A61K38/46
CPC分类号： A61K38/465 , A61K9/0019 , A61K38/47 , C12Y301/04012 , C12Y301/06013 , C12Y302/0102 , C12Y302/01045 , C12Y302/01076 , C12Y304/14
摘要： Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
摘要翻译： 可以使用致病性疾病缺陷的酶的心室内递送来成功地治疗溶酶体贮积病。 可以缓慢执行给药以达到最大效果。 令人惊讶的是，在血脑屏障的两侧看到效果，使其成为影响脑和内脏器官的溶酶体储存疾病的理想递送手段。
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