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11.发明授权Compounds and methods for improving cellular uptake of oligomeric compounds 有权用于改善寡聚化合物的细胞摄取的化合物和方法公开(公告)号：US08318496B2
公开(公告)日：2012-11-27
申请号：US12681593
申请日：2008-10-06
Applicant: Erich Koller , C. Frank Bennett
Inventor： Erich Koller , C. Frank Bennett
CPC classification number: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3517 , C12N2320/10 , C12N2320/32 , C12Q1/025
Abstract: The present invention provides method of optimizing the efficacy and potency of antisense drugs. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipeints for improved cellular uptake.
Abstract translation: 本发明提供了优化反义药物的功效和效力的方法。 在某些实施方案中，本发明提供了用于确定有利的寡核苷酸特征和突变体用于改善细胞摄取的测定法。
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公开(公告)号：US08268803B2
公开(公告)日：2012-09-18
申请号：US12519975
申请日：2007-12-17
Applicant: Frank Bennett , Srikanth Venkatraman , F. George Njoroge , Kevin X. Chen , Neng-Yang Shih , Stuart B. Rosenblum , Joseph A. Kozlowski
Inventor： Frank Bennett , Srikanth Venkatraman , F. George Njoroge , Kevin X. Chen , Neng-Yang Shih , Stuart B. Rosenblum , Joseph A. Kozlowski
IPC: A61K31/437 , C07D209/04 , C07D243/36
CPC classification number: C07D513/04 , C07D401/04 , C07D471/04 , C07D491/04
Abstract: The present invention relates to 5,6-ring annulated indole derivatives of the formula (I), compositions comprising at least one 5,6-ring annulated indole derivatives, and methods of using the 5,6-ring annulated indole derivatives for treating or preventing a viral infection or a virus-related disorder in a patient.
Abstract translation: 本发明涉及式（I）的5,6环环状吲哚衍生物，包含至少一种5,6环环状吲哚衍生物的组合物，以及使用5,6环环状吲哚衍生物用于治疗或 预防患者的病毒感染或病毒相关疾病。
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公开(公告)号：US08211867B2
公开(公告)日：2012-07-03
申请号：US12740211
申请日：2008-10-29
Applicant: C. Frank Bennett , Ayelet Chajut , Christine Esau , Eric G. Marcusson , Noga Yerushalmi
Inventor： C. Frank Bennett , Ayelet Chajut , Christine Esau , Eric G. Marcusson , Noga Yerushalmi
IPC: C12N15/11
CPC classification number: C12N15/113 , A61K31/407 , A61K31/44 , A61K31/4745 , A61K31/513 , A61K31/555 , A61K31/704 , A61K31/7048 , A61K31/7068 , A61K31/7088 , A61K31/7105 , A61K45/06 , C12N2310/113 , C12N2310/141 , C12N2310/315 , C12N2310/321 , C12N2310/3341 , C12N2310/346 , C12N2310/3525
Abstract: Provided herein are methods for the treatment of liver cancer. These methods encompass the administration of a compound comprising a modified oligonucleotide, wherein the modified oligonucleotide is targeted to a miRNA. Also provided herein are compositions for the treatment of liver cancer. Such compositions include compounds comprising a modified oligonucleotide, wherein the modified oligonucleotide is targeted to a miRNA. Certain miRNAs have been identified as overexpressed in liver cancer, such as, for example, hepatocellular carcinoma, and are thus selected for targeting by modified oligonucleotides. Further, certain miRNAs have been identified as overexpressed in hepatocellular carcinoma cells exposed to dioxin, and are thus selected for targeting by modified oligonucleotides. Antisense inhibition of certain of these miRNAs has been found to inhibit cell proliferation and induce apoptosis.
Abstract translation: 本文提供了用于治疗肝癌的方法。 这些方法包括施用包含修饰的寡核苷酸的化合物，其中修饰的寡核苷酸靶向于miRNA。 本文还提供了用于治疗肝癌的组合物。 这样的组合物包括包含修饰的寡核苷酸的化合物，其中修饰的寡核苷酸靶向于miRNA。 某些miRNA已被鉴定为在肝癌中过度表达，例如肝细胞癌，因此被选择用修饰的寡核苷酸靶向。 此外，某些miRNA已被鉴定为在暴露于二恶英的肝细胞癌细胞中过表达，因此被选择用修饰的寡核苷酸靶向。 已经发现某些这些miRNA的反义抑制抑制细胞增殖并诱导细胞凋亡。
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公开(公告)号：US20120157514A1
公开(公告)日：2012-06-21
申请号：US13412307
申请日：2012-03-05
Applicant: Christine Esau , Bridget Lollo , C. Frank Bennett , Susan M. Freier , Richard H. Griffey , Brenda F. Baker , Timothy A. Vickers , Eric G. Marcusson , Erich Koller , Eric E. Swayze , Ravi Jain , Balkrishen Bhat , Eigen Peralta
Inventor： Christine Esau , Bridget Lollo , C. Frank Bennett , Susan M. Freier , Richard H. Griffey , Brenda F. Baker , Timothy A. Vickers , Eric G. Marcusson , Erich Koller , Eric E. Swayze , Ravi Jain , Balkrishen Bhat , Eigen Peralta
IPC: A61K31/712 , C12Q1/68 , A61P37/00 , C12N5/07
CPC classification number: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/3181 , C12N2310/32 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/11 , C12N2320/30 , C12N2320/50 , C12N2310/3525
Abstract: Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.
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公开(公告)号：US20120149757A1
公开(公告)日：2012-06-14
申请号：US13264353
申请日：2010-04-13
Applicant: Adrian R. Krainer , Yimin Hua , Brenda F. Baker , Susan M. Freier , C. Frank Bennett
Inventor： Adrian R. Krainer , Yimin Hua , Brenda F. Baker , Susan M. Freier , C. Frank Bennett
CPC classification number: C07H21/00
Abstract: 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.
Abstract translation: 本文公开了用于调节细胞，组织或动物中SMN2mRNA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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Patent Agency Ranking
公开(公告)号：US08153602B1
公开(公告)日：2012-04-10
申请号：US09315292
申请日：1999-05-20
Applicant: Clarence Frank Bennett , David J. Ecker , Phillip Dan Cook
Inventor： Clarence Frank Bennett , David J. Ecker , Phillip Dan Cook
CPC classification number: A61K48/0075
Abstract: The present invention relates to compositions and methods for the pulmonary delivery of nucleic acids, particularly oligonucleotides. In one preferred embodiment, the compositions and methods of the invention are utilized to effect the pulmonary delivery of an antisense oligonucleotide to an animal in order to modulate the expression of a gene in the animal for investigative, therapeutic or prophylactic purposes.
Abstract translation: 本发明涉及用于肺部递送核酸，特别是寡核苷酸的组合物和方法。 在一个优选的实施方案中，本发明的组合物和方法用于将动物的反义寡核苷酸肺部递送至动物，以调节动物中基因的表达用于调查，治疗或预防目的。
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公开(公告)号：US08106025B2
公开(公告)日：2012-01-31
申请号：US12346940
申请日：2008-12-31
Applicant: C. Frank Bennett , Susan M. Freier , Richard H. Griffey
Inventor： C. Frank Bennett , Susan M. Freier , Richard H. Griffey
CPC classification number: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/113 , C12N2310/14 , C12N2310/141 , C12N2310/31 , C12N2310/315 , C12N2310/317 , C12N2310/3181 , C12N2310/32 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/11 , C12N2320/30 , C12N2320/50 , C12N2310/3525
Abstract: Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding RNAs. Methods of using these compounds for modulation of small non-coding RNAs as well as downstream targets of these RNAs and for diagnosis and treatment of disease associated with small non-coding RNAs are also provided.
Abstract translation: 提供化合物，组合物和方法用于调节小非编码RNA的表达和功能。 组合物包含靶向小的非编码RNA的寡聚化合物。 还提供了使用这些化合物调节小非编码RNA以及这些RNA的下游靶标以及用于诊断和治疗与小非编码RNA相关的疾病的方法。
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公开(公告)号：US20110124591A1
公开(公告)日：2011-05-26
申请号：US12742652
申请日：2008-11-13
Applicant: C. Frank Bennett
Inventor： C. Frank Bennett
IPC: A61K31/7088 , C12N15/85
CPC classification number: C12N15/113 , A61K31/7115 , C12N15/111 , C12N2320/51
Abstract: The present invention provides compounds and methods for modulating expression of a protein, including, but not limited to, modulating splicing of a pre-mRNA to modulate the amount of one or more variants of a protein.
Abstract translation: 本发明提供了用于调节蛋白质表达的化合物和方法，包括但不限于调节前mRNA的剪接以调节蛋白质的一种或多种变体的量。
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公开(公告)号：US20110039334A1
公开(公告)日：2011-02-17
申请号：US12065250
申请日：2006-08-29
Applicant: C. Frank Bennett , Nicholas M. Dean , Ryszard M. Kole , Casey C. Kopczynski
Inventor： C. Frank Bennett , Nicholas M. Dean , Ryszard M. Kole , Casey C. Kopczynski
CPC classification number: C12N15/113 , C12N15/111 , C12N15/1135 , C12N2310/321 , C12N2310/322 , C12N2310/346 , C12N2320/33 , C12N2310/3525 , C12N2310/3533 , C12N2310/3531
Abstract: Disclosed herein are compounds, compositions and methods for modulating splicing of a selected target mRNA. Further provided are uses of the disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Methods of enhancing cellular uptake, modulating tissue distribution and enhancing pharmacological activity of RNase H-independent antisense oligonucleotides are also provided.
Abstract translation: 本文公开了用于调节选定靶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的药物中的用途。 还提供了增强细胞摄取，调节组织分布和增强RNase H非依赖性反义寡核苷酸的药理活性的方法。
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20.发明申请公开(公告)号：US20100292304A1
公开(公告)日：2010-11-18
申请号：US12681593
申请日：2008-10-06
Applicant: Erich Koller , C. Frank Bennett
Inventor： Erich Koller , C. Frank Bennett
IPC: A61K31/7088 , C12Q1/68 , A61P1/16
CPC classification number: C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/3517 , C12N2320/10 , C12N2320/32 , C12Q1/025
Abstract: The present invention provides method of optimizing the efficacy and potency of antisense drugs. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipeints for improved cellular uptake.
Abstract translation: 本发明提供了优化反义药物的功效和效力的方法。 在某些实施方案中，本发明提供了用于确定有利的寡核苷酸特征和突变体用于改善细胞摄取的测定法。
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