公开(公告)号：US20110124591A1

公开(公告)日：2011-05-26

申请号：US12742652

申请日：2008-11-13

申请人： C. Frank Bennett

发明人： C. Frank Bennett

IPC分类号： A61K31/7088 ,  C12N15/85

CPC分类号： C12N15/113 ,  A61K31/7115 ,  C12N15/111 ,  C12N2320/51

摘要： 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.

摘要翻译： 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。

公开(公告)号：US20110039334A1

公开(公告)日：2011-02-17

申请号：US12065250

申请日：2006-08-29

申请人： C. Frank Bennett ,  Nicholas M. Dean ,  Ryszard M. Kole ,  Casey C. Kopczynski

发明人： C. Frank Bennett ,  Nicholas M. Dean ,  Ryszard M. Kole ,  Casey C. Kopczynski

IPC分类号： C12N5/071 ,  C07H21/00

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N15/1135 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/346 ,  C12N2320/33 ,  C12N2310/3525 ,  C12N2310/3533 ,  C12N2310/3531

摘要： 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.

摘要翻译： 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。

公开(公告)号：US20100292304A1

公开(公告)日：2010-11-18

申请号：US12681593

申请日：2008-10-06

申请人： Erich Koller ,  C. Frank Bennett

发明人： Erich Koller ,  C. Frank Bennett

IPC分类号： A61K31/7088 ,  C12Q1/68 ,  A61P1/16

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3517 ,  C12N2320/10 ,  C12N2320/32 ,  C12Q1/025

摘要： 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.

摘要翻译： 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。

公开(公告)号：US20100267814A1

公开(公告)日：2010-10-21

申请号：US12740211

申请日：2008-10-29

申请人： C. Frank Bennett ,  Ayelet Chajut ,  Christine Esau ,  Eric G. Marcusson ,  Noga Yerushalmi

发明人： C. Frank Bennett ,  Ayelet Chajut ,  Christine Esau ,  Eric G. Marcusson ,  Noga Yerushalmi

IPC分类号： A61K31/7125 ,  A61K31/7088 ,  A61K31/712 ,  A61K31/7115 ,  A61P35/00

CPC分类号： 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

摘要： 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.

摘要翻译： 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。

公开(公告)号：US20090298174A1

公开(公告)日：2009-12-03

申请号：US12345780

申请日：2008-12-30

申请人： 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

发明人： 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分类号： C12N5/00

CPC分类号： 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

摘要： 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.

公开(公告)号：US07339051B2

公开(公告)日：2008-03-04

申请号：US10831901

申请日：2004-04-26

申请人： Stanley T. Crooke ,  David J. Ecker ,  Rangarajan Sampath ,  Susan M. Freier ,  Christian Massire ,  Steven A. Hofstadler ,  Kristin Sannes Lowery ,  Eric E. Swayze ,  Brenda F. Baker ,  C. Frank Bennett

发明人： Stanley T. Crooke ,  David J. Ecker ,  Rangarajan Sampath ,  Susan M. Freier ,  Christian Massire ,  Steven A. Hofstadler ,  Kristin Sannes Lowery ,  Eric E. Swayze ,  Brenda F. Baker ,  C. Frank Bennett

IPC分类号： C07H21/02 ,  C07H21/04

CPC分类号： C12N15/1131 ,  C07K16/10 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525

摘要： The present invention provides design and synthesis of oligomeric compounds and compositions that can be administered to reduce the activity of SARS virus in vivo or in vitro, to prevent or treat SARS virus-associated disease, to detect AARS virus, and to diagnose SARS virus-associated diseases.

摘要翻译： 本发明提供可以施用以降低SARS病毒在体内或体外的活性，预防或治疗SARS病毒相关疾病，检测AARS病毒和诊断SARS病毒的寡聚化合物和组合物的设计和合成， 相关疾病

公开(公告)号：US07132530B2

公开(公告)日：2006-11-07

申请号：US10633843

申请日：2003-08-04

申请人： C. Frank Bennett ,  Kenneth Dobie

发明人： C. Frank Bennett ,  Kenneth Dobie

IPC分类号： C07H21/04

CPC分类号： C12N15/1137 ,  A61K38/00 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525 ,  C12Y115/01001 ,  Y02P20/582

摘要： Antisense compounds, compositions and methods are provided for modulating the expression of superoxide dismutase 1, soluble. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding superoxide dismutase 1, soluble. Methods of using these compounds for modulation of superoxide dismutase 1, soluble expression and for treatment of diseases associated with expression of superoxide dismutase 1, soluble are provided.

公开(公告)号：US07115580B1

公开(公告)日：2006-10-03

申请号：US09980953

申请日：2000-05-25

申请人： C. Frank Bennett ,  Timothy A. Vickers ,  James G. Karras

发明人： C. Frank Bennett ,  Timothy A. Vickers ,  James G. Karras

IPC分类号： A61K31/7105 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C12Q1/68 ,  C07H21/02

CPC分类号： C12N15/1138 ,  A61K31/711 ,  A61K38/00 ,  C07H21/00 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3515 ,  Y02P20/582 ,  C12N2310/3525 ,  C12N2310/3521

摘要： Compositions and methods for the diagnosis, prevention and treatment of immune states and disorders amenable to treatment through modulation of T cell activation are provided. In accordance with preferred embodiments, oligonucleotides are provided which are specifically hybridizable with nucleic acids encoding B7 proteins.

摘要翻译： 提供了用于诊断，预防和治疗适于通过调节T细胞活化治疗的免疫状态和障碍的组合物和方法。 根据优选实施方案，提供了与编码B7蛋白的核酸特异性杂交的寡核苷酸。

公开(公告)号：US06746868B1

公开(公告)日：2004-06-08

申请号：US09705365

申请日：2000-11-03

申请人： Philip L. Felgner ,  Olivier Zelphati ,  C. Frank Bennett

发明人： Philip L. Felgner ,  Olivier Zelphati ,  C. Frank Bennett

IPC分类号： C12N1500

CPC分类号： C12N15/67 ,  A61K48/00 ,  C07K14/003 ,  C07K14/005 ,  C12N15/65 ,  C12N2710/16622

摘要： Complexes comprising a nucleic acid molecule and a conjugated peptide nucleic acid (PNA). The PNA may be labeled or conjugated to a protein, peptide, carbohydrate moiety or receptor ligand. These complexes are used to transfect cells to monitoring plasmid biodistribution, promote nuclear localization, induce transcriptional activation, lyse the endosomal compartment and facilitate transfection. These complexes increase the efficiency of expression of a particular gene.

摘要翻译： 包含核酸分子和缀合肽核酸（PNA）的复合物。 PNA可以标记或缀合到蛋白质，肽，碳水化合物部分或受体配体。 这些复合物用于转染细胞以监测质粒生物分布，促进核定位，诱导转录激活，裂解内体室并促进转染。 这些复合物增加特定基因的表达效率。

公开(公告)号：US06372492B1

公开(公告)日：2002-04-16

申请号：US09702251

申请日：2000-10-30

申请人： C. Frank Bennett ,  Lex M. Cowsert

发明人： C. Frank Bennett ,  Lex M. Cowsert

IPC分类号： C12N1500

CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  Y02P20/582 ,  C12N2310/3525

摘要： Antisense compounds, compositions and methods are provided for modulating the expression of Talin. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic acids encoding Talin. Methods of using these compounds for modulation of Talin expression and for treatment of diseases associated with expression of Talin are provided.

摘要翻译： 提供反义化合物，组合物和方法用于调节Talin的表达。 组合物包含靶向编码Talin的核酸的反义化合物，特别是反义寡核苷酸。 提供了使用这些化合物调节Talin表达和治疗与Talin表达相关的疾病的方法。