公开(公告)号：US09663783B2

公开(公告)日：2017-05-30

申请号：US13988296

申请日：2011-11-17

Applicant: Susan M. Freier

Inventor： Susan M. Freier

IPC: C12N15/11 ,  C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3525

Abstract: Disclosed herein are antisense compounds and methods for decreasing alpha-synuclein mRNA and protein expression. Also disclosed herein are methods for treating, preventing, and ameliorating neurodegenerative diseases in an individual in need thereof.

公开(公告)号：US08921331B2

公开(公告)日：2014-12-30

申请号：US13406256

申请日：2012-02-27

Applicant: C. Frank Bennett ,  Susan M. Freier ,  Kenneth W. Dobie

Inventor： C. Frank Bennett ,  Susan M. Freier ,  Kenneth W. Dobie

IPC: A61K48/00 ,  C12N15/113 ,  A61K38/00

CPC classification number: C12N15/1137 ,  A61K38/00 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12Y115/01001 ,  Y02P20/582 ,  C12N2310/3525

Abstract: Methods for slowing disease progression in an individual suffering from familial ALS are provided. Also provided are methods of increasing the survival time of an individual suffering from familial ALS. These methods employ antisense oligonucleotides targeted to SOD1, for use in inhibiting the expression of SOD1 in the central nervous system of an individual suffering from familial ALS.

Abstract translation: 提供了减轻患有家族性ALS的个体疾病进展的方法。 还提供了增加患有家族性ALS的个体的存活时间的方法。 这些方法使用靶向SOD1的反义寡核苷酸，用于抑制患有家族性ALS的个体的中枢神经系统中SOD1的表达。

公开(公告)号：US08791088B2

公开(公告)日：2014-07-29

申请号：US13566922

申请日：2012-08-03

Applicant: C. Frank Bennett ,  Kenneth W. Dobie ,  Susan M. Freier ,  Stanley T. Crooke

Inventor： C. Frank Bennett ,  Kenneth W. Dobie ,  Susan M. Freier ,  Stanley T. Crooke

IPC: C12N15/11

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2320/33

Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.

Abstract translation: 本文公开了用于调节细胞，组织或动物中LMNA表达的化合物，组合物和方法。 还提供了目标验证的方法。 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。 还提供了使用所公开的化合物鉴定所选mRNA的顺式剪接调节元件的方法。

公开(公告)号：US20140107330A1

公开(公告)日：2014-04-17

申请号：US13984260

申请日：2012-02-08

Applicant: Susan M. Freier ,  Eric E. Swayze

Inventor： Susan M. Freier ,  Eric E. Swayze

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3525 ,  C12N2320/50 ,  C12N2310/322 ,  C12N2310/3531 ,  C12N2310/3533 ,  C12N2310/321 ,  C12N2310/3521

Abstract: The present invention provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.

Abstract translation: 本发明提供低聚化合物。 某些这样的寡聚化合物可用于与互补核酸杂交，包括但不限于细胞中的核酸。 在某些实施方案中，杂交导致细胞中靶核酸的活性或表达的调节。

公开(公告)号：US08697663B2

公开(公告)日：2014-04-15

申请号：US12345725

申请日：2008-12-30

Applicant: C. Frank Bennett ,  Susan M. Freier ,  Richard H. Griffey

Inventor： C. Frank Bennett ,  Susan M. Freier ,  Richard H. Griffey

IPC: C12N15/11 ,  A61K48/00 ,  C07H21/02 ,  C07H21/04

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。

公开(公告)号：US08669102B2

公开(公告)日：2014-03-11

申请号：US13059058

申请日：2009-08-14

Applicant: C. Frank Bennett ,  Gene Hung ,  Susan M. Freier ,  Kenneth W. Dobie

Inventor： C. Frank Bennett ,  Gene Hung ,  Susan M. Freier ,  Kenneth W. Dobie

IPC: C12N15/85 ,  C07H21/04 ,  C07H21/02

CPC classification number: A61K31/7125

Abstract: Disclosed herein are compounds and methods for decreasing PrP and preventing, ameliorating, or treating a prion disease or conformational neurodegenerative disorder, in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to PrP include Creutzfeldt-Jakob disease (CJD); variant Creutzfeldt-Jakob Disease (vCJD); Gerstmann-Straussler-Scheinker syndrome; fatal familial insomnia; kuru; Bovine Spongiform Encephalopathy (BSE), e.g. “mad cow disease”; Chronic Wasting Disease (CWD); scrapie; transmissible mink encephalopathy; feline spongiform encephalopathy; ungulate spongiform encephalopathy; Alzheimer's disease; Parkinson's disease; Huntington's disease; and Amyotrophic Lateral Sclerosis (ALS).

Abstract translation: 本文公开了在有需要的个体中减少PrP并预防，改善或治疗朊病毒疾病或构象神经变性疾病的化合物和方法。 通过施用针对PrP的反义化合物可以改善的疾病状况的实例包括克雅氏病（CJD）; 变异克雅氏病（vCJD）; Gerstmann-Straussler-Scheinker综合征; 致命性家族性失眠; 库鲁 牛海绵状脑病（BSE），例如。 “疯牛病”; 慢性疾病（CWD）; scrap子 传染性貂脑病 猫海绵状脑病; 有蹄型海绵状脑病 阿尔茨海默氏病; 帕金森病 亨廷顿氏病 和肌萎缩性侧索硬化（ALS）。

公开(公告)号：US08476421B2

公开(公告)日：2013-07-02

申请号：US12710232

申请日：2010-02-22

Applicant: Susan M. Freier ,  James G. Karras

Inventor： Susan M. Freier ,  James G. Karras

IPC: C07H21/04

Abstract: Compounds, compositions and methods are provided for modulating the expression of STAT5. The compositions comprise oligonucleotides, targeted to nucleic acid encoding STAT5. Methods of using these compounds for modulation of STAT5 expression and for diagnosis and treatment of diseases and conditions associated with expression of STAT5 are provided.

公开(公告)号：US08415465B2

公开(公告)日：2013-04-09

申请号：US13090146

申请日：2011-04-19

Applicant: Susan M. Freier

Inventor： Susan M. Freier

IPC: C07H21/04

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/33 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2310/3525

Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's Disease (HD) progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntington's Disease (HD) in an individual susceptible to Huntington's Disease (HD). Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.

公开(公告)号：US20130035370A1

公开(公告)日：2013-02-07

申请号：US13566922

申请日：2012-08-03

Applicant: C. Frank Bennett ,  Kenneth W. Dobie ,  Susan M. Freier ,  Stanley T. Crooke

Inventor： C. Frank Bennett ,  Kenneth W. Dobie ,  Susan M. Freier ,  Stanley T. Crooke

IPC: A61K31/712 ,  C12N5/071

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2320/33

Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of LMNA in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders. Further provided are methods of identifying cis splicing regulatory elements of a selected mRNA using the disclosed compounds.

公开(公告)号：US20120316221A1

公开(公告)日：2012-12-13

申请号：US13408646

申请日：2012-02-29

Applicant: JAMES G. KARRAS ,  SUSAN GREGORY ,  JEFFREY R. CROSBY ,  MAUSUMEE GUHA ,  DAVID TUNG ,  SUSAN M. FREIER ,  KENNETH W. DOBIE

Inventor： JAMES G. KARRAS ,  SUSAN GREGORY ,  JEFFREY R. CROSBY ,  MAUSUMEE GUHA ,  DAVID TUNG ,  SUSAN M. FREIER ,  KENNETH W. DOBIE

IPC: C07H21/04 ,  A61P29/00 ,  A61P11/00 ,  A61K31/7088

CPC classification number: C12N15/1138 ,  A61K38/00 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/3525

Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of IL-4R alpha in a cell, tissue or animal. Also provided are methods of target validation. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders related to expression of IL 4R-α, airway hyperresponsiveness, and/or pulmonary inflammation.

Abstract translation: 本文公开了用于调节细胞，组织或动物中IL-4Rα的表达的化合物，组合物和方法。 还提供了目标验证的方法。 还提供了所公开的化合物和组合物在制备用于治疗与IL 4R-α的表达，气道高反应性和/或肺部炎症相关的疾病和病症的药物中的用途。