SG11201708468YA - Compositions for modulating c9orf72 expression - Google Patents

Compositions for modulating c9orf72 expression

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Publication number
SG11201708468YA
SG11201708468YA SG11201708468YA SG11201708468YA SG11201708468YA SG 11201708468Y A SG11201708468Y A SG 11201708468YA SG 11201708468Y A SG11201708468Y A SG 11201708468YA SG 11201708468Y A SG11201708468Y A SG 11201708468YA SG 11201708468Y A SG11201708468Y A SG 11201708468YA
Authority
SG
Singapore
Prior art keywords
compositions
c9orf72 expression
modulating
modulating c9orf72
expression
Prior art date
Application number
SG11201708468YA
Inventor
Frank Rigo
Original Assignee
Ionis Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of SG11201708468YA publication Critical patent/SG11201708468YA/en

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • 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
    • A61P25/14—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
    • A61P25/22—Anxiolytics
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
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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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • 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
    • C12N2310/3341—5-Methylcytosine
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
SG11201708468YA 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression SG11201708468YA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562148691P 2015-04-16 2015-04-16
US201562232941P 2015-09-25 2015-09-25
US201562239400P 2015-10-09 2015-10-09
PCT/US2016/027747 WO2016168592A2 (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression

Publications (1)

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SG11201708468YA true SG11201708468YA (en) 2017-11-29

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SG10202001856WA SG10202001856WA (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression
SG11201708468YA SG11201708468YA (en) 2015-04-16 2016-04-15 Compositions for modulating c9orf72 expression

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US (4) US9605263B2 (en)
EP (2) EP3283080B1 (en)
JP (3) JP6606557B2 (en)
KR (2) KR102258516B1 (en)
CN (2) CN113265404A (en)
AU (2) AU2016249002B2 (en)
BR (1) BR112017016068A2 (en)
CA (1) CA2978103A1 (en)
CL (4) CL2017002567A1 (en)
CO (1) CO2017007671A2 (en)
CY (1) CY1122981T1 (en)
DK (1) DK3283080T3 (en)
ES (1) ES2791995T3 (en)
HK (1) HK1250255A1 (en)
HR (1) HRP20200843T1 (en)
HU (1) HUE049233T2 (en)
IL (2) IL253557B (en)
LT (1) LT3283080T (en)
MX (2) MX366128B (en)
MY (1) MY181815A (en)
NZ (1) NZ733882A (en)
PH (2) PH12017501875B1 (en)
PL (1) PL3283080T3 (en)
PT (1) PT3283080T (en)
RS (1) RS60230B1 (en)
RU (1) RU2736574C2 (en)
SA (3) SA520412450B1 (en)
SG (2) SG10202001856WA (en)
SI (1) SI3283080T1 (en)
WO (1) WO2016168592A2 (en)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI0923225A2 (en) 2008-12-02 2016-10-04 Chiralgen Ltd Phosphorus-modified nucleic acid synthesis method
MX342945B (en) 2009-07-06 2016-10-18 Ontorii Inc * Novel nucleic acid prodrugs and methods use thereof.
JP5868324B2 (en) 2010-09-24 2016-02-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ Asymmetric auxiliary group
WO2013012758A1 (en) 2011-07-19 2013-01-24 Ontorii, Inc. Methods for the synthesis of functionalized nucleic acids
CN104684893B (en) 2012-07-13 2016-10-26 日本波涛生命科学公司 Asymmetric auxiliary group
US10443052B2 (en) 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
US9963699B2 (en) 2012-10-15 2018-05-08 Ionis Pharmaceuticals, Inc. Methods for modulating C9ORF72 expression
WO2014062736A1 (en) 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
WO2015054676A2 (en) 2013-10-11 2015-04-16 Isis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
KR20230152178A (en) 2014-01-16 2023-11-02 웨이브 라이프 사이언시스 리미티드 Chiral design
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
US20190142856A1 (en) * 2016-04-13 2019-05-16 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
WO2018081728A1 (en) * 2016-10-31 2018-05-03 Emendobio Inc. Compositions for genome editing
CA3072076A1 (en) 2017-08-08 2019-02-14 Chandra Vargeese Oligonucleotide compositions and methods thereof
WO2019094694A1 (en) 2017-11-10 2019-05-16 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
JP7383298B2 (en) 2018-08-03 2023-11-20 国立大学法人 東京医科歯科大学 TDP-43 aggregation inhibitor
EP3890752A1 (en) 2018-12-06 2021-10-13 Biogen MA Inc. Neurofilament protein for guiding therapeutic intervention in amyotrophic lateral sclerosis
JP2022528487A (en) * 2019-03-29 2022-06-13 ユニバーシティ・オブ・マサチューセッツ Oligonucleotide-based regulation of C9orf72
EP3966328A4 (en) * 2019-05-06 2023-10-18 University Of Massachusetts Anti-c9orf72 oligonucleotides and related methods
MX2022004102A (en) * 2019-10-06 2022-04-26 Wave Life Sciences Ltd Oligonucleotide compositions and methods of use thereof.

Family Cites Families (99)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (en) 1984-07-19 1989-02-17 Centre Nat Rech Scient NOVEL OLIGONUCLEOTIDES, THEIR PREPARATION PROCESS AND THEIR APPLICATIONS AS MEDIATORS IN DEVELOPING THE EFFECTS OF INTERFERONS
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
WO1991006556A1 (en) 1989-10-24 1991-05-16 Gilead Sciences, Inc. 2' modified oligonucleotides
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
ES2116977T3 (en) 1990-05-11 1998-08-01 Microprobe Corp SOLID SUPPORTS FOR NUCLEIC ACID HYBRIDIZATION TESTS AND METHODS TO IMMOBILIZE OLIGONUCLEOTIDES IN A COVALENT WAY.
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
EP0538194B1 (en) 1991-10-17 1997-06-04 Novartis AG Bicyclic nucleosides, oligonucleotides, their method of preparation and intermediates therein
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
EP1044987B1 (en) 1991-12-24 2006-02-15 Isis Pharmaceuticals, Inc. Gapped 2'-modified oligonucleotides
FR2687679B1 (en) 1992-02-05 1994-10-28 Centre Nat Rech Scient OLIGOTHIONUCLEOTIDES.
EP0577558A2 (en) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclic nucleosides having bicyclic rings, oligonucleotides therefrom, process for their preparation, their use and intermediates
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi Acyclic nucleoside analogs and oligonucleotide sequences containing them
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (en) 1997-03-07 2006-03-15 武 今西 Novel bicyclonucleosides and oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
DE69829760T3 (en) 1997-09-12 2016-04-14 Exiqon A/S BI- AND TRI-CYCLIC-NUCLEOSIDE, NUCLEOTIDE AND OLIGONUCLEOTIDE ANALOG
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
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
US5998148A (en) 1999-04-08 1999-12-07 Isis Pharmaceuticals Inc. Antisense modulation of microtubule-associated protein 4 expression
DK1178999T3 (en) 1999-05-04 2007-08-06 Santaris Pharma As L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP2003519231A (en) 1999-12-30 2003-06-17 カー・ユ・ルーベン・リサーチ・アンド・ディベロップメント Cyclohexene nucleic acid
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
WO2003004602A2 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
EP1562971B1 (en) 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004044132A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Modified oligonucleotides for use in rna interference
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
WO2005021570A1 (en) 2003-08-28 2005-03-10 Gene Design, Inc. Novel artificial nucleic acids of n-o bond crosslinkage type
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
WO2005116204A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
AU2005252663B2 (en) 2004-06-03 2011-07-07 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US20080261905A1 (en) 2004-11-08 2008-10-23 K.U. Leuven Research And Development Modified Nucleosides for Rna Interference
JP5425474B2 (en) 2006-01-26 2014-02-26 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 Composition and use thereof for huntingtin
DK2314594T3 (en) 2006-01-27 2014-10-27 Isis Pharmaceuticals Inc 6-modified bicyclic nucleic acid analogues
EP2023939B1 (en) 2006-05-05 2012-06-27 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of pcsk9
WO2007134181A2 (en) 2006-05-11 2007-11-22 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
US20100280094A1 (en) 2006-12-14 2010-11-04 Novartis Ag Compositions and methods to treat muscular & cardiovascular disorders
WO2008101157A1 (en) 2007-02-15 2008-08-21 Isis Pharmaceuticals, Inc. 5'-substituted-2'-f modified nucleosides and oligomeric compounds prepared therefrom
US8278425B2 (en) 2007-05-30 2012-10-02 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US8278426B2 (en) 2007-06-08 2012-10-02 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
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
US8278283B2 (en) 2007-07-05 2012-10-02 Isis Pharmaceuticals, Inc. 6-disubstituted or unsaturated bicyclic nucleic acid analogs
US20090123928A1 (en) 2007-10-11 2009-05-14 The Johns Hopkins University Genomic Landscapes of Human Breast and Colorectal Cancers
AU2008324068A1 (en) * 2007-11-05 2009-05-14 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
WO2009100320A2 (en) 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
DK2361256T3 (en) 2008-09-24 2013-07-01 Isis Pharmaceuticals Inc Cyclohexenyl-nucleic acid analogues
EP2356129B1 (en) 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2010120820A1 (en) 2009-04-13 2010-10-21 Isis Pharmaceuticals, Inc. 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
EP2462153B1 (en) 2009-08-06 2015-07-29 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
WO2012005898A2 (en) 2010-06-15 2012-01-12 Alnylam Pharmaceuticals, Inc. Chinese hamster ovary (cho) cell transcriptome, corresponding sirnas and uses thereof
US20130237585A1 (en) * 2010-07-19 2013-09-12 University Of Rochester Modulation of dystrophia myotonica-protein kinase (dmpk) expression
CA3077910A1 (en) * 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
DK2655621T3 (en) 2010-12-20 2018-08-13 Massachusetts Gen Hospital Polycomb-associated non-coding RNAS
US20140050778A1 (en) 2010-12-28 2014-02-20 University Of Rochester Nucleic acid binding compounds, methods of making, and use thereof
KR20190092590A (en) 2011-04-01 2019-08-07 아이오니스 파마수티컬즈, 인코포레이티드 Modulation of signal transducer and activator of transcription 3 (stat3) expression
DK3505528T3 (en) * 2011-04-21 2021-02-08 Ionis Pharmaceuticals Inc MODULATION OF HEPATITIS B VIRUS (HBV) EXPRESSION
EP3202914B2 (en) 2011-08-31 2023-06-28 The University of Manchester Method for treating a neurodegenerative disease
US20140255936A1 (en) 2011-09-09 2014-09-11 Mayo Foundation For Medical Education And Research Detecting frontotemporal dementia and amyotrophic lateral sclerosis
IN2014CN03749A (en) * 2011-10-25 2015-09-25 Isis Pharmaceuticals Inc
US20130157885A1 (en) * 2011-11-17 2013-06-20 Rheonix, Inc. System and methods for selective molecular analysis
JP6317675B2 (en) * 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 Oligonucleotides for treating prolonged repeat disease
EP2788087A4 (en) 2011-12-06 2015-08-26 Ohio State Innovation Foundation Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease
JP6174678B2 (en) 2012-04-23 2017-08-02 バイオマリン テクノロジーズ ベー．フェー． RNA regulatory oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
ES2820548T3 (en) * 2012-05-24 2021-04-21 Ionis Pharmaceuticals Inc Methods and compositions to modulate the expression of apolipoprotein (a)
WO2014062736A1 (en) * 2012-10-15 2014-04-24 Isis Pharmaceuticals, Inc. Methods for monitoring c9orf72 expression
US9963699B2 (en) 2012-10-15 2018-05-08 Ionis Pharmaceuticals, Inc. Methods for modulating C9ORF72 expression
US10443052B2 (en) * 2012-10-15 2019-10-15 Ionis Pharmaceuticals, Inc. Compositions for modulating C9ORF72 expression
LT2948777T (en) 2013-01-22 2019-09-25 Deutsches Zentrum Für Neurodegenerative Erkrankungen E.V. Dipeptide-repeat proteins as therapeutic target in neurodegenerative diseases with hexanucleotide repeat expansion
WO2015054676A2 (en) * 2013-10-11 2015-04-16 Isis 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
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
US11260073B2 (en) 2015-11-02 2022-03-01 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating C90RF72
US20190142856A1 (en) 2016-04-13 2019-05-16 Ionis Pharmaceuticals, Inc. Methods for reducing c9orf72 expression
US10399845B2 (en) 2017-09-22 2019-09-03 Emco Wheaton Retail Corporation Testable overfill prevention valve for a liquid storage container

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