SG10202112949PA - Reducing intron retention - Google Patents

Reducing intron retention

Info

Publication number
SG10202112949PA
SG10202112949PA SG10202112949PA SG10202112949PA SG10202112949PA SG 10202112949P A SG10202112949P A SG 10202112949PA SG 10202112949P A SG10202112949P A SG 10202112949PA SG 10202112949P A SG10202112949P A SG 10202112949PA SG 10202112949P A SG10202112949P A SG 10202112949PA
Authority
SG
Singapore
Prior art keywords
intron retention
reducing
reducing intron
retention
intron
Prior art date
Application number
SG10202112949PA
Other languages
English (en)
Inventor
Igor Vorechovsky
Jana Kralovicova
Original Assignee
Univ Southampton
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 Univ Southampton filed Critical Univ Southampton
Publication of SG10202112949PA publication Critical patent/SG10202112949PA/en

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/113Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/33Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Analytical Chemistry (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
SG10202112949PA 2014-06-16 2015-06-16 Reducing intron retention SG10202112949PA (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
GBGB1410693.4A GB201410693D0 (en) 2014-06-16 2014-06-16 Splicing modulation

Publications (1)

Publication Number Publication Date
SG10202112949PA true SG10202112949PA (en) 2021-12-30

Family

ID=51266659

Family Applications (2)

Application Number Title Priority Date Filing Date
SG11201610480XA SG11201610480XA (en) 2014-06-16 2015-06-16 Reducing intron retention
SG10202112949PA SG10202112949PA (en) 2014-06-16 2015-06-16 Reducing intron retention

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG11201610480XA SG11201610480XA (en) 2014-06-16 2015-06-16 Reducing intron retention

Country Status (12)

Country Link
US (6) US9745577B2 (fr)
EP (2) EP3155124B1 (fr)
JP (3) JP7043169B2 (fr)
KR (1) KR102638276B1 (fr)
CN (1) CN107073137A (fr)
AU (2) AU2015275870B2 (fr)
CA (1) CA2951208A1 (fr)
ES (1) ES2907033T3 (fr)
GB (1) GB201410693D0 (fr)
IL (2) IL295051B2 (fr)
SG (2) SG11201610480XA (fr)
WO (1) WO2015193651A1 (fr)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
CN107109411B (zh) * 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
ES2879995T3 (es) 2015-12-10 2021-11-23 Ptc Therapeutics Inc Métodos para el tratamiento de la enfermedad de Huntington
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
CA3005131A1 (fr) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Oligomeres antisens pour le traitement de la sclerose tubereuse de bourneville
JP7036723B2 (ja) * 2015-12-14 2022-03-15 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
US20190330626A1 (en) * 2016-07-15 2019-10-31 Ionis Pharmaceuticals, Inc. Compounds and methods for use in dystrophin transcript
JP7211933B2 (ja) * 2016-07-15 2023-01-24 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 転写プロセシングの調節のための化合物及び方法
WO2018226622A1 (fr) 2017-06-05 2018-12-13 Ptc Therapeutics, Inc. Composés permettant de traiter la maladie de huntington
CA3067592A1 (fr) 2017-06-28 2019-01-03 Ptc Therapeutics, Inc. Methodes de traitement de la maladie de huntington
EP3644996B1 (fr) 2017-06-28 2023-07-26 PTC Therapeutics, Inc. Procédés de traitement de la maladie de huntington
WO2019028440A1 (fr) 2017-08-04 2019-02-07 Skyhawk Therapeutics, Inc. Méthodes et compositions permettant de moduler l'épissageé
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
WO2019111791A1 (fr) * 2017-12-07 2019-06-13 第一三共株式会社 Oligonucléotide antisens apte à annuler la rétention d'introns dans le gène de la dystrophine
JP7399870B2 (ja) 2018-03-27 2023-12-18 ピーティーシー セラピューティクス, インコーポレイテッド ハンチントン病を処置するための化合物
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2020005873A1 (fr) 2018-06-27 2020-01-02 Ptc Therapeutics, Inc. Composés hétérocycliques et hétéroaryles pour le traitement de la maladie de huntington
JP7476199B2 (ja) 2018-08-20 2024-04-30 ロジコン, インコーポレイテッド Scn1a脳症の治療のためのscn2aを標的とするアンチセンスオリゴヌクレオチド
JP2022521467A (ja) 2019-02-05 2022-04-08 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
JP2022519323A (ja) 2019-02-06 2022-03-22 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN110718265B (zh) * 2019-09-05 2021-02-26 复旦大学 靶向生物毒素的g-四联体式核酸适配体三级结构预测方法
JPWO2021112106A1 (fr) * 2019-12-02 2021-06-10
PE20221913A1 (es) 2020-02-28 2022-12-23 Ionis Pharmaceuticals Inc Compuestos y metodos para modular smn2
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc opa1 antisense oligomers for the treatment of conditions and diseases
WO2023288111A2 (fr) * 2021-07-16 2023-01-19 Aptah Bio, Inc. Compositions polynucléotidiques et procédés de régulation de l'expression génique
WO2024155739A1 (fr) * 2023-01-18 2024-07-25 Aptah Bio, Inc. Compositions polynucléotidiques et méthodes de traitement des maladies neurodégénératives
WO2024155740A1 (fr) * 2023-01-18 2024-07-25 Aptah Bio, Inc. Compositions polynucléotidiques et méthodes de traitement du cancer

Family Cites Families (254)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4476116A (en) 1982-12-10 1984-10-09 Syntex (U.S.A.) Inc. Polypeptides/chelating agent nasal compositions having enhanced peptide absorption
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
EP0639582B1 (fr) 1985-03-15 1998-09-16 Antivirals Inc. Réactif et procédé d'analyse de polynucléotides
US4866042A (en) 1987-11-18 1989-09-12 Neuwelt Edward A Method for the delivery of genetic material across the blood brain barrier
US6294520B1 (en) 1989-03-27 2001-09-25 Albert T. Naito Material for passage through the blood-brain barrier
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US5151510A (en) 1990-04-20 1992-09-29 Applied Biosystems, Inc. Method of synethesizing sulfurized oligonucleotide analogs
DK0549615T3 (da) 1990-08-13 2006-07-03 Isis Pharmaceuticals Inc Sukkermodificerede oligonukleotider der påviser og modulerer genekspression
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
AU4684993A (en) 1992-07-23 1994-02-14 Isis Pharmaceuticals, Inc. Novel 2'-o-alkyl nucleosides and phosphoramidites processes for the preparation and uses thereof
WO1994008026A1 (fr) 1992-09-25 1994-04-14 Rhone-Poulenc Rorer S.A. Vecteurs d'adenovirus pour le transfert de genes etrangers dans des cellules du systeme nerveux central, en particulier du cerveau
CN1123038A (zh) 1993-05-11 1996-05-22 北卡罗来纳大学查珀尔希尔分校 阻止异常剪接的反义寡核苷酸及其使用方法
US6232452B1 (en) 1993-12-24 2001-05-15 Julian R. Sampson Tuberous sclerosis 2 gene and uses thereof
US5656612A (en) 1994-05-31 1997-08-12 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
FR2727867B1 (fr) 1994-12-13 1997-01-31 Rhone Poulenc Rorer Sa Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux
US6166197A (en) 1995-03-06 2000-12-26 Isis Pharmaceuticals, Inc. Oligomeric compounds having pyrimidine nucleotide (S) with 2'and 5 substitutions
AU725262B2 (en) 1996-02-14 2000-10-12 Isis Pharmaceuticals, Inc. Sugar-modified gapped oligonucleotides
GB9605808D0 (en) 1996-03-20 1996-05-22 Isis Innovation CFTR gene regulator
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
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
ATE312935T1 (de) 1997-06-03 2005-12-15 Regulatorische sequenzen für die in-vivo- expression einer heterologen dns-sequenz in endothelzellen und ihre verwendungen.
US6963589B1 (en) 1997-07-03 2005-11-08 Canon Kabushiki Kaisha Information processing apparatus for and method of transmitting and/or receiving broadcast signal
US6391452B1 (en) 1997-07-18 2002-05-21 Bayer Corporation Compositions for nasal drug delivery, methods of making same, and methods of removing residual solvent from pharmaceutical preparations
GB9716231D0 (en) 1997-07-31 1997-10-08 Amersham Int Ltd Base analogues
US7572582B2 (en) 1997-09-12 2009-08-11 Exiqon A/S Oligonucleotide analogues
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
EP2341058A3 (fr) 1997-09-12 2011-11-23 Exiqon A/S Analogues d'oligonucléotides
US7045610B2 (en) 1998-04-03 2006-05-16 Epoch Biosciences, Inc. Modified oligonucleotides for mismatch discrimination
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US7071324B2 (en) 1998-10-13 2006-07-04 Brown University Research Foundation Systems and methods for sequencing by hybridization
US20030224514A1 (en) 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of PPAR-delta expression
US6436657B1 (en) 1998-12-18 2002-08-20 E. I. Du Pont De Nemours And Company Polynucleotides encoding aminomethyltransferases
CN1273478C (zh) 1999-02-12 2006-09-06 三共株式会社 新型核苷及低聚核苷酸类似物
AU777049B2 (en) 1999-03-18 2004-09-30 Qiagen Gmbh Xylo-LNA analogues
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US6734291B2 (en) 1999-03-24 2004-05-11 Exiqon A/S Synthesis of [2.2.1]bicyclo nucleosides
EP1163250B1 (fr) 1999-03-24 2006-07-12 Exiqon A/S Synthese améliorée de ¬2.2.1|bicyclo-nucléosides
US6383752B1 (en) 1999-03-31 2002-05-07 Hybridon, Inc. Pseudo-cyclic oligonucleobases
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6083482A (en) 1999-05-11 2000-07-04 Icn Pharmaceuticals, Inc. Conformationally locked nucleosides and oligonucleotides
US6531591B1 (en) 1999-07-07 2003-03-11 Exiqon A/S Synthesis of stable quinone and photoreactive ketone phosphoramidite reagents for solid phase synthesis of photoreactive-oligomer conjugates
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
US6677445B1 (en) 1999-08-27 2004-01-13 Chiron Corporation Chimeric antisense oligonucleotides and cell transfecting formulations thereof
US6187586B1 (en) 1999-12-29 2001-02-13 Isis Pharmaceuticals, Inc. Antisense modulation of AKT-3 expression
US6784291B2 (en) 2000-05-04 2004-08-31 Avi Biopharma, Inc. Splice-region antisense composition and method
US6809194B1 (en) 2000-05-10 2004-10-26 Chiron Corporation Akt3 inhibitors
CA2697031C (fr) 2000-07-13 2017-10-31 The Johns Hopkins University School Of Medicine Detection et traitement de polykystose renale
WO2002018388A1 (fr) 2000-08-29 2002-03-07 Takeshi Imanishi Analogues de nucleosides et derives d'oligonucleotides renfermant ces analogues
PL366039A1 (en) 2000-09-02 2005-01-24 Grunenthal Gmbh Antisense oligonucleotides against vr1
JP4413493B2 (ja) 2000-10-04 2010-02-10 サンタリス ファーマ アー/エス プリンlna類似体の改善された合成方法
JP2002345489A (ja) 2000-11-03 2002-12-03 Astrazeneca Ab 化学物質
ATE287413T1 (de) 2001-07-12 2005-02-15 Santaris Pharma As Verfahren zur herstellung des lna phosphoramidite
AU2002336367A1 (en) 2001-08-16 2003-03-03 The Regents Of The University Of Michigan Adamts13 genes and proteins and variants, and uses thereof
EP1446412B1 (fr) 2001-09-04 2012-03-07 Exiqon A/S Compositions d'acides nucleiques verrouilles et utilisations
US6936589B2 (en) 2001-09-28 2005-08-30 Albert T. Naito Parenteral delivery systems
EP1442137A4 (fr) 2001-11-07 2005-08-31 Applera Corp Nucleotides universels pour analyse d'acides nucleiques
EP2354148B1 (fr) 2002-02-13 2013-09-04 Takeshi Imanishi Analogues de nucléoside et dérive d'oligonucléotides comportant un analogue de nucléotide
DE60315444T2 (de) 2002-05-08 2008-04-30 Santaris Pharma A/S Synthese von locked nucleic acid-derivaten
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004001010A2 (fr) 2002-06-21 2003-12-31 Ptc Therapeutics, Inc. Methodes permettant d'identifier de petites molecules modulant une terminaison de traduction prematuree et une degradation d'arnm a mediation non sens
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
JP2005535318A (ja) 2002-08-12 2005-11-24 ユニヴェルシテ ドゥ シェルブルック プレメッセンジャーrnaのスプライス部位選択の再プログラミング方法
JP2005535332A (ja) 2002-08-12 2005-11-24 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン 結節性硬化症の診断法および治療法
AU2003291753B2 (en) 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1560840B1 (fr) 2002-11-05 2015-05-06 Isis Pharmaceuticals, Inc. Compositions comprenant des nucleosides modifies en 2' de substitution destinees a la modulation de gene
DK2284269T3 (en) 2002-11-18 2017-10-23 Roche Innovation Ct Copenhagen As Antisense design
US7790867B2 (en) * 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
EP2216407B1 (fr) 2003-03-07 2016-01-13 Alnylam Pharmaceuticals, Inc. Compositions thérapeutiques
EP2141234B1 (fr) 2003-03-21 2016-04-27 Roche Innovation Center Copenhagen A/S Analogues de petits Arn interférents (SIRNA)
WO2004083432A1 (fr) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation de la reconnaissance d'exons dans le pre-arnm par interference avec la structure d'arn secondaire
EP1620450A4 (fr) 2003-04-13 2011-01-19 Enzon Pharmaceuticals Inc Promedicaments oligonucleotidiques polymeres
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20070009899A1 (en) * 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
GB0326578D0 (en) 2003-11-14 2003-12-17 Univ Belfast Cancer diagnosis and therapy
US20050221354A1 (en) 2004-02-18 2005-10-06 Wyeth Nucleic acid arrays for monitoring expression profiles of drug target genes
CA2566519C (fr) 2004-05-14 2020-04-21 Rosetta Genomics Ltd. Micro-arn et utilisations connexes
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US20070087376A1 (en) 2004-08-30 2007-04-19 Potashkin Judith A Splice variants of pre-mRNA transcripts as biomarkers in idiopathic neurodegenerative diseases
US20060134670A1 (en) 2004-11-19 2006-06-22 Fabrice Piu Enabling tools to identify ligands for hormone nuclear receptors
US8211864B2 (en) 2005-01-26 2012-07-03 Medical College Of Georgia Research Institute Compositions and methods for the intracellular disruption of VEGF and VEGFR-2 by intraceptors
WO2007094755A2 (fr) 2005-02-04 2007-08-23 Massachusetts Institute Of Technology Compositions et procédés de modulation de la fonction cognitive
JP5202296B2 (ja) 2005-04-01 2013-06-05 ユニバーシティ オブ フロリダ リサーチ ファウンデーション,インコーポレイティド 肝臓傷害のバイオマーカー
WO2006119494A2 (fr) * 2005-05-04 2006-11-09 Exagen Diagnostics, Inc. Biomarqueurs de leucemie myelogene aigue
PL1902141T3 (pl) 2005-06-17 2012-12-31 Baxalta Inc Kompozycje o działaniu trombolitycznym zawierające ADAMTS13
PL1910395T3 (pl) 2005-06-23 2013-03-29 Cold Spring Harbor Laboratory Kompozycja i sposób modulacji splicingu SMN2
SI3611266T1 (sl) 2005-08-23 2023-02-28 The Trustees Of The University Of Pennsylvania Modificirani nukleozidi, ki vsebujejo RNA, in postopki za njihovo uporabo
WO2007028065A2 (fr) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Composes oligomeriques chimeriques pour la modulation de l'epissage
WO2007047913A2 (fr) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions et procédés pour la modulation de l'expression du gène lmna
WO2007048628A2 (fr) 2005-10-28 2007-05-03 Max Planck Gesellschaft Structures de molecules d'arn-guide actives et leur procede de selection
WO2007048629A2 (fr) 2005-10-28 2007-05-03 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modulation de l'efficacite du silençage d'un arn a l'aide de proteines argonautes
US20100022495A1 (en) 2005-11-01 2010-01-28 President And Fellows Of Harvard College Modulating endoplasmic reticulum stress in the treatment of tuberous sclerosis
US7785834B2 (en) * 2005-11-10 2010-08-31 Ercole Biotech, Inc. Soluble TNF receptors and their use in treatment of disease
EP1792981A1 (fr) 2005-12-02 2007-06-06 Humboldt-Universität zu Berlin Protéines pour la préparation de microginines, acides nucléique encodant un cluster de gènes microginines et procédés pour la production de microginines
PL2161038T3 (pl) 2006-01-26 2014-06-30 Ionis Pharmaceuticals Inc Kompozycje i ich zastosowanie ukierunkowane na huntingtynę
AU2007211082B2 (en) 2006-01-27 2012-09-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
EP2314594B1 (fr) 2006-01-27 2014-07-23 Isis Pharmaceuticals, Inc. Analogues d'acide nucléique bicyclique modifiés en position 6
US8007790B2 (en) 2006-04-03 2011-08-30 Stowers Institute For Medical Research Methods for treating polycystic kidney disease (PKD) or other cyst forming diseases
WO2007143315A2 (fr) 2006-05-05 2007-12-13 Isis Pharmaceutical, Inc. Composés et procédés de modulation de l'expression de pcsk9
WO2007134181A2 (fr) 2006-05-11 2007-11-22 Isis Pharmaceuticals, Inc. Analogues d'acides nucléiques bicycliques modifiés en 5'
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
EP1857548A1 (fr) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Procédé et moyen permettant d'induire un saut d'exon
ATE522607T1 (de) 2006-06-08 2011-09-15 Amino Up Chemical Co Ltd Sense-oligonukleotid mit fähigkeit zur kontrolle der expression von inos sowie dieses umfassende zusammensetzung
WO2008001778A1 (fr) 2006-06-27 2008-01-03 Takeda Pharmaceutical Company Limited Animal génétiquement modifié et son utilisation
CA3081362C (fr) 2006-07-24 2024-04-09 Athena Diagnostics, Inc. Procede de detection de l'apparition de la maladie polykystique renale autosomale dominante (mprad) en fonction de la presence d'une modification de sequence nucleotidique dans la polykystose renale type dominant (pkd) 1
DK2410053T4 (da) 2006-10-18 2020-08-31 Ionis Pharmaceuticals Inc Antisense-forbindelser
US20090264353A1 (en) * 2007-10-19 2009-10-22 Santaris Pharma A/S Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
DK2099461T3 (da) 2006-11-13 2012-07-02 Santaris Pharma As LNA Nukleoside Phosphoramidates
US8293684B2 (en) 2006-11-29 2012-10-23 Exiqon Locked nucleic acid reagents for labelling nucleic acids
US8048998B2 (en) 2007-01-19 2011-11-01 Exiqon A/S Mediated cellular delivery of LNA oligonucleotides
EP2641971A1 (fr) 2007-01-29 2013-09-25 Isis Pharmaceuticals, Inc. Composés et procédés pour moduler lýexpression d'une protéine
EP2130835B1 (fr) 2007-03-09 2012-05-23 Riken Composé ayant une structure dérivée de mononucléoside ou de mononucléotide, d'acide nucléique, une substance de marquage et un procédé et un kit de détection d'acide nucléique
AR065648A1 (es) 2007-03-09 2009-06-24 Pioneer Hi Bred Int Identificacion de transportadores de amonio (amts) para la regulacion del metabolismo de nitrogeno en plantas
US8461124B2 (en) 2007-03-15 2013-06-11 Jyoti Chattopadhyaya Five- and six-membered conformationally locked 2′,4′-carbocyclic ribo-thymidines for the treatment of infections and cancer
EP2149605B1 (fr) 2007-03-22 2013-07-03 Santaris Pharma A/S Composés ARN antagonistes courts pour la modulation de l'ARNm cible
DK2170917T3 (da) 2007-05-30 2012-10-08 Isis Pharmaceuticals Inc N-Substituerede bicycliske nukleinsyreanaloge med aminomethylenbro
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
CA2692579C (fr) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. Analogues d'acides nucleiques bicycliques disubstitues en position 6
EP2188298B1 (fr) 2007-08-15 2013-09-18 Isis Pharmaceuticals, Inc. Analogues d'acide nucléique de tétrahydropyrane
CA2704049A1 (fr) 2007-10-26 2009-04-30 Academisch Ziekenhuis Leiden Moyens et procedes pour contrebalancer des troubles musculaires
US8637478B2 (en) 2007-11-13 2014-01-28 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
US8546556B2 (en) 2007-11-21 2013-10-01 Isis Pharmaceuticals, Inc Carbocyclic alpha-L-bicyclic nucleic acid analogs
CA3043911A1 (fr) 2007-12-04 2009-07-02 Arbutus Biopharma Corporation Lipides de ciblage
US8846386B2 (en) 2007-12-18 2014-09-30 University Of Kentucky Research Foundation sVEGFR-2 and its role in lymphangiogenesis modulation
WO2009084472A1 (fr) 2007-12-28 2009-07-09 Public University Corporation Yokohama City University Procédé de dépistage d'une épilepsie rebelle contractée pendant la période néo-natale et la petite enfance
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
US8530640B2 (en) 2008-02-07 2013-09-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
WO2009151691A2 (fr) 2008-03-13 2009-12-17 Celera Corporation Polymorphismes génétiques associés à une thrombose veineuse, leurs procédés de détection et leurs utilisations
US9290534B2 (en) 2008-04-04 2016-03-22 Ionis Pharmaceuticals, Inc. Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside
WO2009124295A2 (fr) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Composés oligomères comprenant des nucléosides bicycliques et ayant une toxicité réduite
EP2119783A1 (fr) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Procédé pour l'omission efficace de l'exon (44) dans la dystrophie musculaire de Duchenne et moyens connexes
JP5559159B2 (ja) 2008-06-11 2014-07-23 バイオニュークレオン ソチエタ・レスポンサビリタ・リミタータ 修飾オリゴヌクレオチドを使用するhrp−3の阻害
EP2151248A1 (fr) 2008-07-30 2010-02-10 Johann Bauer Molécules trans-splicing pre-mARN améliorées et leurs utilisations
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
US8501805B2 (en) 2008-09-24 2013-08-06 Isis Pharmaceuticals, Inc. Substituted alpha-L-bicyclic nucleosides
JP2012507989A (ja) 2008-11-07 2012-04-05 センター ホスピタライヤー ユニヴェルシテール サント−ジュスティーヌ Syngap1機能不全ならびに精神遅滞の診断および治療用途でのその使用
JP5971948B2 (ja) 2008-12-04 2016-08-17 クルナ・インコーポレーテッド Vegfに対する天然アンチセンス転写物の抑制による血管内皮増殖因子(vegf)関連疾患の治療
WO2010080509A1 (fr) 2008-12-19 2010-07-15 Philadelphia Health And Education Corporation Compositions et méthodes de diminution d'une infection virale et de l'inflammation associée à l'infection virale
US20100166784A1 (en) * 2008-12-30 2010-07-01 The Washington University Method and compositions for modulating th17 cell development
EP2381965B1 (fr) 2009-01-14 2020-05-06 Drexel University Modulation de pre-arnm au moyen d'oligonucleotides de modulation d'epissage servant d'agents therapeutiques dans le traitement d'une maladie
CA2761248C (fr) 2009-05-08 2023-03-14 Joseph Collard Traitement de maladies liees a la famille de la dystrophine par inhibition du produit antisens naturel de transcription vers la famille de la dmd
MX361732B (es) 2009-06-17 2018-12-14 Cold Spring Harbor Laboratory Composiciones y metodos para la modulacion de division de smn2 en un sujeto.
EP2275545A1 (fr) 2009-07-16 2011-01-19 Julius-Maximilians-Universität Würzburg Utilisation de micro ARN-24 et/ou ses cibles pour le traitement et la prévention de l'ischémie et induction de l'angiogénèse
WO2011017521A2 (fr) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Analogues d'acides nucléiques cyclohexoses bicycliques
US20120252877A1 (en) 2009-08-14 2012-10-04 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
CA3047466A1 (fr) 2009-09-08 2011-03-17 Laboratory Corporation Of America Holdings Compositions et procedes pour diagnostiquer des troubles du spectre autistique
WO2011032034A2 (fr) 2009-09-10 2011-03-17 University Of Idaho Nucléotides et oligonucléotides à conformation restreinte à fonctionnalité nucléobase pour cibler des acides nucléiques
AU2010312751B2 (en) 2009-10-29 2014-07-24 Osaka University Bridged artificial nucleoside and nucleotide
KR102581868B1 (ko) 2009-11-12 2023-10-04 더 유니버시티 오브 웨스턴 오스트레일리아 안티센스 분자 및 이를 이용한 질환 치료방법
WO2011066312A1 (fr) 2009-11-25 2011-06-03 Elitech Holding B.V. Antagonistes d'arnmi d'oligonucléotides de molécules se liant au sillon mineur (mgb)
WO2011085102A1 (fr) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Nucléosides bicycliques à base modifiée et composés oligomères préparés à partir de ceux-ci
JP6006120B2 (ja) 2010-02-08 2016-10-12 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. 対立遺伝子多様体の選択的低減
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011119842A1 (fr) 2010-03-25 2011-09-29 The J. David Gladstone Institutes Compositions et procédés pour traiter des troubles neurologiques
US9006415B2 (en) 2010-04-06 2015-04-14 Massachusetts Institute Of Technology Targeted delivery of nucleic acids
ES2638309T3 (es) 2010-04-19 2017-10-19 Nlife Therapeutics S.L. Composiciones y métodos para la distribución selectiva de moléculas de oligonucleótidos a tipos específicos de neuronas
US20110269735A1 (en) 2010-04-19 2011-11-03 Celera Corporation Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
US9156873B2 (en) 2010-04-28 2015-10-13 Isis Pharmaceuticals, Inc. Modified 5′ diphosphate nucleosides and oligomeric compounds prepared therefrom
CA2799944A1 (fr) 2010-05-20 2011-11-24 University Of Rochester Procedes et compositions lies a la modulation de l'autophagie
WO2011156278A1 (fr) 2010-06-07 2011-12-15 Isis Pharmaceuticals, Inc. Nucléosides bicycliques et composés oligomères élaborés à partir de ces nucléosides
WO2011156202A1 (fr) 2010-06-08 2011-12-15 Isis Pharmaceuticals, Inc. 2'‑amino- et 2'‑thio-nucléosides bicycliques substitués et composés oligomères préparés à partir de ces derniers
WO2011159836A2 (fr) 2010-06-15 2011-12-22 Isis Pharmaceuticals, Inc. Composés et procédés pour moduler l'interaction entre des protéines et des acides nucléiques cibles
US9771579B2 (en) 2010-06-23 2017-09-26 Curna, Inc. Treatment of sodium channel, voltage-gated, alpha subunit (SCNA) related diseases by inhibition of natural antisense transcript to SCNA
CA2815212A1 (fr) 2010-10-22 2012-04-26 Curna, Inc. Traitement de maladies associees a l'alpha-l-iduronidase (idua) par inhibition du transcrit antisens endogene de idua
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
US20130309246A1 (en) 2011-02-02 2013-11-21 The Trustees Of Princeton University Jagged1 as a marker and therapeutic target for breast cancer bone metastasis
WO2012138487A2 (fr) 2011-04-07 2012-10-11 The Board Of Regents Of The University Of Texas System Modulation oligonucléotidique de l'épissage
US9644035B2 (en) 2011-04-08 2017-05-09 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
US9957558B2 (en) 2011-04-28 2018-05-01 Life Technologies Corporation Methods and compositions for multiplex PCR
US20140357558A1 (en) 2011-06-24 2014-12-04 Cold Spring Harbor Laboratory Compositions and methods for treatment of spinal muscular atrophy
ES2626488T3 (es) 2011-07-19 2017-07-25 Wave Life Sciences Pte. Ltd. Procedimientos para la síntesis de ácidos nucleicos funcionalizados
US8592156B2 (en) 2011-08-08 2013-11-26 Roche Molecular Systems, Inc. Predicting response to anti-CD20 therapy in DLBCL patients
EP3640332A1 (fr) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Complexes oligomères-conjugués et leur utilisation
KR101991980B1 (ko) 2011-09-06 2019-06-21 큐알엔에이, 인크. 소형 분자로 전압-개폐된 나트륨 채널 (SCNxA)의 알파 아단위에 관련된 질환의 치료
US9453261B2 (en) 2011-09-20 2016-09-27 The George Washington University Alternative splicing variants of genes associated with prostate cancer risk and survival
US20140343127A1 (en) 2011-11-11 2014-11-20 Santaris Pharma A/S a corporation Compounds for the modulation of smn2 splicing
US9534222B2 (en) 2011-11-15 2017-01-03 University Of Utah Research Foundation Morpholinos, morpholino upregulating, and associated methods
EP2966180B1 (fr) 2011-11-29 2017-08-16 Life Technologies Corporation Procédés et compositions pour pcr multiplexe
US20150025231A1 (en) 2012-01-11 2015-01-22 Cold Spring Harbor Laboratory Compositions and methods for modulation of ikbkap splicing
EP2812342B1 (fr) 2012-02-08 2017-11-15 Ionis Pharmaceuticals, Inc. Modulation d'arn par ciblage de répétition
WO2013119916A2 (fr) 2012-02-10 2013-08-15 Ptc Therapeutics, Inc. Composés destinés au traitement de l'amyotrophie spinale
US8846885B2 (en) 2012-02-17 2014-09-30 Ajinomoto Co., Inc. Oligonucleotide with protected base
WO2013154798A1 (fr) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Analogues tricycliques d'acide nucléique
US9914922B2 (en) 2012-04-20 2018-03-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US20140378526A1 (en) 2012-05-11 2014-12-25 City Of Hope Design of nucleic acid binding molecules with non-watson crick and non-canonical pairing based on artificial mutation consensus sequences to counter escape mutations
US9109001B2 (en) 2012-05-22 2015-08-18 Idenix Pharmaceuticals, Inc. 3′,5′-cyclic phosphoramidate prodrugs for HCV infection
US9296778B2 (en) 2012-05-22 2016-03-29 Idenix Pharmaceuticals, Inc. 3′,5′-cyclic phosphate prodrugs for HCV infection
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
SG11201500232UA (en) 2012-07-13 2015-04-29 Wave Life Sciences Pte Ltd Chiral control
US8729263B2 (en) 2012-08-13 2014-05-20 Novartis Ag 1,4-disubstituted pyridazine analogs there of and methods for treating SMN-deficiency-related conditions
IN2015DN01765A (fr) 2012-08-20 2015-05-29 Univ California
AU2013319788B2 (en) 2012-09-24 2019-04-11 Yissum Reasearch Development Company of the Hebrew University Of Jerusalem Ltd. Restoration of the CFTR function by splicing modulation
WO2014049536A2 (fr) 2012-09-25 2014-04-03 Universidade De Lisboa Ciblage de médicaments pour fibrose kystique et autres pathologies
WO2014052638A1 (fr) 2012-09-27 2014-04-03 Idenix Pharmaceuticals, Inc. Esters et malonates de promédicaments à base de s-acyl-2-thioéthyle (sate)
UA116639C2 (uk) 2012-10-09 2018-04-25 Рег'Юлес Терап'Ютікс Інк. Способи лікування синдрому альпорта
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2014066915A2 (fr) 2012-10-26 2014-05-01 Smith Larry J Procédés et compositions pour produire une activité ss-arni ayant une puissance accrue
WO2014078749A1 (fr) 2012-11-15 2014-05-22 The Regents Of The University Of California Oligonucléotides modulant l'épissage qui inhibent le cancer
EP2935304A1 (fr) 2012-12-19 2015-10-28 IDENIX Pharmaceuticals, Inc. 4'-fluoro-nucléosides pour le traitement du vhc
CN103667438B (zh) 2013-01-07 2015-04-01 赵晨 一种筛查HRDs致病突变的方法及涉及的基因芯片杂交探针设计方法
US20150361497A1 (en) 2013-01-18 2015-12-17 Anna Mary ROSE Therapeutics and diagnostics based on minisatellite repeat element 1 (msr1)
EP3778618A1 (fr) 2013-02-04 2021-02-17 Ionis Pharmaceuticals, Inc. Composés antisens sélectifs et leurs utilisations
WO2014126229A1 (fr) 2013-02-18 2014-08-21 塩野義製薬株式会社 Nucléoside et nucléotide possédant une structure hétérocyclique azotée
US9309275B2 (en) 2013-03-04 2016-04-12 Idenix Pharmaceuticals Llc 3′-deoxy nucleosides for the treatment of HCV
US9339541B2 (en) 2013-03-04 2016-05-17 Merck Sharp & Dohme Corp. Thiophosphate nucleosides for the treatment of HCV
US9187515B2 (en) 2013-04-01 2015-11-17 Idenix Pharmaceuticals Llc 2′,4′-fluoro nucleosides for the treatment of HCV
WO2014172698A1 (fr) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions et procédés de modulation d'acides nucléiques par une dégradation médiée des éléments non-sens
CN111593051A (zh) 2013-05-01 2020-08-28 Ionis制药公司 组合物和方法
WO2014198890A1 (fr) 2013-06-13 2014-12-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Procédés et compositions pharmaceutiques pour le traitement de la protoporphyrie érythropoïétique
WO2014201413A1 (fr) 2013-06-14 2014-12-18 Isis Pharmaceuticals, Inc. Composés et procédés permettant de moduler l'arn non codant
MX363456B (es) 2013-06-25 2019-03-25 Hoffmann La Roche Compuestos para tratar atrofia muscular espinal.
MX2016002044A (es) 2013-08-16 2016-08-17 Rana Therapeutics Inc Composiciones y metodos para modular el acido ribonucleico.
EP3033423A4 (fr) 2013-08-16 2017-04-26 Rana Therapeutics Inc. Régulateurs épigénétiques de la frataxine
JP2016531570A (ja) 2013-08-16 2016-10-13 ラナ セラピューティクス インコーポレイテッド ユークロマチン領域を標的とするオリゴヌクレオチド
EP3033425A4 (fr) 2013-08-16 2017-07-26 Rana Therapeutics, Inc. Compositions et procédés de modulation de l'expression de la frataxine
CN105392790B (zh) 2013-08-19 2019-04-19 豪夫迈·罗氏有限公司 4H-吡啶并[1,2-a]嘧啶-4-酮化合物制备用于预防或治疗癌症的药物的用途
US10647983B2 (en) 2013-09-04 2020-05-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mRNA decay
WO2015035231A1 (fr) 2013-09-05 2015-03-12 Sarepta Therapeutics, Inc. Inclusion de l'exon 2 induite par antisens dans une alpha-glucosidase acide
DK3044315T3 (da) 2013-09-11 2019-05-20 Synthena Ag Nukleinsyrer og fremgangsmåder til behandling af pompes sygdom
CN106661580B (zh) 2014-06-10 2022-02-15 鹿特丹伊拉斯谟大学医疗中心 用于治疗庞帕病的反义寡核苷酸
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
GB201411468D0 (en) 2014-06-27 2014-08-13 Univ Edinburgh Novel treatment for endometriosis
AU2015304945B2 (en) 2014-08-20 2022-04-07 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof
WO2016027168A2 (fr) 2014-08-20 2016-02-25 Lifesplice Pharma Llc Oligonucléotides modulateurs d'épissage et leurs procédés d'utilisation
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
WO2016061509A1 (fr) 2014-10-17 2016-04-21 The Broad Institute, Inc. Compositions et procédés de traitement de la dystrophie musculaire
US10822369B2 (en) 2014-11-14 2020-11-03 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of proteins
GB201421379D0 (en) 2014-12-02 2015-01-14 Isis Innovation Ltd And Medical Res Council Molecule
AU2016209295B2 (en) 2015-01-21 2021-08-12 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
EP3256126B1 (fr) 2015-02-09 2024-03-27 F. Hoffmann-La Roche AG Composés pour le traitement du cancer
US9840709B2 (en) 2015-02-20 2017-12-12 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
BR112017018383B1 (pt) 2015-02-27 2023-04-25 Murdoch University Compostos anti-sentido que induzem a inclusão de éxon2, composições farmacêuticas que compreendem ditos compostos e usos dos mesmos para tratar doença de armazenamento de glicogênio tipo ii
RU2017137410A (ru) 2015-04-03 2019-05-06 Ионис Фармасьютикалз, Инк. Соединения и способы модулирования экспрессии tmprss6
EP4249472A3 (fr) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Procédés de modulation de l'épissage de l'arn
EP3352872A4 (fr) 2015-09-24 2019-07-10 The Trustees of the University of Pennsylvania Dérivés de triptycène pour stabiliser des jonctions d'acide nucléique
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
GB2591194B (en) 2015-10-09 2021-10-13 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JP7036723B2 (ja) 2015-12-14 2022-03-15 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
WO2017106210A1 (fr) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Oligomères antisens pour le traitement du syndrome d'alagille
JP7054675B2 (ja) 2015-12-14 2022-04-14 コールド スプリング ハーバー ラボラトリー 網膜色素変性症18と網膜色素変性症13の処置のための組成物と方法
JP2019500345A (ja) 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 肝臓病の処置のための組成物および方法
JP7049249B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 中枢神経系疾患の処置のための組成物および方法
CN109312343B (zh) 2015-12-14 2022-09-27 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
WO2017106292A1 (fr) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions et méthodes de traitement de maladies rénales
EP3389782A4 (fr) 2015-12-14 2019-07-31 Cold Spring Harbor Laboratory Oligomères antisens pour le traitement de la polykystose rénale
CA3005131A1 (fr) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Oligomeres antisens pour le traitement de la sclerose tubereuse de bourneville
SG11201909203WA (en) 2017-04-03 2019-11-28 Encoded Therapeutics Inc Tissue selective transgene expression
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
BR112020007881A2 (pt) 2017-10-23 2020-12-22 Stoke Therapeutics, Inc. Oligômeros anti-senso para o tratamento de condições e doenças baseadas no decaimento de rna mediado por nonsense

Also Published As

Publication number Publication date
AU2021290279A1 (en) 2022-01-27
IL249392A0 (en) 2017-02-28
US20230183693A1 (en) 2023-06-15
KR102638276B1 (ko) 2024-02-16
JP2023011885A (ja) 2023-01-24
IL295051A (en) 2022-09-01
GB201410693D0 (en) 2014-07-30
US9714422B2 (en) 2017-07-25
US10538764B2 (en) 2020-01-21
KR20170029512A (ko) 2017-03-15
US20240309377A1 (en) 2024-09-19
WO2015193651A1 (fr) 2015-12-23
EP4015648A1 (fr) 2022-06-22
CN107073137A (zh) 2017-08-18
EP3155124A1 (fr) 2017-04-19
CA2951208A1 (fr) 2015-12-23
JP7532136B2 (ja) 2024-08-13
JP7043169B2 (ja) 2022-03-29
US20160244762A1 (en) 2016-08-25
WO2015193651A4 (fr) 2016-03-10
AU2015275870B2 (en) 2021-10-14
IL295051B2 (en) 2024-08-01
US20160017322A1 (en) 2016-01-21
JP2017519766A (ja) 2017-07-20
IL249392B (en) 2022-09-01
US9745577B2 (en) 2017-08-29
US20200231969A1 (en) 2020-07-23
EP3155124B1 (fr) 2021-11-24
ES2907033T3 (es) 2022-04-21
IL295051B1 (en) 2024-04-01
SG11201610480XA (en) 2017-01-27
AU2015275870A1 (en) 2017-02-02
US20180002694A1 (en) 2018-01-04
US11390869B2 (en) 2022-07-19
JP2020189849A (ja) 2020-11-26
US11891605B2 (en) 2024-02-06

Similar Documents

Publication Publication Date Title
IL249392A0 (en) reduced intron retention
GB201520799D0 (en) None
AU361515S (en) Dishrack
AU362944S (en) Lapboard
GB201413050D0 (en) None
GB201516266D0 (en) None
GB201516146D0 (en) None
GB201505423D0 (en) Not published
HK1225829A1 (zh) 程序
DK3191586T3 (en) Cellobiosephosphorylase
DK3178931T3 (en) Anti-orai1-antistof
LT3237349T (lt) Patobulintas biologinis rišiklis
DK3002465T3 (en) Hydrauliksystem
HK1219846A1 (zh) 內衣
DK3194944T3 (en) Tomografiapparat
DK3145380T3 (en) Toiletventilationssystem
GB201409138D0 (en) None
GB201604697D0 (en) None
GB201604696D0 (en) None
AU5594P (en) Lanoregon Mandevilla sanderi
AU5593P (en) Lanmissouri Mandevilla sanderi
AU5585P (en) Lanutah Mandevilla sanderi
AU5598P (en) Lanidaho Mandevilla sanderi
AU5584P (en) Laniowa Mandevilla sanderi
AU5583P (en) Lanminnesota Mandevilla sanderi