JP6679476B2 - Ｃ９ｏｒｆ７２発現を調節するための組成物 - Google Patents

Ｃ９ｏｒｆ７２発現を調節するための組成物 Download PDF

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JP6679476B2
JP6679476B2 JP2016521622A JP2016521622A JP6679476B2 JP 6679476 B2 JP6679476 B2 JP 6679476B2 JP 2016521622 A JP2016521622 A JP 2016521622A JP 2016521622 A JP2016521622 A JP 2016521622A JP 6679476 B2 JP6679476 B2 JP 6679476B2
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certain embodiments
c9orf72
modified oligonucleotide
modified
antisense
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JP2016533717A5 (enExample
JP2016533717A (ja
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フレアー，スーザン・エム
リゴ，フランク
シング，プリヤム
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--

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JP2016521622A 2013-10-11 2014-10-11 Ｃ９ｏｒｆ７２発現を調節するための組成物 Expired - Fee Related JP6679476B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201361890108P 2013-10-11 2013-10-11
US61/890,108 2013-10-11
US201361891313P 2013-10-15 2013-10-15
US61/891,313 2013-10-15
US201361919540P 2013-12-20 2013-12-20
US61/919,540 2013-12-20
US201461927903P 2014-01-15 2014-01-15
US61/927,903 2014-01-15
US201461980502P 2014-04-16 2014-04-16
US61/980,502 2014-04-16
PCT/US2014/060194 WO2015054676A2 (en) 2013-10-11 2014-10-11 Compositions for modulating c9orf72 expression

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JP2016533717A JP2016533717A (ja) 2016-11-04
JP2016533717A5 JP2016533717A5 (enExample) 2017-11-24
JP6679476B2 true JP6679476B2 (ja) 2020-04-15

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JP2020047126A Ceased JP2020115865A (ja) 2013-10-11 2020-03-18 Ｃ９ｏｒｆ７２発現を調節するための組成物

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US (3) US10221414B2 (enExample)
EP (2) EP3055414A4 (enExample)
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KR (1) KR20160062069A (enExample)
CN (2) CN112080502A (enExample)
AU (2) AU2014331652B2 (enExample)
BR (1) BR112016007751A2 (enExample)
CA (1) CA2926408A1 (enExample)
CL (3) CL2016000839A1 (enExample)
IL (1) IL244759A0 (enExample)
MX (2) MX2016004651A (enExample)
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PH (1) PH12016500603A1 (enExample)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021184720A (ja) * 2015-04-16 2021-12-09 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals, Inc. Ｃ９ｏｒｆ７２発現を調節するための組成物

Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102282155B (zh) 2008-12-02 2017-06-09 日本波涛生命科学公司 磷原子修饰的核酸的合成方法
WO2011005761A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc Novel nucleic acid prodrugs and methods use thereof
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
WO2014062686A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for modulating c9orf72 expression
BR112015008399A8 (pt) 2012-10-15 2017-10-03 Ionis Pharmaceuticals Inc Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
EP3055414A4 (en) * 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
CN113278617A (zh) 2014-01-16 2021-08-20 波涛生命科学有限公司 手性设计
US10280418B2 (en) 2014-03-18 2019-05-07 Univeristy Of Massachusetts RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
BR112017028194B1 (pt) 2015-07-10 2023-03-14 Ionis Pharmaceuticals, Inc Composto oligomérico modulador de diaciglicerol aciltransferase 2 (dgat2), composição, e seus usos
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
CN105663129B (zh) * 2015-12-29 2020-05-15 山东大学 用于治疗肌萎缩侧索硬化和额颞叶痴呆的化合物与应用
CA3017532A1 (en) * 2016-04-13 2017-10-19 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
CN110177544A (zh) 2016-11-29 2019-08-27 普尔泰克健康有限公司 用于递送治疗剂的外泌体
JP7327803B2 (ja) 2017-05-09 2023-08-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症（ａｌｓ）を処置する方法
WO2019032607A1 (en) 2017-08-08 2019-02-14 Wave Life Sciences Ltd. OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS
AU2018338188B2 (en) 2017-09-22 2025-02-20 University Of Massachusetts SOD1 dual expression vectors and uses thereof
CA3083582A1 (en) * 2017-10-23 2019-05-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
US10905707B2 (en) * 2017-11-10 2021-02-02 University Of Massachusetts Compositions and methods for the treatment of expanded repeat-associated disorders
EP3737387A1 (en) 2018-01-12 2020-11-18 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
BR112020014011A2 (pt) 2018-01-12 2020-12-08 Bristol-Myers Squibb Company Oligonucleotídeos antissenso dirigidos à alfa-sinucleína e usos dos mesmos
WO2020055917A1 (en) * 2018-09-10 2020-03-19 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating cln3 expression
JP7505788B2 (ja) * 2018-10-01 2024-06-25 ユナイテッド ニューロサイエンス Ｃ９ｏｒｆ７２に由来するジペプチドリピートタンパク質に対して指向化されたペプチド免疫原コンストラクト
MA54383A (fr) 2018-12-06 2021-10-13 Biogen Ma Inc Protéine neurofilamenteuse pour guider une intervention thérapeutique dans la sclérose laterale amyotrophique
EP3918073A4 (en) * 2019-01-29 2023-11-22 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION
JP2022528487A (ja) * 2019-03-29 2022-06-13 ユニバーシティ・オブ・マサチューセッツ Ｃ９ｏｒｆ７２のオリゴヌクレオチドベースの調節
US11629347B2 (en) 2019-05-06 2023-04-18 University Of Massachusetts Anti-C9ORF72 oligonucleotides and related methods
MX2022006433A (es) * 2019-12-13 2022-06-23 Alnylam Pharmaceuticals Inc Composiciones de agentes de acido ribonucleico de interferencia (arni) del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72) y metodos de uso de los mismos.
WO2021159008A2 (en) 2020-02-07 2021-08-12 Maze Therapeutics, Inc. Compositions and methods for treating neurodegenerative diseases
WO2021174031A2 (en) * 2020-02-28 2021-09-02 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating splicing of pre-mrna
US12344839B2 (en) * 2020-03-27 2025-07-01 University Of Massachusetts Dual-acting siRNA based modulation of C9orf72
JP7787094B2 (ja) * 2020-04-09 2025-12-16 アソシエーション・アンスティトゥート・ドゥ・マイオロジー 筋萎縮性側索硬化症を治療するためのアンチセンス配列
UY39344A (es) 2020-07-28 2022-02-25 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app
CA3213590A1 (en) 2021-04-06 2022-10-13 Maze Therapeutics, Inc. Compositions and methods for treating tdp-43 proteinopathy
WO2024077109A1 (en) 2022-10-05 2024-04-11 Maze Therapeutics, Inc. Unc13a antisense oligonucleotides and uses thereof
WO2025107038A1 (en) * 2023-11-23 2025-05-30 Murdoch University Method of treating motor neurone disease

Family Cites Families (79)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
DK1695979T3 (da) 1991-12-24 2011-10-10 Isis Pharmaceuticals Inc Gappede modificerede oligonukleotider
RU95114435A (ru) 1992-12-14 1997-05-20 Ханивелл Инк. (Us) Система с бесщеточным двигателем постоянного тока
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5681940A (en) 1994-11-02 1997-10-28 Icn Pharmaceuticals Sugar modified nucleosides and oligonucleotides
WO2005121368A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Chimeric gapped oligomeric compositions
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US5998148A (en) 1999-04-08 1999-12-07 Isis Pharmaceuticals Inc. Antisense modulation of microtubule-associated protein 4 expression
KR100782896B1 (ko) 1999-05-04 2007-12-06 엑시콘 에이/에스 Ｌ-리보-ｌｎａ 유사체
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
DE19935303A1 (de) * 1999-07-28 2001-02-08 Aventis Pharma Gmbh Oligonukleotide zur Inhibierung der Expression von humanem eg5
USH2191H1 (en) 2000-10-24 2007-06-05 Snp Consortium Identification and mapping of single nucleotide polymorphisms in the human genome
US20050019915A1 (en) 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
AU2003291755A1 (en) 2002-11-05 2004-06-07 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7217807B2 (en) 2002-11-26 2007-05-15 Rosetta Genomics Ltd Bioinformatically detectable group of novel HIV regulatory genes and uses thereof
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
JP4731324B2 (ja) 2003-08-28 2011-07-20 武 今西 Ｎ−ｏ結合性架橋構造型新規人工核酸
AU2004274021B2 (en) 2003-09-18 2009-08-13 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
US20050261233A1 (en) 2004-04-21 2005-11-24 Sanjay Bhanot Modulation of glucose-6-phosphatase translocase expression
AU2005248147A1 (en) 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
WO2007056113A2 (en) 2005-11-02 2007-05-18 Cylene Pharmaceuticals, Inc. Methods for targeting quadruplex sequences
DK2161038T3 (da) 2006-01-26 2014-03-03 Isis Pharmaceuticals Inc Sammensætninger og deres anvendelser rettet mod Huntington
US7399845B2 (en) 2006-01-27 2008-07-15 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
JP5731115B2 (ja) 2006-05-05 2015-06-10 アイシス ファーマシューティカルズ， インコーポレーテッド 遺伝子発現を調節するための化合物および方法
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
AU2007306361A1 (en) * 2006-10-09 2008-04-17 Santaris Pharma A/S RNA antagonist compounds for the modulation of PCSK9
BRPI0719995A2 (pt) 2006-12-14 2014-03-18 Novartis Ag Composições e métodos para tratar distúrbios musculares & cardiovasculares
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
WO2008150729A2 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
DK2173760T4 (en) 2007-06-08 2016-02-08 Isis Pharmaceuticals Inc Carbocyclic bicyclic nukleinsyreanaloge
EP2014769B1 (en) 2007-06-18 2010-03-31 Commissariat à l'Energie Atomique Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
US20090123928A1 (en) 2007-10-11 2009-05-14 The Johns Hopkins University Genomic Landscapes of Human Breast and Colorectal Cancers
US20110152346A1 (en) * 2007-11-05 2011-06-23 Baltic Technology Development Ltd. Use of Oligonucleotides with Modified Bases in Hybridization of Nucleic Acids
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
US8530640B2 (en) 2008-02-07 2013-09-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2010019270A1 (en) 2008-08-14 2010-02-18 Isis Pharmaceuticals, Inc. Modulation of prion expression
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
US20120149757A1 (en) 2009-04-13 2012-06-14 Krainer Adrian R Compositions and methods for modulation of smn2 splicing
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US20140004565A1 (en) 2009-07-06 2014-01-02 Alnylam Pharmaceuticals, Inc. Cell-based bioprocessing
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011135396A1 (en) 2010-04-30 2011-11-03 Cellectis Method for modulating double-strand break-induced homologous recombination
WO2011156673A2 (en) * 2010-06-10 2011-12-15 Isis Pharmaceuticals, Inc. Modulation of phosphoenolpyruvate carboxykinase-mitochondrial (pepck-m) expression
WO2012005898A2 (en) 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
AU2011282243B2 (en) 2010-07-19 2016-09-22 Ionis Pharmaceuticals, Inc. Modulation of nuclear-retained RNA
EP2655621B1 (en) 2010-12-20 2018-05-23 The General Hospital Corporation Polycomb-associated non-coding rnas
WO2012092367A1 (en) 2010-12-28 2012-07-05 University Of Rochester Nucleic acid binding compounds, methods of making, and use thereof
CN107012144A (zh) 2011-04-01 2017-08-04 Ionis制药公司 信号转导及转录激活蛋白3(stat3)表达的调节
SG10201604074TA (en) 2011-04-21 2016-07-28 Isis Pharmaceuticals Inc Modulation of hepatitis b virus (hbv) expression
US9896729B2 (en) 2011-08-31 2018-02-20 The University Of Manchester Method for diagnosing a neurodegenerative disease
WO2013036833A1 (en) 2011-09-09 2013-03-14 Mayo Foundation For Medical Education And Research Detecting frontotemporal dementia and amyotrophic lateral sclerosis
CN103930571A (zh) 2011-11-17 2014-07-16 瑞尼克斯有限公司 用于选择性分子分析的系统和方法
JP6317675B2 (ja) 2011-11-30 2018-04-25 サレプタ セラピューティクス， インコーポレイテッド 延長リピート病を処置するためのオリゴヌクレオチド
WO2013086207A1 (en) 2011-12-06 2013-06-13 The Ohio State University Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
EP3228712A1 (en) 2012-04-23 2017-10-11 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
WO2014062686A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for modulating c9orf72 expression
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
BR112015008399A8 (pt) 2012-10-15 2017-10-03 Ionis Pharmaceuticals Inc Composto para modular a expressão de c90rf72, seu uso, oligonucleotídeo modificado, composto de fita dupla e composição
US10066007B2 (en) 2013-01-22 2018-09-04 Deutsches Zentrum Für Neurodegenerative Erkrankungen E.V. (Dzne) Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion
EP3055414A4 (en) 2013-10-11 2017-07-19 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
WO2016024205A1 (en) 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
US10392447B2 (en) 2014-09-30 2019-08-27 Neurimmune Holding Ag Human-derived anti-dipeptide repeats (DPRs) antibody
US10538762B2 (en) 2014-10-14 2020-01-21 The Board Of Regents Of The University Of Texas System Allele selective inhibition of mutant C9orf72 foci expression by duplex RNAS targeting the expanded hexanucleotide repeat
WO2016168592A2 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
WO2017079291A1 (en) 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
SG10201510101XA (en) 2015-12-09 2017-07-28 Au Optronics Corp Evaporation apparatus and evaporation method
CA3017532A1 (en) 2016-04-13 2017-10-19 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
CA3038548A1 (en) 2016-09-30 2018-04-05 Regeneron Pharmaceuticals, Inc. Non-human animals having a hexanucleotide repeat expansion in a c9orf72 locus

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021184720A (ja) * 2015-04-16 2021-12-09 アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals, Inc. Ｃ９ｏｒｆ７２発現を調節するための組成物

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