IL259222A - Antisense oligomers for the treatment of autosomal dominant 5-mental retardation and Derva syndrome - Google Patents

Antisense oligomers for the treatment of autosomal dominant 5-mental retardation and Derva syndrome

Info

Publication number
IL259222A
IL259222A IL259222A IL25922218A IL259222A IL 259222 A IL259222 A IL 259222A IL 259222 A IL259222 A IL 259222A IL 25922218 A IL25922218 A IL 25922218A IL 259222 A IL259222 A IL 259222A
Authority
IL
Israel
Prior art keywords
treatment
mental retardation
autosomal dominant
antisense oligomers
dravet syndrome
Prior art date
Application number
IL259222A
Other languages
English (en)
Hebrew (he)
Original Assignee
Cold Spring Harbor Laboratory
Stoke Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Cold Spring Harbor Laboratory, Stoke Therapeutics Inc filed Critical Cold Spring Harbor Laboratory
Publication of IL259222A publication Critical patent/IL259222A/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Analytical Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Immunology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Pathology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
IL259222A 2015-12-14 2018-05-08 Antisense oligomers for the treatment of autosomal dominant 5-mental retardation and Derva syndrome IL259222A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267251P 2015-12-14 2015-12-14
PCT/US2016/066708 WO2017106377A1 (en) 2015-12-14 2016-12-14 Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome

Publications (1)

Publication Number Publication Date
IL259222A true IL259222A (en) 2018-07-31

Family

ID=59057535

Family Applications (1)

Application Number Title Priority Date Filing Date
IL259222A IL259222A (en) 2015-12-14 2018-05-08 Antisense oligomers for the treatment of autosomal dominant 5-mental retardation and Derva syndrome

Country Status (11)

Country Link
US (2) US11083745B2 (enExample)
EP (2) EP3933041B1 (enExample)
JP (2) JP7049248B2 (enExample)
KR (1) KR102604132B1 (enExample)
CN (2) CN109312343B (enExample)
AU (2) AU2016370653A1 (enExample)
CA (1) CA3005256A1 (enExample)
ES (1) ES2882500T3 (enExample)
IL (1) IL259222A (enExample)
SG (1) SG11201804443UA (enExample)
WO (1) WO2017106377A1 (enExample)

Families Citing this family (32)

* 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
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of 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
ES2882500T3 (es) * 2015-12-14 2021-12-02 Cold Spring Harbor Laboratory Oligómeros antisentido para el tratamiento del síndrome de Dravet
SI3673080T1 (sl) * 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
EP3700570B1 (en) * 2017-10-23 2025-01-08 Stoke Therapeutics, Inc. Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
JP7401432B2 (ja) * 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
WO2019213525A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
CA3110211A1 (en) * 2018-08-20 2020-02-27 Rogcon, Inc. Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies
KR102814622B1 (ko) * 2018-09-26 2025-05-29 케이스 웨스턴 리저브 유니버시티 단백질 발현 증가용 및/또는 반가불충분성 장애 치료용 방법 및 조성물
US20220049250A1 (en) * 2018-12-04 2022-02-17 Stichting Katholieke Universiteit Antisense Oligonucleotides Rescue Aberrant Splicing of ABCA4
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns
KR20210134003A (ko) * 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
JP2022532998A (ja) * 2019-04-26 2022-07-21 ストーク セラピューティクス，インク． 選択的イントロンのスプライシングを調節するための方法及び組成物
WO2020243651A1 (en) 2019-05-29 2020-12-03 Encoded Therapeutics, Inc. Compositions and methods for selective gene regulation
CA3147970A1 (en) * 2019-08-19 2021-02-25 Isabel AZNAREZ Compositions and methods for modulating splicing and protein expression
US11618900B2 (en) * 2019-11-01 2023-04-04 The Johns Hopkins University Modulating SYNGAP
EP4069844A4 (en) * 2019-12-04 2024-03-20 Pai, Athma A. ANTI-SLC6A1 OLIGONUCLEOTIDES AND RELATED METHODS
AU2020395835A1 (en) * 2019-12-06 2022-06-23 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
CN115516094A (zh) * 2020-02-28 2022-12-23 芝加哥大学 包含反式作用翻译活化剂的方法和组合物
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
CN115916977A (zh) * 2020-05-11 2023-04-04 弗洛里神经科学与心理健康研究所 用于治疗与syngap1中功能失去突变相关的障碍的组合物和方法
CN114480651B (zh) * 2022-02-14 2023-10-20 天津市泌尿外科研究所 Pcat1的反义寡核苷酸及其在制备抑制前列腺癌核酸药物中的应用
JP2025507683A (ja) * 2022-02-24 2025-03-21 キュー－ステート バイオサイエンシーズ， インコーポレイテッド Ｓｙｎｇａｐハプロ不全のための治療剤
JP2025540101A (ja) * 2022-12-01 2025-12-11 キャンプ４ セラピューティクス コーポレイション 調節ｒｎａを標的とするアンチセンスオリゴヌクレオチドを用いたｓｙｎｇａｐ１遺伝子転写の調節
EP4642913A2 (en) * 2022-12-28 2025-11-05 The University of Chicago Splice-switching oligonucleotides for treating syngap1-associated disorders
CN116334086A (zh) * 2023-03-29 2023-06-27 广州医科大学附属第二医院 一种用于修复或抑制SCN1A基因异常剪接的U1-snRNA及其用途
EP4705455A1 (en) * 2023-05-03 2026-03-11 Stoke Therapeutics, Inc. Compounds and methods for treating human subjects
AU2024367273A1 (en) * 2023-10-23 2026-05-14 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating genetic epilepsies
WO2025199503A1 (en) * 2024-03-22 2025-09-25 Stoke Therapeutics, Inc. Methods for treating conditions and diseases
WO2026065401A1 (zh) * 2024-09-30 2026-04-02 时夕(广州)生物科技有限公司 一种用于调控SCN1A基因mRNA和蛋白表达的方法和化合物

Family Cites Families (272)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5914396A (en) 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US7101993B1 (en) 1990-01-11 2006-09-05 Isis Pharmaceuticals, Inc. Oligonucleotides containing 2′-O-modified purines
US5151510A (en) 1990-04-20 1992-09-29 Applied Biosystems, Inc. Method of synethesizing sulfurized oligonucleotide analogs
DE69133511T2 (de) 1990-08-13 2006-09-28 Isis Pharmaceuticals, Inc., Carlsbad Zuckermodifizierte oligonukleotide zum erkennen und steuern der genexpression
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
JP3015464B2 (ja) 1992-07-23 2000-03-06 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 新規２’−０−アルキルヌクレオシドおよびホスホロアミダイトの製造法およびその使用
JPH08501686A (ja) 1992-09-25 1996-02-27 ローン−プーラン・ロレ・ソシエテ・アノニム 中枢神経系、特に脳における細胞への外来遺伝子の転移のためのアデノウィルスベクター
EP1897942A1 (en) 1993-05-11 2008-03-12 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
WO1995018225A1 (en) 1993-12-24 1995-07-06 Medical Research Council Polycystic kidney disease 1 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
KR19990082476A (ko) 1996-02-14 1999-11-25 하우스 한스 루돌프, 긴터 베아트리체 당-변형되어 갭이 형성된 올리고뉴클레오티드
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
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
USRE44779E1 (en) 1997-03-07 2014-02-25 Santaris Pharma A/S Bicyclonucleoside and oligonucleotide analogues
AU8435698A (en) 1997-06-03 1998-12-21 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Regulatory sequences capable of conferring expression of a heterologous dna sequence in endothelial cells in vivo and uses thereof
US6963589B1 (en) 1997-07-03 2005-11-08 Canon Kabushiki Kaisha Information processing apparatus for and method of transmitting and/or receiving broadcast signal
GB9716231D0 (en) 1997-07-31 1997-10-08 Amersham Int Ltd Base analogues
CA2303299C (en) 1997-09-12 2016-02-23 Exiqon A/S Oligonucleotide 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
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
PT1152009E (pt) 1999-02-12 2005-03-31 Sankyo Co Novos analogos de nucleosidos e oligonucleotidos
EP1161439B1 (en) 1999-03-18 2010-04-21 Exiqon A/S 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
DK1163250T3 (da) 1999-03-24 2006-11-13 Exiqon As Forbedret syntese af [2.2.1]bicyclonukleosider
US6383752B1 (en) 1999-03-31 2002-05-07 Hybridon, Inc. Pseudo-cyclic oligonucleobases
NZ514348A (en) 1999-05-04 2004-05-28 Exiqon As 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
WO2001083740A2 (en) 2000-05-04 2001-11-08 Avi Biopharma, Inc. Splice-region antisense composition and method
US6809194B1 (en) 2000-05-10 2004-10-26 Chiron Corporation Akt3 inhibitors
US7553644B2 (en) 2000-07-13 2009-06-30 The Johns Hopkins University School Of Medicine Detection and treatment of polycystic kidney disease
US7053199B2 (en) 2000-08-29 2006-05-30 Takeshi Imanishi Nucleoside analogs and oligonucleotide derivatives containing these analogs
HUP0301805A3 (en) 2000-09-02 2005-12-28 Gruenenthal Gmbh Antisense oligonucleotides against vanilloid receptor 1
WO2002028875A2 (en) 2000-10-04 2002-04-11 Cureon A/S Improved synthesis of purine locked nucleic acid analogues
JP2002345489A (ja) 2000-11-03 2002-12-03 Astrazeneca Ab 化学物質
JP4402454B2 (ja) 2001-07-12 2010-01-20 サンタリス ファーマ アー／エス Ｌｎａホスホラミダイトの製造法
AU2002336367A1 (en) 2001-08-16 2003-03-03 The Regents Of The University Of Michigan Adamts13 genes and proteins and variants, and uses thereof
WO2003020739A2 (en) 2001-09-04 2003-03-13 Exiqon A/S Novel lna compositions and uses thereof
US6936589B2 (en) 2001-09-28 2005-08-30 Albert T. Naito Parenteral delivery systems
WO2003040395A2 (en) 2001-11-07 2003-05-15 Applera Corporation Universal nucleotides for nucleic acid analysis
EP1481983A4 (en) 2002-02-13 2008-04-02 Takeshi Imanishi NUCLEOSIDE ANALOGUE AND OLIGONUCLEOTIDE DERIVATIVES CONTAINING A NUCLEOTIDE ANALOGON THEREOF
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
JP4476802B2 (ja) 2002-05-08 2010-06-09 サンタリス ファーマ アー／エス ロックト核酸誘導体の製造
WO2004001010A2 (en) 2002-06-21 2003-12-31 Ptc Therapeutics, Inc. METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY
WO2004015106A1 (en) 2002-08-12 2004-02-19 UNIVERSITé DE SHERBROOKE Methods to reprogram splice site selection in pre-messenger rnas
EP1546369A4 (en) 2002-08-12 2007-01-17 Univ Michigan DIAGNOSIS AND TREATMENT OF DISEASES THROUGH DEFECTS IN THE TUBEROUS SKLEROSEPFAD
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004043979A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US9045518B2 (en) 2002-11-18 2015-06-02 Santaris Pharma A/S Amino-LNA, thio-LNA and alpha-L-oxy-LN
US7790867B2 (en) 2002-12-05 2010-09-07 Rosetta Genomics Inc. Vaccinia virus-related nucleic acids and microRNA
WO2004080406A2 (en) 2003-03-07 2004-09-23 Alnylam Pharmaceuticals Therapeutic compositions
US8653252B2 (en) 2003-03-21 2014-02-18 Santaris Pharma A/S Short interfering RNA (siRNA) analogues
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
MXPA05011022A (es) 2003-04-13 2006-04-27 Enzon Pharmaceuticals Inc Profarmacos oligonucleotidos polimericos.
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
WO2005111211A2 (en) 2004-05-14 2005-11-24 Rosetta Genomics Ltd. Micronas and uses thereof
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US20080299104A1 (en) * 2004-07-01 2008-12-04 California Institute Of Technology Methods for detecting agents involved in neuronal apoptosis and compositions thereof
US20070087376A1 (en) 2004-08-30 2007-04-19 Potashkin Judith A Splice variants of pre-mRNA transcripts as biomarkers in idiopathic neurodegenerative diseases
CA2588646A1 (en) 2004-11-19 2006-05-26 Acadia Pharmaceuticals Inc. Methods to identify ligands of hormone nuclear receptors
WO2006081311A2 (en) 2005-01-26 2006-08-03 Medical College Of Georgia Research Institute Compositions and methods for the intracellular disruption of vegf and vegfr-2 by intraceptors
US7718625B2 (en) 2005-01-27 2010-05-18 University Of South Florida Polynucleotides targeted against the extended 5′-UTR region of argininosuccinate synthase and uses thereof
US20100150839A1 (en) 2005-02-04 2010-06-17 Massachusetts Institute Of Technology Compositions and Methods for Modulating Cognitive Function
US7645584B2 (en) 2005-04-01 2010-01-12 University Of Florida Research Foundation, Inc. Biomarkers of liver injury
JP2008546376A (ja) * 2005-06-16 2008-12-25 バイオノミックス リミテッド Ｓｃｎ１ａ遺伝子における変異を検出することによっててんかんを診断および処置するための方法
AU2006257073B2 (en) 2005-06-17 2011-08-04 Children's Medical Center Corporation ADAMTS13-comprising compositions having thrombolytic activity
ES2397113T3 (es) 2005-06-23 2013-03-04 Isis Pharmaceuticals, Inc. Composiciones y procedimientos para modular el corte y empalme de SMN2
HUE043492T2 (hu) 2005-08-23 2019-08-28 Univ Pennsylvania Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
EP1945765A2 (en) 2005-10-28 2008-07-23 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Structures of active guide rna molecules and method of selection
WO2007048629A2 (en) 2005-10-28 2007-05-03 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Modulation of rna silencing efficiency by argonaute proteins
WO2007053747A2 (en) 2005-11-01 2007-05-10 President And Fellows Of Harvard College Modulating endoplasmic reticulum stress in the treatment of tuberous sclerosis
WO2007056113A2 (en) 2005-11-02 2007-05-18 Cylene Pharmaceuticals, Inc. Methods for targeting quadruplex sequences
US7785834B2 (en) 2005-11-10 2010-08-31 Ercole Biotech, Inc. Soluble TNF receptors and their use in treatment of disease
EP1792981A1 (en) 2005-12-02 2007-06-06 Humboldt-Universität zu Berlin Microginin producing proteins and nucleic acids encoding a microginin gene cluster as well as methods for creating microginins
SI2161038T1 (sl) 2006-01-26 2014-05-30 Isis Pharmaceuticals, Inc. Sestavki in njihove uporabe, usmerjeni proti huntingtinu
ES2351674T3 (es) 2006-01-27 2011-02-09 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos modificados en la posición 6.
US8129515B2 (en) 2006-01-27 2012-03-06 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
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
DK2021472T3 (da) 2006-05-05 2011-09-19 Isis Pharmaceuticals Inc Forbindelser og fremgangsmåder til modulering af genekspression
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US8927704B2 (en) 2006-06-08 2015-01-06 Amino Up Chemical Co., Ltd Sense oligonucleotide capable of controlling the expression of iNOS and composition comprising the same
JPWO2008001778A1 (ja) 2006-06-27 2009-11-26 武田薬品工業株式会社 遺伝子改変動物およびその用途
CA2993381C (en) 2006-07-24 2020-07-14 Athena Diagnostics, Inc. Method of detecting the occurrence of adpkd based on presence of nucleotide sequence alteration in pkd1
CA2667055C (en) 2006-10-18 2017-05-09 Isis Pharmaceuticals, Inc. Antisense compounds
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
WO2007054100A2 (en) 2006-11-13 2007-05-18 Santaris Pharma A/S Lna nucleoside 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
US20100093836A1 (en) 2007-01-29 2010-04-15 Isis Pharmaceuticals, Inc Compounds and methods for modulating protein expression
US8383792B2 (en) 2007-03-09 2013-02-26 Riken Compound having structure derived from mononucleoside or mononucleotide, nucleic acid, labeling substance, and method and kit for detection of nucleic acid
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 (en) 2007-03-22 2013-07-03 Santaris Pharma A/S Short RNA antagonist compounds for the modulation of target mRNA
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
EP2176280B2 (en) 2007-07-05 2015-06-24 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
WO2009023855A2 (en) 2007-08-15 2009-02-19 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
EP2203173B1 (en) 2007-10-26 2015-12-23 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2219680A2 (en) 2007-11-13 2010-08-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
CN102006890A (zh) 2007-12-04 2011-04-06 阿尔尼拉姆医药品有限公司 靶向脂质
US8846386B2 (en) 2007-12-18 2014-09-30 University Of Kentucky Research Foundation sVEGFR-2 and its role in lymphangiogenesis modulation
WO2009084472A1 (ja) 2007-12-28 2009-07-09 Public University Corporation Yokohama City University 新生児期～乳児期発症の難治性てんかんの検出方法
WO2009100320A2 (en) 2008-02-07 2009-08-13 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
EP2276855B1 (en) 2008-03-13 2016-05-11 Celera Corporation Genetic polymorphisms associated wiith venous thrombosis, methods of detection and uses thereof
WO2009124238A1 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
US8846639B2 (en) 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
EP2304031A2 (en) 2008-06-11 2011-04-06 Bionucleon S.r.l. Inhibition of hrp-3 using modified oligonucleotides
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
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
CA2735129C (en) * 2008-11-07 2012-06-26 Centre Hospitalier Universitaire Sainte-Justine Syngap1 dysfunctions and uses thereof in diagnostic and therapeutic applications for mental retardation
MX2011005912A (es) 2008-12-04 2011-06-17 Opko Curna Llc Tratamiento de enfermedades relacionadas con factor de crecimiento endotelial vascular por inhibicion de transcrito antisentido natural para factor de crecimiento endotelial vascular.
US9006194B2 (en) 2008-12-19 2015-04-14 Drexel University Compositions and methods for diminishing viral infection and inflammation associated with viral infection
US20100166784A1 (en) 2008-12-30 2010-07-01 The Washington University Method and compositions for modulating th17 cell development
DK2381965T3 (da) 2009-01-14 2020-07-27 Univ Drexel Modulation af præ-mrna under anvendelse af splejsningsmodulerende oligonukleotider som terapeutiske midler til behandling af sygdom
WO2010129861A2 (en) 2009-05-08 2010-11-11 Curna, Inc. Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
CN115227709A (zh) 2009-06-17 2022-10-25 冷泉港实验室 用于在对象中调节smn2剪接的组合物和方法
EP2275545A1 (en) 2009-07-16 2011-01-19 Julius-Maximilians-Universität Würzburg Use of microRNA-24 and/or its targets for the treatment and prevention of ischemia and induction of angiogenesis
US9012421B2 (en) 2009-08-06 2015-04-21 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2011020118A1 (en) 2009-08-14 2011-02-17 Theramind Research, Llc Methods and compositions for treatment of tuberous sclerosis complex
CA3047466A1 (en) 2009-09-08 2011-03-17 Laboratory Corporation Of America Holdings Compositions and methods for diagnosing autism spectrum disorders
WO2011032034A2 (en) 2009-09-10 2011-03-17 University Of Idaho Nucleobase-functionalized conformationally restricted nucleotides and oligonucleotides for targeting nucleic acids
US8541562B2 (en) 2009-10-29 2013-09-24 Osaka University Bridged artificial nucleoside and nucleotide
BR122023023194A2 (pt) 2009-11-12 2023-12-26 The University Of Western Australia Oligonucleotídeo antissentido, composição e respectivo uso
EP2547769B1 (en) 2009-11-25 2016-04-06 ELITechGroup B.V. Minor groove binder (mgb)-oligonucleotide mirna antagonists
WO2011085102A1 (en) 2010-01-11 2011-07-14 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
EP3561060A1 (en) 2010-02-08 2019-10-30 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
JP2013529181A (ja) 2010-03-25 2013-07-18 ザ ジェイ． デヴィッド グラッドストーン インスティテューツ 神経障害を治療するための組成物および方法
WO2011127210A1 (en) 2010-04-06 2011-10-13 Massachusetts Institute Of Technology Targeted delivery of nucleic acids
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
LT2560687T (lt) 2010-04-19 2017-09-25 Nlife Therapeutics S.L. Kompozicijos ir būdai, skirti selektyviam oligonukleotido molekulių pristatymui specifiniams neuronų tipams
US9156873B2 (en) 2010-04-28 2015-10-13 Isis Pharmaceuticals, Inc. Modified 5′ diphosphate nucleosides and oligomeric compounds prepared therefrom
US8802642B2 (en) 2010-04-28 2014-08-12 Iowa State University Research Foundation, Inc. Spinal muscular atrophy treatment via targeting SMN2 catalytic core
US20130184223A1 (en) 2010-05-20 2013-07-18 University Of Rochester Methods and compositions related to modulating autophagy
WO2011156278A1 (en) 2010-06-07 2011-12-15 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011156202A1 (en) 2010-06-08 2011-12-15 Isis Pharmaceuticals, Inc. Substituted 2 '-amino and 2 '-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
EP2582397A4 (en) 2010-06-15 2014-10-29 Isis Pharmaceuticals Inc COMPOUNDS AND METHOD FOR MODULATING INTERACTION BETWEEN PROTEINS AND TARGET NUCLEIC ACIDS
KR101914309B1 (ko) 2010-06-23 2018-11-02 큐알엔에이, 인크. 전압 작동 나트륨 통로, 알파 소단위(scna)에 대한 자연 안티센스 전사체의 저해에 의한 전압 작동 나트륨 통로, 알파 소단위(scna) 관련된 질환의 치료
JP6049623B2 (ja) 2010-10-22 2016-12-21 カッパーアールエヌエー，インコーポレイテッド α‐Ｌ‐イズロニダーゼ（ＩＤＵＡ）への天然アンチセンス転写物の阻害によるＩＤＵＡ関連疾患の治療
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
WO2012106529A1 (en) 2011-02-02 2012-08-09 The Trustees Of Princeton University Jagged1 as a marker and therapeutic target for breast cancer bone metastasis
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9644035B2 (en) 2011-04-08 2017-05-09 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
WO2012149438A1 (en) 2011-04-28 2012-11-01 Life Technologies Corporation Methods and compositions for multiplex pcr
DK2718437T3 (en) 2011-06-10 2018-08-06 Inst Nat Sante Rech Med Methods for the Treatment of Liver's Congenital Amaurosis
WO2012178146A1 (en) 2011-06-24 2012-12-27 Isis Pharmaceuticals, Inc. Compositions and methods for treatment of spinal muscular atrophy
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
US8592156B2 (en) 2011-08-08 2013-11-26 Roche Molecular Systems, Inc. Predicting response to anti-CD20 therapy in DLBCL patients
EP3640332A1 (en) 2011-08-29 2020-04-22 Ionis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
EA033653B1 (ru) 2011-09-05 2019-11-13 Stichting Katholieke Univ Антисмысловые олигонуклеотиды для лечения врожденного амавроза лебера
US10583128B2 (en) 2011-09-06 2020-03-10 Curna, Inc. Treatment of diseases related to alpha subunits of sodium channels, voltage-gated (SCNxA) with small molecules
US9453261B2 (en) 2011-09-20 2016-09-27 The George Washington University Alternative splicing variants of genes associated with prostate cancer risk and survival
JP2014533944A (ja) 2011-11-11 2014-12-18 サンタリス ファーマ アー／エスＳａｎｔａｒｉｓ Ｐｈａｒｍａ Ａ／Ｓ Ｓｍｎ２スプライシングの調節のための化合物
WO2013074814A2 (en) 2011-11-15 2013-05-23 University Of Utah Research Fourdation Morpholinos, morpholino upregulating, and associated methods
EP2966180B1 (en) 2011-11-29 2017-08-16 Life Technologies Corporation Methods and compositions for multiplex pcr
WO2013106770A1 (en) 2012-01-11 2013-07-18 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of ikbkap splicing
EP3330278A1 (en) 2012-02-08 2018-06-06 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
ES2697174T3 (es) 2012-02-10 2019-01-22 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
US8846885B2 (en) 2012-02-17 2014-09-30 Ajinomoto Co., Inc. Oligonucleotide with protected base
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
EP2839006B1 (en) 2012-04-20 2018-01-03 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
US9296778B2 (en) 2012-05-22 2016-03-29 Idenix Pharmaceuticals, Inc. 3′,5′-cyclic phosphate prodrugs for HCV infection
EP2852604B1 (en) 2012-05-22 2017-04-12 Idenix Pharmaceuticals LLC 3',5'-cyclic phosphoramidate prodrugs for hcv infection
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
CN112007045B (zh) 2012-07-13 2026-01-06 波涛生命科学有限公司 手性控制
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
EP2885313A4 (en) 2012-08-20 2016-03-09 Univ California POLYNUCLEOTIDES WITH BIOREVERSIBLE GROUPS
EP2898072A1 (en) 2012-09-24 2015-07-29 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Restoration of the cftr function by splicing modulation
WO2014049536A2 (en) 2012-09-25 2014-04-03 Universidade De Lisboa Drug targets for cystic fibrosis and other conditions
WO2014052638A1 (en) 2012-09-27 2014-04-03 Idenix Pharmaceuticals, Inc. Esters and malonates of sate prodrugs
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
US20150291957A1 (en) 2012-10-26 2015-10-15 Larry J. Smith METHODS AND COMPOSITIONS TO PRODUCE ss-RNAi ACTIVITY WITH ENHANCED POTENCY
US9909128B2 (en) 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
EP2935304A1 (en) 2012-12-19 2015-10-28 IDENIX Pharmaceuticals, Inc. 4'-fluoro nucleosides for the treatment of hcv
CN104004826B (zh) 2013-01-07 2016-03-02 赵晨 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用
CA2897522A1 (en) 2013-01-18 2014-07-24 Anna Mary Rose Therapeutics and diagnostics based on minisatellite repeat element 1 (msr1)
US10260069B2 (en) 2013-02-04 2019-04-16 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
EP2957567B1 (en) 2013-02-18 2019-06-05 Shionogi & Co., Ltd. Nucleoside and nucleotide, having nitrogen-containing hetercycle structure
US9309275B2 (en) 2013-03-04 2016-04-12 Idenix Pharmaceuticals Llc 3′-deoxy nucleosides for the treatment of HCV
WO2014137930A1 (en) 2013-03-04 2014-09-12 Idenix Pharmaceuticals, Inc. Thiophosphate nucleosides for the treatment of hcv
EP2981542B1 (en) 2013-04-01 2021-09-15 Idenix Pharmaceuticals LLC 2',4'-fluoro nucleosides for the treatment of hcv
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
CA2921162A1 (en) 2013-05-01 2014-11-06 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
US9549909B2 (en) 2013-05-03 2017-01-24 The Katholieke Universiteit Leuven Method for the treatment of dravet syndrome
US9637744B2 (en) 2013-06-13 2017-05-02 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and pharmaceutical compositions for the treatment of erythropoietic protoporphyria
WO2014201413A1 (en) 2013-06-14 2014-12-18 Isis Pharmaceuticals, Inc. Compounds and methods for modulating non-coding rna
AR096684A1 (es) 2013-06-25 2016-01-27 Ptc Therapeutics Inc Compuestos para tratar atrofia muscular espinal
WO2015023939A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating expression of frataxin
US20150050738A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Compositions and methods for modulating rna
EP3033423A4 (en) 2013-08-16 2017-04-26 Rana Therapeutics Inc. Epigenetic regulators of frataxin
EP3033422A4 (en) 2013-08-16 2017-08-02 Rana Therapeutics Inc. Oligonucleotides targeting euchromatin regions of genes
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
ES2779302T3 (es) 2013-09-04 2020-08-14 Cold Spring Harbor Laboratory Reducción de la degradación de ARNm con mediación sin sentido
KR102275504B1 (ko) 2013-09-05 2021-07-09 사렙타 쎄러퓨틱스, 인코퍼레이티드 산성 알파-글루코시다제 내 안티센스-유도된 엑손2 포함
WO2015036451A1 (en) 2013-09-11 2015-03-19 Synthena Ag Nucleic acids and methods for the treatment of pompe disease
CN105940101A (zh) 2013-11-21 2016-09-14 纪念斯隆-凯特琳癌症中心 由人多能干细胞特化功能性颅基板衍生物
US10119168B2 (en) 2014-03-12 2018-11-06 The Brigham And Women's Hospital, Inc. Methods for the treatment of kidney fibrosis
US20170044540A1 (en) 2014-04-22 2017-02-16 Mina Therapeutics Limited Sarna compositions and methods of use
WO2015190922A1 (en) 2014-06-10 2015-12-17 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of pompe disease
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
WO2016027168A2 (en) 2014-08-20 2016-02-25 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof
GB2547586A (en) 2014-08-20 2017-08-23 Lifesplice Pharma Llc Splice modulating oligonucleotides and methods of use thereof(In the PCT request)
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3207048A4 (en) 2014-10-17 2018-05-30 The Broad Institute, Inc. Compositions and methods of treating muscular dystrophy
AU2015346042A1 (en) 2014-11-14 2017-04-27 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
CN114642735A (zh) 2015-01-21 2022-06-21 菲泽尔克斯公司 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统
WO2016128343A1 (en) 2015-02-09 2016-08-18 F. Hoffmann-La Roche Ag Compounds for the treatment of cancer
EP3259356B1 (en) 2015-02-20 2021-12-01 Rosalind Franklin University of Medicine and Science Antisense compounds targeting genes associated with cystic fibrosis
CA2977528A1 (en) 2015-02-27 2016-09-01 Sarepta Therapeutics, Inc. Antisense-induced exon2 inclusion in acid alpha-glucosidase
HK1249140A1 (zh) 2015-04-03 2018-10-26 Ionis Pharmaceuticals, Inc. 用於调节tmprss6表达的化合物和方法
US10668171B2 (en) 2015-05-30 2020-06-02 Ptc Therapeutics, Inc. Methods for modulating RNA splicing
CA3000660A1 (en) 2015-09-24 2017-03-30 The Trustees Of The University Of Pennsylvania Triptycene derivatives for nucleic acid junction stabilization
GB2591194B (en) 2015-10-09 2021-10-13 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
AU2016334804B2 (en) 2015-10-09 2022-03-31 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
HK1251965A1 (zh) 2015-10-30 2019-05-10 Ptc医疗公司 用於治疗癫痫的方法
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3390635A4 (en) 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
ES2882500T3 (es) * 2015-12-14 2021-12-02 Cold Spring Harbor Laboratory Oligómeros antisentido para el tratamiento del síndrome de Dravet
WO2017106370A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of eye diseases
JP2018538287A (ja) 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
EP4104867A3 (en) 2015-12-14 2023-03-01 Cold Spring Harbor Laboratory Compositions and methods for treatment of central nervous system diseases
JP2018538288A (ja) 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー アラジール症候群の処置のためのアンチセンスオリゴマー
CA3005249A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of kidney diseases
EP3389672A4 (en) 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHOD FOR THE TREATMENT OF LIVER DISEASES
CA3005254A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Compositions and methods for treatment of retinitis pigmentosa 18 and retinitis pigmentosa 13
EP3443001B1 (en) 2016-04-11 2025-04-30 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
EP3512870B1 (en) 2016-09-16 2022-08-03 Olipass Corporation Scn9a antisense oligonucleotides
EA201992358A1 (ru) 2017-04-03 2020-03-24 Инкоудид Терапьютикс, Инк. Тканеселективная экспрессия трансгена
CA3059775A1 (en) 2017-04-13 2018-10-18 Ovid Therapeutics Inc. Methods of treating developmental encephalopathies
JP2020519594A (ja) 2017-05-09 2020-07-02 ゾゲニクス インターナショナル リミテッド フェンフルラミンを用いるドゥーゼ症候群の治療方法
WO2018213491A1 (en) 2017-05-16 2018-11-22 Praxis Precision Medicines, Inc. Methods of treating epilepsy and neurodevelopmental disorders
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
EP3700570B1 (en) 2017-10-23 2025-01-08 Stoke Therapeutics, Inc. Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases
JP7401432B2 (ja) 2017-12-01 2023-12-19 エンコーデッド セラピューティクス， インコーポレイテッド 操作されたｄｎａ結合タンパク質
AU2019243155A1 (en) 2018-03-27 2020-10-15 Factor Bioscience Inc. Nucleic acid-based therapeutics
US12121563B2 (en) 2018-04-09 2024-10-22 Allen Institute Rescuing voltage-gated sodium channel function in inhibitory neurons
US12259378B2 (en) 2018-05-25 2025-03-25 Praxis Precision Medicines, Inc. Dynamic clamps and methods of use thereof
WO2019236750A2 (en) 2018-06-05 2019-12-12 Tufts Medical Center, Inc. Targeted treatment of autism spectrum disorder and other neurological or psychiatric disorders
AU2019291050A1 (en) 2018-06-22 2020-12-24 F. Hoffmann-La Roche Ag Oligonucleotides for modulating SCN9A expression
CA3110211A1 (en) 2018-08-20 2020-02-27 Rogcon, Inc. Antisense oligonucleotides targeting scn2a for the treatment of scn1a encephalopathies
US10905778B2 (en) 2018-09-26 2021-02-02 Case Western Reserve University Methods and compositions for treating a premature stop codon-mediated disorder
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머

Also Published As

Publication number Publication date
EP3390636A1 (en) 2018-10-24
AU2016370653A1 (en) 2018-06-21
SG11201804443UA (en) 2018-06-28
JP2019501892A (ja) 2019-01-24
US11083745B2 (en) 2021-08-10
CA3005256A1 (en) 2017-06-22
CN116059236A (zh) 2023-05-05
EP3390636B1 (en) 2021-05-19
JP2022062140A (ja) 2022-04-19
JP7049248B2 (ja) 2022-04-06
AU2023202216A1 (en) 2023-07-06
KR102604132B1 (ko) 2023-11-17
EP3933041A1 (en) 2022-01-05
ES2882500T3 (es) 2021-12-02
EP3390636A4 (en) 2019-08-14
WO2017106377A1 (en) 2017-06-22
KR20180093977A (ko) 2018-08-22
US20180369275A1 (en) 2018-12-27
CN109312343B (zh) 2022-09-27
CN109312343A (zh) 2019-02-05
EP3933041B1 (en) 2024-01-31
US20220088058A1 (en) 2022-03-24

Similar Documents

Publication Publication Date Title
IL273495A (en) Treatment of fragile X syndrome with cannabidiol
EP3390636A4 (en) ANTISENSE OLIGOMERS FOR THE TREATMENT OF AUTOSOMAL-DOMINANT MENTAL RETARDATION 5 AND DRAVET SYNDROME
GB2548873B (en) Use of Cannabidiol in the Treatment of SturgeWeber Syndrome
IL250016A0 (en) Antisense oligonucleotides for the treatment of type 2 syndrome
IL259441B1 (en) Materials and methods for the treatment of titin-based myopathies and other titinopathy
GB201621480D0 (en) Use of cannabinoids in the treatment of angelman syndrome
IL290993A (en) Methods of diagnosing and treating tourette syndrome
ZA201800499B (en) Methods of treating lennox¿gastaut syndrome using fenfluramine
EP3068289A4 (en) Prevention and treatment of bed exits, falls, and other conditions
IL268125A (en) Pridopidine for the treatment of fragile x syndrome
IL259856A (en) A preparation for the treatment of Crigler-Najjar syndrome
ZA201706945B (en) Fixed dose combinations comprising etc1002 and one or more statins for treating or reducing cardiovascular risk
IL274146A (en) Antisense therapy in Engelmann syndrome
PL3362449T3 (pl) Pochodne sybiriliny do zastosowania do zapobiegania i/lub leczenia zaburzeń związanych z nekroptozą komórkową
IL244343A0 (en) Methods for the treatment of fragile x syndrome and related disorders
PT3209317T (pt) Terlipressina para tratamento da síndrome hepatorrenal de tipo 1
IL265693A (en) Prevention and treatment of diabetic kidney disease
AP2015008338A0 (en) Compounds for the treatment and prevention of retroviral infections
HUE066511T2 (hu) Készítmények hunter-szindróma kezelésére
GB201718483D0 (en) Pharmaceutical composition for treatment of meopausal syndrome and preparation method thereof
PT3191112T (pt) Compostos de dipeptidilcetoamida e sua utilização para o tratamento e/ou prevenção da acumulação de gordura
HK1262955A1 (en) Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
HK1262747A1 (en) Antisense oligomers for treatment of alagille syndrome
PL3280430T3 (pl) Peptyd QBP1 do stosowania w zapobieganiu lub leczeniu zespołu stresu pourazowego, ostrego zaburzenia stresowego oraz ogólnego zespołu adaptacyjnego
GB201518489D0 (en) Methods for the treatment and prevention of ebola