CA2459347C - Locked nucleic acid (lna) compositions and uses thereof - Google Patents

Locked nucleic acid (lna) compositions and uses thereof Download PDF

Info

Publication number
CA2459347C
CA2459347C CA2459347A CA2459347A CA2459347C CA 2459347 C CA2459347 C CA 2459347C CA 2459347 A CA2459347 A CA 2459347A CA 2459347 A CA2459347 A CA 2459347A CA 2459347 C CA2459347 C CA 2459347C
Authority
CA
Canada
Prior art keywords
nucleic acid
target
acid molecule
lna
molecule
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
CA2459347A
Other languages
English (en)
French (fr)
Other versions
CA2459347A1 (en
Inventor
Jesper Wengel
Sakari Kauppinen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Qiagen GmbH
Original Assignee
Exiqon AS
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 Exiqon AS filed Critical Exiqon AS
Publication of CA2459347A1 publication Critical patent/CA2459347A1/en
Application granted granted Critical
Publication of CA2459347C publication Critical patent/CA2459347C/en
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA2459347A 2001-09-04 2002-09-04 Locked nucleic acid (lna) compositions and uses thereof Expired - Lifetime CA2459347C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US31703401P 2001-09-04 2001-09-04
US60/317,034 2001-09-04
US32396701P 2001-09-22 2001-09-22
US60/323,967 2001-09-22
PCT/IB2002/003911 WO2003020739A2 (en) 2001-09-04 2002-09-04 Novel lna compositions and uses thereof

Publications (2)

Publication Number Publication Date
CA2459347A1 CA2459347A1 (en) 2003-03-13
CA2459347C true CA2459347C (en) 2012-10-09

Family

ID=26980727

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2459347A Expired - Lifetime CA2459347C (en) 2001-09-04 2002-09-04 Locked nucleic acid (lna) compositions and uses thereof

Country Status (6)

Country Link
US (1) US7060809B2 (https=)
EP (1) EP1446412B1 (https=)
JP (1) JP2005514005A (https=)
AU (1) AU2002334307A1 (https=)
CA (1) CA2459347C (https=)
WO (1) WO2003020739A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210010064A1 (en) * 2018-03-14 2021-01-14 The Trustees Of The University Of Pennsylvania Enrichment of nucleic acids

Families Citing this family (320)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
US7226739B2 (en) 2001-03-02 2007-06-05 Isis Pharmaceuticals, Inc Methods for rapid detection and identification of bioagents in epidemiological and forensic investigations
US20030027135A1 (en) 2001-03-02 2003-02-06 Ecker David J. Method for rapid detection and identification of bioagents
US7666588B2 (en) 2001-03-02 2010-02-23 Ibis Biosciences, Inc. Methods for rapid forensic analysis of mitochondrial DNA and characterization of mitochondrial DNA heteroplasmy
US20040121309A1 (en) 2002-12-06 2004-06-24 Ecker David J. Methods for rapid detection and identification of bioagents in blood, bodily fluids, and bodily tissues
US7718354B2 (en) 2001-03-02 2010-05-18 Ibis Biosciences, Inc. Methods for rapid identification of pathogens in humans and animals
US7217510B2 (en) 2001-06-26 2007-05-15 Isis Pharmaceuticals, Inc. Methods for providing bacterial bioagent characterizing information
US8073627B2 (en) 2001-06-26 2011-12-06 Ibis Biosciences, Inc. System for indentification of pathogens
EP2213737B1 (en) 2002-02-01 2012-11-07 Life Technologies Corporation Double-stranded oligonucleotides
US20060009409A1 (en) 2002-02-01 2006-01-12 Woolf Tod M Double-stranded oligonucleotides
US20030166282A1 (en) * 2002-02-01 2003-09-04 David Brown High potency siRNAS for reducing the expression of target genes
US7176002B2 (en) * 2002-05-16 2007-02-13 Applera Corporation Universal-tagged oligonucleotide primers and methods of use
AU2003276666A1 (en) * 2002-06-12 2003-12-31 Ambion, Inc. Methods and compositions relating to polypeptides with rnase iii domains that mediate rna interference
US20040248094A1 (en) * 2002-06-12 2004-12-09 Ford Lance P. Methods and compositions relating to labeled RNA molecules that reduce gene expression
US20100075423A1 (en) * 2002-06-12 2010-03-25 Life Technologies Corporation Methods and compositions relating to polypeptides with rnase iii domains that mediate rna interference
US20060147924A1 (en) * 2002-09-11 2006-07-06 Ramsing Neils B Population of nucleic acids including a subpopulation of lna oligomers
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
JP2006516193A (ja) 2002-12-06 2006-06-29 アイシス・ファーマシューティカルス・インコーポレーテッド ヒトおよび動物における病原体の迅速な同定方法
US8046171B2 (en) 2003-04-18 2011-10-25 Ibis Biosciences, Inc. Methods and apparatus for genetic evaluation
US8057993B2 (en) 2003-04-26 2011-11-15 Ibis Biosciences, Inc. Methods for identification of coronaviruses
US7964343B2 (en) 2003-05-13 2011-06-21 Ibis Biosciences, Inc. Method for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture
US8158354B2 (en) 2003-05-13 2012-04-17 Ibis Biosciences, Inc. Methods for rapid purification of nucleic acids for subsequent analysis by mass spectrometry by solution capture
BRPI0410886A (pt) 2003-06-03 2006-07-04 Isis Pharmaceuticals Inc composto de filamento duplo, composição farmacêutica, sal farmaceuticamente aceitável, métodos de modificação do ácido nucleico que codifica a survivina humana, de inibição da expressão da suvivina em células ou tecidos, e de tratamento de uma condição associada com a expressão ou superexpressão da suvivina, e, oligonucleotìdeo de rnai de filamento único
US20050089889A1 (en) * 2003-06-20 2005-04-28 Ramsing Niels B. Probes, libraries and kits for analysis of mixtures of nucleic acids and methods for constructing the same
US20070269803A1 (en) * 2003-06-26 2007-11-22 Khalil Arar Fluorogenic Nucleic Acid Probes Including Lna for Methods to Detect and/or Quantify Nucleic Acid Analytes
US7956175B2 (en) 2003-09-11 2011-06-07 Ibis Biosciences, Inc. Compositions for use in identification of bacteria
US8546082B2 (en) 2003-09-11 2013-10-01 Ibis Biosciences, Inc. Methods for identification of sepsis-causing bacteria
US8097416B2 (en) 2003-09-11 2012-01-17 Ibis Biosciences, Inc. Methods for identification of sepsis-causing bacteria
US7480382B2 (en) * 2003-09-30 2009-01-20 Microsoft Corporation Image file container
US7666592B2 (en) 2004-02-18 2010-02-23 Ibis Biosciences, Inc. Methods for concurrent identification and quantification of an unknown bioagent
US8119336B2 (en) 2004-03-03 2012-02-21 Ibis Biosciences, Inc. Compositions for use in identification of alphaviruses
US8569474B2 (en) * 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
US8192937B2 (en) 2004-04-07 2012-06-05 Exiqon A/S Methods for quantification of microRNAs and small interfering RNAs
EP2458619B1 (en) 2004-05-24 2017-08-02 Ibis Biosciences, Inc. Mass spectrometry with selective ion filtration by digital thresholding
US20050266411A1 (en) 2004-05-25 2005-12-01 Hofstadler Steven A Methods for rapid forensic analysis of mitochondrial DNA
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
DE102004034343B4 (de) * 2004-07-10 2007-08-30 Olfert Landt Verfahren zum Nachweis von Spuren genomischer Varianten mittels Nukleinsäure-Amplifikationsreaktionen
US7811753B2 (en) 2004-07-14 2010-10-12 Ibis Biosciences, Inc. Methods for repairing degraded DNA
WO2006135400A2 (en) * 2004-08-24 2006-12-21 Isis Pharmaceuticals, Inc. Methods for rapid identification of recombinant organisms
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
US20060166238A1 (en) * 2004-12-22 2006-07-27 Ramsing Niels B Probes, libraries and kits for analysis of mixtures of nucleic acids and methods for constructing the same
US20060142228A1 (en) * 2004-12-23 2006-06-29 Ambion, Inc. Methods and compositions concerning siRNA's as mediators of RNA interference
WO2006069584A2 (en) 2004-12-29 2006-07-06 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF microRNAs AND THEIR TARGET mRNAs
US8071306B2 (en) 2005-01-25 2011-12-06 Merck Sharp & Dohme Corp. Methods for quantitating small RNA molecules
AU2006213686A1 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
US8084207B2 (en) 2005-03-03 2011-12-27 Ibis Bioscience, Inc. Compositions for use in identification of papillomavirus
WO2006094238A2 (en) 2005-03-03 2006-09-08 Isis Pharmaceuticals, Inc. Compositions for use in identification of adventitious viruses
US7838502B2 (en) * 2005-05-06 2010-11-23 University Of Massachusetts Medical School Compositions and methods to modulate H. influenzae pathogenesis
US20100041738A1 (en) * 2005-06-20 2010-02-18 Avaris Ab Hybridization-stabilizing construct
CA2616281C (en) 2005-07-21 2014-04-22 Isis Pharmaceuticals, Inc. Methods for rapid identification and quantitation of mitochondrial dna variants
CA2617693A1 (en) 2005-08-17 2007-02-22 Medexis S.A. Composition and method for determination of ck19 expression
US20070087336A1 (en) * 2005-10-17 2007-04-19 Rangarajan Sampath Compositions for use in identification of influenza viruses
JP5030110B2 (ja) * 2005-12-21 2012-09-19 サムスン エレクトロニクス カンパニー リミテッド バイオメモリディスクドライブ装置及びそれを用いた分析方法
US8288354B2 (en) 2005-12-28 2012-10-16 The Scripps Research Institute Natural antisense and non-coding RNA transcripts as drug targets
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
JP5342881B2 (ja) 2006-01-27 2013-11-13 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 ６−修飾された二環式核酸類似体
CA2648132C (en) * 2006-04-03 2019-05-28 Santaris Pharma A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
US8088582B2 (en) * 2006-04-06 2012-01-03 Ibis Biosciences, Inc. Compositions for the use in identification of fungi
EP2023940B1 (en) 2006-05-05 2011-06-22 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of sglt2
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
JP5420412B2 (ja) 2006-09-14 2014-02-19 アイビス バイオサイエンシズ インコーポレイティッド 病原体の同定のための標的全ゲノム増幅方法
WO2008046911A2 (en) 2006-10-20 2008-04-24 Exiqon A/S Novel human micrornas associated with cancer
US8188255B2 (en) 2006-10-20 2012-05-29 Exiqon A/S Human microRNAs associated with cancer
JP2010510807A (ja) * 2006-11-27 2010-04-08 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 高コレステロール血症を治療するための方法
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP3536788A1 (en) 2006-12-21 2019-09-11 QIAGEN GmbH Microrna target site blocking oligos and uses thereof
JP5680304B2 (ja) 2007-02-23 2015-03-04 アイビス バイオサイエンシズ インコーポレイティッド 迅速な法医学的ｄｎａ分析法
US20080274458A1 (en) * 2007-05-01 2008-11-06 Latham Gary J Nucleic acid quantitation methods
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
US9598724B2 (en) 2007-06-01 2017-03-21 Ibis Biosciences, Inc. Methods and compositions for multiple displacement amplification of nucleic acids
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
DK2548962T3 (en) 2007-09-19 2016-04-11 Applied Biosystems Llc Sirna sequence-independent modification formats to reduce off-target phenotype effects in RNAI and stabilized forms thereof
KR101110013B1 (ko) * 2007-10-05 2012-02-29 (주)바이오니아 서열 내에 어베이직 부분을 포함하는 ｐｃｒ 증폭용프라이머
US8546556B2 (en) * 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
EP2229457A4 (en) 2007-12-14 2011-01-26 Minitube America Inc SEX-SPECIFIC SEPARATION OF SPERM AND EMBRYOES
WO2009100320A2 (en) 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
DK2285819T3 (da) 2008-04-04 2013-12-02 Isis Pharmaceuticals Inc Oligomere forbindelser omfattende neutralt bundne, terminale bicykliske nukleosider
US8148163B2 (en) 2008-09-16 2012-04-03 Ibis Biosciences, Inc. Sample processing units, systems, and related methods
WO2010033599A2 (en) 2008-09-16 2010-03-25 Ibis Biosciences, Inc. Mixing cartridges, mixing stations, and related kits, systems, and methods
EP2344893B1 (en) 2008-09-16 2014-10-15 Ibis Biosciences, Inc. Microplate handling systems and methods
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
WO2010040112A2 (en) * 2008-10-03 2010-04-08 Curna, Inc. Treatment of apolipoprotein-a1 related diseases by inhibition of natural antisense transcript to apolipoprotein-a1
ES2600781T3 (es) 2008-12-04 2017-02-10 Curna, Inc. Tratamiento para enfermedades relacionadas con el factor de crecimiento del endotelio vascular (vegf) mediante la inhibición de transcritos antisentido naturales de vegf
CN102361985B (zh) 2008-12-04 2017-06-20 库尔纳公司 通过抑制肿瘤抑制基因的天然反义转录物治疗肿瘤抑制基因相关性疾病
RU2620970C2 (ru) 2008-12-04 2017-05-30 КьюРНА,Инк., Лечение связанных с эритропоэтином (еро) заболеваний путем ингибирования природного антисмыслового транскрипта к еро
WO2010080493A2 (en) * 2008-12-18 2010-07-15 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, Centers For Disease Control And Prevention Real time polymerase chain reaction detection of legionella pneumophila and differentiation form other legionella species
AU2010207826B2 (en) 2009-02-02 2013-10-03 Qiagen Gmbh Method for quantification of small RNA species
EP2396803A4 (en) 2009-02-12 2016-10-26 Ibis Biosciences Inc IONIZATION PROBE ASSEMBLIES
WO2010093904A2 (en) 2009-02-12 2010-08-19 Curna, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
KR101805199B1 (ko) * 2009-02-12 2017-12-05 큐알엔에이, 인크. 신경교세포 유래된 신경영양성 인자 (ｇｄｎｆ)에 대한 자연 안티센스 전사체의 저해에 의한 신경교세포 유래된 신경영양성 인자 (ｇｄｎｆ) 관련된 질환의 치료
EP2408919B1 (en) 2009-03-16 2017-10-18 CuRNA, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
CA2755404C (en) 2009-03-17 2020-03-24 Curna, Inc. Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
EP3249053A1 (en) 2009-03-27 2017-11-29 Life Technologies Corporation Methods, compositions, and kits for detecting allelic variants
CN102459596B (zh) 2009-05-06 2016-09-07 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
KR101722541B1 (ko) 2009-05-06 2017-04-04 큐알엔에이, 인크. Ｔｔｐ에 대한 천연 안티센스 전사체의 억제에 의한 트리스테트라프롤린 관련된 질환의 치료
ES2618572T3 (es) 2009-05-08 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con la familia de la distrofina mediante inhibición de un transcrito antisentido natural para la familia de dmd
WO2010135329A2 (en) 2009-05-18 2010-11-25 Curna, Inc. Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
CA2762987A1 (en) 2009-05-22 2010-11-25 Joseph Collard Treatment of transcription factor e3 (tfe3) and insulin receptor substrate 2 (irs2) related diseases by inhibition of natural antisense transcript to tfe3
WO2010135692A2 (en) 2009-05-22 2010-11-25 Asuragen, Inc. Mirna biomarkers of prostate disease
KR101704988B1 (ko) 2009-05-28 2017-02-08 큐알엔에이, 인크. 항바이러스 유전자에 대한 천연 안티센스 전사체의 억제에 의한 항바이러스 유전자 관련된 질환의 치료
ES2804764T3 (es) 2009-06-01 2021-02-09 Halo Bio Rnai Therapeutics Inc Polinucleótidos para la interferencia de ARN multivalente, composiciones y métodos de uso de los mismos
ES2629339T3 (es) 2009-06-16 2017-08-08 Curna, Inc. Tratamiento de enfermedades relacionadas con la paraoxonasa 1 (pon1) por inhibición de transcrito antisentido natural a pon1
CA2765700C (en) 2009-06-16 2021-01-05 Opko Curna, Llc Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
ES2618894T3 (es) 2009-06-24 2017-06-22 Curna, Inc. Tratamiento de enfermedades relacionadas con el receptor del factor de necrosis tumoral 2 (tnfr2) por inhibición del transcrito natural antisentido para tnfr2
CN102482672B (zh) 2009-06-26 2016-11-09 库尔纳公司 通过抑制唐氏综合征基因的天然反义转录物治疗唐氏综合征基因相关疾病
EP2290102A1 (en) 2009-07-08 2011-03-02 Administración General De La Communidad Autónoma De Euskadi Methods for the diagnosis of multiple sclerosis based on its microRNA expression profiling
US9194877B2 (en) 2009-07-17 2015-11-24 Ibis Biosciences, Inc. Systems for bioagent indentification
US8950604B2 (en) 2009-07-17 2015-02-10 Ibis Biosciences, Inc. Lift and mount apparatus
US20120252869A1 (en) 2009-07-24 2012-10-04 Opko Curna, Llc Treatment of sirtuin (sirt) related diseases by inhibition of natural antisense transcript to a sirtuin (sirt)
ES2585360T3 (es) 2009-08-05 2016-10-05 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen de la insulina (INS) por inhibición de la transcripción antisentido natural en un gen de la insulina (INS)
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
ES2599986T3 (es) 2009-08-11 2017-02-06 Curna, Inc. Tratamiento de enfermedades relacionadas con adiponectina (ADIPOQ) mediante la inhibición de un transcrito antisentido natural de una adiponectina (ADIPOQ)
EP2789693B1 (en) 2009-08-13 2017-10-04 Life Technologies Corporation Amelogenin SNP on chromosome X
CN102482670B (zh) 2009-08-21 2018-06-15 库尔纳公司 通过抑制‘hsp70-相互作用蛋白的c末端’(chip)的天然反义转录物而治疗chip相关疾病
EP2470657B1 (en) 2009-08-25 2019-10-23 CuRNA, Inc. Treatment of 'iq motif containing gtpase activating protein' (iqgap) related diseases by inhibition of natural antisense transcript to iqgap
EP2470897A4 (en) 2009-08-28 2013-05-29 Asuragen Inc MICRO-RNA BIOMARKERS OF PULMONARY DISEASE
WO2011032034A2 (en) 2009-09-10 2011-03-17 University Of Idaho Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids
DK2480669T3 (en) 2009-09-25 2018-02-12 Curna Inc TREATMENT OF FILAGGRIN- (FLG) RELATED DISEASES BY MODULATING FLG EXPRESSION AND ACTIVITY
EP2488668B1 (en) 2009-10-15 2023-07-26 Life Technologies Corporation Novel human single nucleotide polymorphisms
ES2628739T3 (es) 2009-10-15 2017-08-03 Ibis Biosciences, Inc. Amplificación por desplazamiento múltiple
AU2010319314C1 (en) 2009-11-13 2016-09-01 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
CA2782366A1 (en) 2009-12-16 2011-07-14 Opko Curna, Llc Treatment of membrane bound transcription factor peptidase, site 1 (mbtps1) related diseases by inhibition of natural antisense transcript to mbtps1
NO2516648T3 (https=) 2009-12-23 2018-04-07
RU2619185C2 (ru) 2009-12-23 2017-05-12 Курна, Инк. Лечение заболеваний, связанных с разобщающим белком 2 (ucp2), путем ингибирования природного антисмыслового транскрипта к ucp2
US8921334B2 (en) 2009-12-29 2014-12-30 Curna, Inc. Treatment of nuclear respiratory factor 1 (NRF1) related diseases by inhibition of natural antisense transcript to NRF1
US8962585B2 (en) 2009-12-29 2015-02-24 Curna, Inc. Treatment of tumor protein 63 (p63) related diseases by inhibition of natural antisense transcript to p63
DK2519632T3 (en) 2009-12-31 2018-07-23 Curna Inc TREATMENT OF INSULIN RECEPTOR SUBSTRATE 2- (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPTION TO IRS2 AND TRANSCRIPTION FACTOR E3 (TFE3)
DK2521784T3 (en) 2010-01-04 2018-03-12 Curna Inc TREATMENT OF INTERFERON REGULATORY FACTOR 8- (IRF8) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IRF8
KR101853509B1 (ko) 2010-01-06 2018-04-30 큐알엔에이, 인크. 췌장 발달 유전자에 대한 천연 안티센스 전사체의 억제에 의한 췌장 발달 유전자와 관련된 질환의 치료
KR101854926B1 (ko) 2010-01-11 2018-05-04 큐알엔에이, 인크. 성 호르몬 결합 글로불린 (ｓｈｂｇ)에 대한 자연 안티센스 전사체의 저해에 의한 성 호르몬 결합 글로불린 (ｓｈｂｇ) 관련된 질환의 치료
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
DK2529015T3 (en) 2010-01-25 2018-02-26 Curna Inc TREATMENT OF RNASE H1-RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO RNASE H1
RU2608496C2 (ru) 2010-02-22 2017-01-18 Курна, Инк. Лечение заболеваний, связанных с пирролин-5 карбоксилатредуктазой 1(pycr1), путем ингибирования природного антисмыслового транскрипта к pycr1
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
EP2545173A2 (en) 2010-03-12 2013-01-16 Sarepta Therapeutics, Inc. Antisense modulation of nuclear hormone receptors
EP3210611B1 (en) 2010-03-12 2019-08-21 The Brigham and Women's Hospital, Inc. Methods of treating vascular inflammatory disorders
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
US9506057B2 (en) 2010-03-26 2016-11-29 Integrated Dna Technologies, Inc. Modifications for antisense compounds
AU2011230496B2 (en) 2010-03-26 2015-09-17 Integrated Dna Technologies, Inc. Methods for enhancing nucleic acid hybridization
US8980856B2 (en) 2010-04-02 2015-03-17 Curna, Inc. Treatment of colony-stimulating factor 3 (CSF3) related diseases by inhibition of natural antisense transcript to CSF3
KR101900962B1 (ko) 2010-04-09 2018-09-20 큐알엔에이, 인크. 섬유아세포 성장 인자 21 (ｆｇｆ21)에 대한 자연 안티센스 전사체의 저해에 의한 섬유아세포 성장 인자 21 (ｆｇｆ21) 관련된 질환의 치료
US8980253B2 (en) 2010-04-26 2015-03-17 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of cysteinyl-tRNA synthetase
CA2797362C (en) 2010-04-27 2020-12-08 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of isoleucyl trna synthetases
KR101869570B1 (ko) 2010-04-28 2018-06-20 아이오니스 파마수티컬즈, 인코포레이티드 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물
WO2011139853A2 (en) 2010-04-28 2011-11-10 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of alanyl trna synthetases
CA2797374C (en) 2010-04-29 2021-02-16 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of asparaginyl trna synthetases
CA2797393C (en) 2010-04-29 2020-03-10 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of valyl trna synthetases
US9212390B2 (en) 2010-04-30 2015-12-15 Exiqon A/S In situ hybridization method and buffer
CN103140233B (zh) 2010-05-03 2017-04-05 Atyr 医药公司 与甲硫氨酰‑tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的发现
US8961961B2 (en) 2010-05-03 2015-02-24 a Tyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related protein fragments of arginyl-tRNA synthetases
RU2018110642A (ru) 2010-05-03 2019-02-27 Курна, Инк. Лечение заболеваний, связанных с сиртуином (sirt), путем ингибирования природного антисмыслового транскрипта к сиртуину (sirt)
CN103096912A (zh) 2010-05-03 2013-05-08 Atyr医药公司 与苯丙氨酰-α-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
JP6008844B2 (ja) 2010-05-04 2016-10-19 エータイアー ファーマ， インコーポレイテッド ｐ３８ＭＵＬＴＩ−ｔＲＮＡ合成酵素複合体のタンパク質フラグメントに関連した治療用、診断用および抗体組成物の革新的発見
ES2816898T3 (es) 2010-05-13 2021-04-06 Sarepta Therapeutics Inc Compuestos que modulan la actividad de señalización de las interleucinas 17 y 23
EP2568996B1 (en) 2010-05-14 2017-10-04 aTyr Pharma, Inc. Therapeutic, diagnostic, and antibody compositions related to protein fragments of phenylalanyl-beta-trna synthetases
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
CN103096914B (zh) 2010-05-17 2015-08-12 Atyr医药公司 与亮氨酰-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的创新发现
CA2799207C (en) 2010-05-26 2019-03-26 Curna, Inc. Treatment of atonal homolog 1 (atoh1) related diseases by inhibition of natural antisense transcript to atoh1
JP5917497B2 (ja) 2010-05-26 2016-05-18 カッパーアールエヌエー，インコーポレイテッド メチオニンスルホキシドレダクターゼａ（ｍｓｒａ）に対する天然アンチセンス転写物の阻害によるｍｓｒａ関連疾患の治療
CA2800375C (en) 2010-05-27 2021-03-09 Atyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glutaminyl-trna synthetases
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011163499A2 (en) 2010-06-23 2011-12-29 Opko Curna, Llc Treatment of sodium channel, voltage-gated, alpha subunit (scna) related diseases by inhibition of natural antisense transcript to scna
EP2593125B1 (en) 2010-07-12 2017-11-01 aTyr Pharma, Inc. Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of glycyl-trna synthetases
CN103068982B (zh) 2010-07-14 2017-06-09 库尔纳公司 通过抑制盘状大同系物(dlg)的天然反义转录物而治疗dlg相关疾病
AU2011293294B2 (en) 2010-08-25 2016-03-24 Pangu Biopharma Limited Innovative discovery of therapeutic, diagnostic, and antibody compositions related to protein fragments of Tyrosyl-tRNA synthetases
AU2011299233B2 (en) 2010-09-07 2016-09-15 Integrated Dna Technologies, Inc. Modifications for antisense compounds
WO2012040403A1 (en) 2010-09-21 2012-03-29 Life Technologies Corporation Se33 mutations impacting genotype concordance
ES2640755T3 (es) 2010-10-06 2017-11-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la Sialidasa 4 (neu4) mediante inhibición del transcrito antisentido natural al gen neu4
US20140031250A1 (en) 2010-10-07 2014-01-30 David Tsai Ting Biomarkers of Cancer
US9222088B2 (en) 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
EP3260540A1 (en) 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
ES2657590T3 (es) 2010-11-23 2018-03-06 Curna, Inc. Tratamiento de enfermedades relacionadas con nanog mediante inhibición del transcrito antisentido natural a nanog
EP2663323B1 (en) 2011-01-14 2017-08-16 The General Hospital Corporation Methods targeting mir-128 for regulating cholesterol/lipid metabolism
EP2718439B1 (en) 2011-06-09 2017-08-09 CuRNA, Inc. Treatment of frataxin (fxn) related diseases by inhibition of natural antisense transcript to fxn
BR112013033488B8 (pt) 2011-06-27 2022-11-22 Eisai R&D Man Co Ltd Métodos para facilitar o diagnóstico do comprometimento cognitivo devido a doença de alzheimer ou comprometimento cognitivo leve (mci)e kit
EP2546358A1 (en) 2011-07-15 2013-01-16 Laboratorios Del. Dr. Esteve, S.A. Methods and reagents for efficient control of HIV progression
EP2732035A2 (en) 2011-07-15 2014-05-21 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
KR101991980B1 (ko) 2011-09-06 2019-06-21 큐알엔에이, 인크. 소형 분자로 전압-개폐된 나트륨 채널 (SCNxA)의 알파 아단위에 관련된 질환의 치료
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CA2851280C (en) 2011-10-11 2021-05-18 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
EP2788487B1 (en) 2011-12-08 2018-04-04 Sarepta Therapeutics, Inc. Oligonucleotide analogues targeting human lmna
JP2015502365A (ja) * 2011-12-12 2015-01-22 オンコイミューニン，インコーポレイティド オリゴヌクレオチドのイン−ビボ送達
US20150031750A1 (en) 2012-03-15 2015-01-29 The Scripps Research Institute Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
ES2434853B1 (es) 2012-06-12 2014-09-30 Fundación Centro Nacional De Investigaciones Cardiovasculares Carlos Iii Marcador molecular de potencia terapéutica de células madre mesenquimales humanas y sus usos
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US10086093B2 (en) 2013-02-28 2018-10-02 The General Hospital Corporation miRNA profiling compositions and methods of use
WO2014164874A2 (en) 2013-03-13 2014-10-09 Becton, Dickinson And Company Methods and compositions for modulation of amplification efficiency
US9273349B2 (en) 2013-03-14 2016-03-01 Affymetrix, Inc. Detection of nucleic acids
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
DK2991656T3 (da) 2013-05-01 2020-03-23 Ionis Pharmaceuticals Inc Sammensætninger og fremgangsmåder til modulering af apolipoprotein c-iii-ekspression
ES2523016B1 (es) 2013-05-20 2015-09-09 3P Biopharmaceuticals Vectores alfavirales y líneas celulares para la producción de proteínas recombinantes
CA2951816A1 (en) * 2013-06-12 2014-12-18 Oncoimmunin, Inc. Systemic in vivo delivery of oligonucleotides
DE102013011304A1 (de) * 2013-07-02 2015-01-22 Technische Universität Dresden Verfahren und Anordnung zur Erfassung von Bindungsereignissen von Molekülen
JP6618910B2 (ja) 2013-09-05 2019-12-11 サレプタ セラピューティクス，インコーポレイテッド 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
AU2014375224A1 (en) 2014-01-05 2016-07-28 Biomirna Holdings Ltd Lung cancer determinations using miRNA ratios
WO2015120382A1 (en) 2014-02-07 2015-08-13 The Johns Hopkins University Predicting response to epigenetic drug therapy
WO2015126523A1 (en) * 2014-02-21 2015-08-27 Clontech Laboratories, Inc. Spin columns comprising poly(acid) membrane separation matrices, and methods of making and using the same
RS60707B1 (sr) 2014-04-01 2020-09-30 Biogen Ma Inc Kompozicije za modulaciju ekspresije sod-1
DK3137476T3 (da) 2014-04-28 2019-11-18 Ionis Pharmaceuticals Inc Linker-modificerede oligomerforbindelser
SG11201608502TA (en) 2014-05-01 2016-11-29 Ionis Pharmaceuticals Inc Compositions and methods for modulating complement factor b expression
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US10301624B2 (en) 2014-06-25 2019-05-28 The General Hospital Corporation Targeting human satellite II (HSATII)
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
EP3221445B1 (en) 2014-11-20 2021-07-14 The Regents of The University of California Compositions and methods related to hematologic recovery
WO2016094330A2 (en) 2014-12-08 2016-06-16 20/20 Genesystems, Inc Methods and machine learning systems for predicting the liklihood or risk of having cancer
WO2016092045A1 (en) 2014-12-11 2016-06-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and kits for predicting medically refractory acute severe colitis
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US10961532B2 (en) 2015-04-07 2021-03-30 The General Hospital Corporation Methods for reactivating genes on the inactive X chromosome
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
WO2016177774A1 (en) 2015-05-04 2016-11-10 Academisch Medisch Centrum Method of quantifying mirnas using normalization
US10849917B2 (en) 2015-06-01 2020-12-01 Sarepta Therapeutics, Inc. Antisense-induced exon exclusion in type VII collagen
CA2990852A1 (en) 2015-06-26 2016-12-29 Beth Israel Deaconess Medical Center, Inc. Cancer therapy targeting tetraspanin 33 (tspan33) in myeloid derived suppressor cells
JP2018525037A (ja) 2015-08-24 2018-09-06 ハロー−バイオ・アールエヌエーアイ・セラピューティックス、インコーポレイテッド 遺伝子発現の調節のためのポリヌクレオチドナノ粒子及びその使用
EP3858993A1 (en) 2015-10-09 2021-08-04 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
EP3359685B1 (en) 2015-10-09 2026-01-28 University Of Southampton Modulation of gene expression for deregulated protein expression
EP4089175A1 (en) 2015-10-13 2022-11-16 Duke University Genome engineering with type i crispr systems in eukaryotic cells
EP4454637A3 (en) 2015-11-16 2025-01-08 Olix Pharmaceuticals, Inc. Treatment of age-related macular degeneration using rna complexes that target myd88 or tlr3
EA201891317A3 (ru) 2015-11-30 2019-04-30 Дьюк Юниверсити Терапевтические мишени для коррекции гена дистрофина человека с помощью редактирования генов и способы их применения
US11058709B1 (en) 2015-12-04 2021-07-13 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
KR102825946B1 (ko) 2016-02-02 2025-06-27 올릭스 주식회사 IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
US11234996B2 (en) 2016-02-25 2022-02-01 The Brigham And Women's Hospital, Inc. Treatment methods for fibrosis targeting SMOC2
AU2017234678A1 (en) 2016-03-16 2018-08-16 Ionis Pharmaceuticals, Inc. Methods of modulating KEAP1
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CA3021267A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
CN109477109B (zh) 2016-04-29 2022-09-23 萨勒普塔医疗公司 靶向人lmna的寡核苷酸类似物
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MA49775A (fr) 2016-05-24 2020-06-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
MA45183A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
TWI737736B (zh) 2016-05-24 2021-09-01 美商薩羅塔治療公司 製備磷醯二胺嗎啉代寡聚物之方法
EP3471781A4 (en) 2016-06-17 2020-05-06 Ionis Pharmaceuticals, Inc. MODULATION OF GYS1 EXPRESSION
TW201811807A (zh) 2016-06-30 2018-04-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
WO2018017754A1 (en) 2016-07-19 2018-01-25 Duke University Therapeutic applications of cpf1-based genome editing
WO2018017814A1 (en) 2016-07-20 2018-01-25 President And Fellows Of Harvard College Peptidoglycan glycosyltransferase inhibitors of sed proteins for treating bacterial infections
WO2018053142A2 (en) 2016-09-14 2018-03-22 President And Fellows Of Harvard College Methods and compositions for modulating erythropoiesis
CA3037046A1 (en) 2016-10-31 2018-05-03 University Of Massachusetts Targeting microrna-101-3p in cancer therapy
WO2018102745A1 (en) 2016-12-02 2018-06-07 Cold Spring Harbor Laboratory Modulation of lnc05 expression
WO2018112033A1 (en) 2016-12-13 2018-06-21 President And Fellows Of Harvard College Methods and compositions for targeting tumor-infiltrating tregs
SI3554554T1 (sl) 2016-12-19 2023-02-28 Sarepta Therapeutics, Inc. Oligomerni konjugati, ki preskakujejo ekson za mišično distrofijo
IL297528A (en) 2016-12-19 2022-12-01 Sarepta Therapeutics Inc Exon-skipping oligomer conjugates for muscular dystrophy
SMT202200366T1 (it) 2016-12-19 2022-11-18 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
US11180756B2 (en) 2017-03-09 2021-11-23 Ionis Pharmaceuticals Morpholino modified oligomeric compounds
EP3609521A4 (en) 2017-04-14 2021-06-16 University of Massachusetts TARGETING OF CELL TROPISM RECEPTORS TO INHIBIT INFECTION BY THE CYTOMEGALOVIRUS
CN110536694A (zh) 2017-04-20 2019-12-03 Atyr 医药公司 用于治疗肺部炎症的组合物和方法
US11197884B2 (en) 2017-08-18 2021-12-14 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
WO2019067975A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
WO2019067979A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
JP2020536058A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
WO2019079637A2 (en) 2017-10-18 2019-04-25 Sarepta Therapeutics, Inc. ANTISENSE OLIGOMERIC COMPOUNDS
WO2019144061A1 (en) 2018-01-19 2019-07-25 Duke University Genome engineering with crispr-cas systems in eukaryotes
MX2020009765A (es) * 2018-03-20 2021-01-08 Tokyo Inst Tech Oligonucleotido antisentido reducido en toxicidad.
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US12582702B2 (en) 2018-05-11 2026-03-24 University Of Massachusetts Methods for improving leptin sensitivity for the treatment of obesity and diabetes
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3806868A4 (en) 2018-06-13 2022-06-22 Sarepta Therapeutics, Inc. EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY
CN110669839B (zh) * 2018-07-03 2022-04-15 北京福安华生物科技有限公司 一种检测egfr基因t790m突变的人工模拟分子信标与试剂盒
TW202020153A (zh) 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
EP3833762A4 (en) 2018-08-09 2022-09-28 Verseau Therapeutics, Inc. Oligonucleotide compositions for targeting ccr2 and csf1r and uses thereof
EP3843845B1 (en) 2018-08-29 2026-03-11 University Of Massachusetts Inhibition of protein kinases to treat friedreich ataxia
US11273137B2 (en) 2018-09-04 2022-03-15 Board Of Trustees Of Michigan State University Methods and compositions to prevent and treat disorders associated with mutations in the ODC1 gene
EP3846824B1 (en) 2018-09-07 2025-06-18 The General Hospital Corporation Compositions and methods for immune checkpoint inhibition
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
BR112021008967A2 (pt) 2018-11-15 2021-08-17 Ionis Pharmaceuticals, Inc. moduladores da expressão de irf5
MX2021006737A (es) 2018-12-13 2021-09-23 Sarepta Therapeutics Inc Conjugados de oligómeros de salto de exones para distrofia muscular.
CN112654714B (zh) 2018-12-17 2024-10-11 伊卢米纳剑桥有限公司 用于测序的引物寡核苷酸
TW202540426A (zh) 2019-02-27 2025-10-16 美商Ionis製藥公司 Malat1表現之調節劑
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
CN114040799A (zh) 2019-03-12 2022-02-11 哈佛大学校长及研究员协会 治疗癌症的方法和组合物
EP3719144A1 (en) 2019-04-05 2020-10-07 Fundación para la Investigación Biomédica del Hospital Universitario de la Paz (FIBHULP) Mir-151a-3p as an universal endogenous control for exosome cargo normalization
EP3955966A1 (en) 2019-04-18 2022-02-23 Sarepta Therapeutics, Inc. Compositions for treating muscular dystrophy
WO2020226960A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
US20220226269A1 (en) 2019-06-12 2022-07-21 President And Fellows Of Harvard College Methods and compositions for modulation of an interspecies gut bacterial pathway for levodopa metabolism
EP3999664A1 (en) 2019-07-19 2022-05-25 Fundación para la Investigación Biomédica del Hospital Universitario de la Paz (FIBHULP) Method for determining the response to treatment of a patient affected by non-small cell lung carcinoma (nsclc)
EP3956450B1 (en) 2019-07-26 2025-08-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
EP4013767A4 (en) 2019-08-15 2023-10-25 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
CN115485385A (zh) 2020-03-04 2022-12-16 瑞泽恩制药公司 用于使肿瘤细胞对免疫疗法敏感的方法和组合物
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
US20230285357A1 (en) 2020-05-27 2023-09-14 The Regents Of The University Of California Compositions and methods for transdifferentiating cells
WO2022056454A2 (en) 2020-09-14 2022-03-17 President And Fellows Of Harvard College Methods and compositions for treating hpv-positive cancers
IL301306A (en) 2020-09-16 2023-05-01 Harvard College Methods of treating an individual that has failed an anti-pd-1/anti-pd-l1 therapy
MX2024003690A (es) 2021-09-30 2024-06-19 Sarepta Therapeutics Inc Oligonucleótidos antisentido que tienen una o más unidades abásicas.
JP2024539223A (ja) 2021-10-22 2024-10-28 サレプタ セラピューティクス， インコーポレイテッド 末梢ミエリンタンパク質２２関連疾患の処置のためのモルフォリノオリゴマー
EP4433592A1 (en) 2021-11-18 2024-09-25 Circularis Biotechnologies, Inc. Compositions and methods for production of circular nucleic acid molecules
EP4437106A2 (en) 2021-11-24 2024-10-02 Prime Medicine, Inc. Methods and compositions for inhibiting mismatch repair
WO2023150181A1 (en) 2022-02-01 2023-08-10 President And Fellows Of Harvard College Methods and compositions for treating cancer
IL318625A (en) 2022-07-29 2025-03-01 Regeneron Pharma Compositions and methods for transferrin receptor (TFR)-mediated delivery to brain and muscle
US20260049309A1 (en) 2022-08-15 2026-02-19 Novo Nordisk A/S Regulation of activity of rnai molecules
TW202424186A (zh) 2022-08-25 2024-06-16 美商生命編輯治療學公司 Rna引導核酸酶中介基因編輯用之具鎖核酸引導 rna之化學修飾
WO2024064237A2 (en) 2022-09-21 2024-03-28 Sarepta Therapeutics, Inc. Dmd antisense oligonucleotide-mediated exon skipping efficiency
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
AU2023379457A1 (en) 2022-11-14 2025-05-15 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
JP2024129413A (ja) * 2023-03-13 2024-09-27 Ｋｍバイオロジクス株式会社 Ｔ細胞受容体遺伝子再構成断片（ＴＲＥＣ）、Ｉｇκ鎖遺伝子再構成断片（ＫＲＥＣ）およびＳＭＮ１遺伝子の量を定量ＰＣＲにより同時に測定する方法およびそのための新規プライマー
US20250066776A1 (en) 2023-04-27 2025-02-27 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of chronic kidney disease
WO2024233864A2 (en) 2023-05-10 2024-11-14 Dicerna Pharmaceuticals, Inc. Galnac-conjugated rnai oligonucleotides
WO2025038750A2 (en) 2023-08-14 2025-02-20 President And Fellows Of Harvard College Methods and compositions for treating cancer
WO2025054459A1 (en) 2023-09-08 2025-03-13 Dicerna Pharmaceuticals, Inc. Rnai oligonucleotide conjugates
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies

Family Cites Families (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4806463A (en) * 1986-05-23 1989-02-21 Worcester Foundation For Experimental Biology Inhibition of HTLV-III by exogenous oligonucleotides
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
DK0820483T3 (da) 1995-04-07 2001-01-02 Mogens Havsteen Jakobsen Fremgangsmåde til fotokemisk immobilisering af ligander under anvendelse af quinoner
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
JP2002515514A (ja) * 1998-05-21 2002-05-28 アイシス・ファーマシューティカルス・インコーポレーテッド オリゴヌクレオチドの局所送逹のための組成物及び方法
CN1350542A (zh) 1999-03-18 2002-05-22 埃克西库恩公司 木－lna类似物
JP4768132B2 (ja) 1999-03-24 2011-09-07 エクシコン エ／エス ［２．２．１］ビシクロヌクレオシドの改良された製法
EP1178999B1 (en) 1999-05-04 2007-03-14 Santaris Pharma A/S L-ribo-lna analogues
US6083482A (en) * 1999-05-11 2000-07-04 Icn Pharmaceuticals, Inc. Conformationally locked nucleosides and oligonucleotides
US7205105B2 (en) * 1999-12-08 2007-04-17 Epoch Biosciences, Inc. Real-time linear detection probes: sensitive 5′-minor groove binder-containing probes for PCR analysis
US6352858B1 (en) * 2000-09-11 2002-03-05 Isis Pharmaceuticals, Inc. Antisense modulation of BTAK expression
US6440739B1 (en) * 2001-07-17 2002-08-27 Isis Pharmaceuticals, Inc. Antisense modulation of glioma-associated oncogene-2 expression
US20040092462A1 (en) * 2002-11-13 2004-05-13 Isis Pharmaceuticals Inc. Modulation of endothelial differentiation gene 2 expression
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20210010064A1 (en) * 2018-03-14 2021-01-14 The Trustees Of The University Of Pennsylvania Enrichment of nucleic acids

Also Published As

Publication number Publication date
AU2002334307A1 (en) 2003-03-18
EP1446412B1 (en) 2012-03-07
WO2003020739A2 (en) 2003-03-13
US20030224377A1 (en) 2003-12-04
JP2005514005A (ja) 2005-05-19
WO2003020739A3 (en) 2004-06-03
EP1446412A2 (en) 2004-08-18
CA2459347A1 (en) 2003-03-13
US7060809B2 (en) 2006-06-13

Similar Documents

Publication Publication Date Title
CA2459347C (en) Locked nucleic acid (lna) compositions and uses thereof
US9464106B2 (en) Oligonucleotides useful for detecting and analyzing nucleic acids of interest
EP1015469B2 (en) Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
US6670461B1 (en) Oligonucleotide analogues
US8153365B2 (en) Oligonucleotide analogues
EP1178999B1 (en) L-ribo-lna analogues
US7144995B2 (en) Fluorescent nitrogenous base and nucleosides incorporating same
WO2003039523A2 (en) OLIGONUCLEOTIDES MODIFIED WITH NOVEL α-L-RNA ANALOGUES
JP2003508405A (ja) ２−アザプリン化合物及びその使用
WO2004035819A2 (en) Oligonucleotide analogues for detecting and analyzing nucleic acids
AU2002325599B2 (en) Oligonucleotide analogues

Legal Events

Date Code Title Description
EEER Examination request
MKEX Expiry

Effective date: 20220906