HRP20181824T1 - Protusmislene molekule i postupci za liječenje patoloških stanja - Google Patents

Protusmislene molekule i postupci za liječenje patoloških stanja

Info

Publication number
HRP20181824T1
HRP20181824T1 HRP20181824TT HRP20181824T HRP20181824T1 HR P20181824 T1 HRP20181824 T1 HR P20181824T1 HR P20181824T T HRP20181824T T HR P20181824TT HR P20181824 T HRP20181824 T HR P20181824T HR P20181824 T1 HRP20181824 T1 HR P20181824T1
Authority
HR
Croatia
Prior art keywords
procedures
treatment
pathological conditions
miscellaneous
molecules
Prior art date
Application number
HRP20181824TT
Other languages
English (en)
Croatian (hr)
Inventor
Stephen Wilton
Sue Fletcher
Abbie Adams
Penny Meloni
Original Assignee
The University Of Western Australia
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
Priority claimed from AU2009905549A external-priority patent/AU2009905549A0/en
Application filed by The University Of Western Australia filed Critical The University Of Western Australia
Publication of HRP20181824T1 publication Critical patent/HRP20181824T1/hr

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    • 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
    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/351—Conjugate
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Oncology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
HRP20181824TT 2009-11-12 2010-11-12 Protusmislene molekule i postupci za liječenje patoloških stanja HRP20181824T1 (hr)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2009905549A AU2009905549A0 (en) 2009-11-12 Antisense Molecules and Methods for Treating Pathologies
EP10829367.1A EP2499249B1 (en) 2009-11-12 2010-11-12 Antisense molecules and methods for treating pathologies
PCT/AU2010/001520 WO2011057350A1 (en) 2009-11-12 2010-11-12 Antisense molecules and methods for treating pathologies

Publications (1)

Publication Number Publication Date
HRP20181824T1 true HRP20181824T1 (hr) 2019-01-25

Family

ID=43991096

Family Applications (1)

Application Number Title Priority Date Filing Date
HRP20181824TT HRP20181824T1 (hr) 2009-11-12 2010-11-12 Protusmislene molekule i postupci za liječenje patoloških stanja

Country Status (23)

Country Link
US (7) US8637483B2 (enExample)
EP (2) EP2499249B1 (enExample)
JP (6) JP5963678B2 (enExample)
KR (9) KR102487132B1 (enExample)
CN (2) CN105838714B (enExample)
AU (5) AU2010317599B2 (enExample)
BR (2) BR112012011195B1 (enExample)
CA (1) CA2780563C (enExample)
CY (1) CY1121198T1 (enExample)
DK (1) DK2499249T3 (enExample)
ES (1) ES2693459T3 (enExample)
HR (1) HRP20181824T1 (enExample)
HU (1) HUE040445T2 (enExample)
IL (6) IL297299A (enExample)
LT (1) LT2499249T (enExample)
NZ (2) NZ626359A (enExample)
PL (1) PL2499249T3 (enExample)
PT (1) PT2499249T (enExample)
RS (1) RS58079B1 (enExample)
SI (1) SI2499249T1 (enExample)
SM (1) SMT201800579T1 (enExample)
TR (1) TR201816523T4 (enExample)
WO (1) WO2011057350A1 (enExample)

Families Citing this family (102)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
EP1766010B1 (en) 2004-06-28 2011-02-16 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
DK3133160T3 (en) 2008-10-24 2019-04-01 Sarepta Therapeutics Inc EXON SKIP COMPOSITIONS FOR DMD
BR112012011195B1 (pt) 2009-11-12 2024-02-15 The University Of Western Australia Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica
JP6141018B2 (ja) 2009-12-24 2017-06-07 バイオマリン テクノロジーズ ベー．フェー． 炎症性障害を治療するための分子
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
WO2012109296A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
ES2748868T3 (es) 2011-12-28 2020-03-18 Nippon Shinyaku Co Ltd Acido nucleico antisentido
JP6928025B2 (ja) * 2012-01-27 2021-09-01 バイオマリン テクノロジーズ ベー．フェー． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
JP2015509922A (ja) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
DE102012103041A1 (de) * 2012-04-10 2013-10-10 Eberhard-Karls-Universität Tübingen Universitätsklinikum Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden
CN110257379B (zh) * 2012-07-03 2023-08-11 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
EP3885439A1 (en) * 2012-12-20 2021-09-29 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
KR20220061280A (ko) * 2013-03-14 2022-05-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
EP3633035A1 (en) * 2013-03-14 2020-04-08 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
WO2014172669A1 (en) * 2013-04-20 2014-10-23 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
MX2016002044A (es) * 2013-08-16 2016-08-17 Rana Therapeutics Inc Composiciones y metodos para modular el acido ribonucleico.
DK3118311T3 (en) * 2014-03-12 2019-03-11 Nippon Shinyaku Co Ltd Antisense nucleic acid
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP4596047A3 (en) * 2014-06-17 2025-10-22 Nippon Shinyaku Co., Ltd. Antisense nucleic acid for use in treating duchenne muscular dystrophy
ES3014437T3 (en) * 2014-08-09 2025-04-22 The Res Institute At Nationwide Childrens Hospital Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
CN106714845A (zh) * 2014-08-11 2017-05-24 得克萨斯州大学系统董事会 通过crispr/cas9介导的基因编辑预防肌营养不良
CN106714826A (zh) 2014-09-07 2017-05-24 西莱克塔生物科技公司 用于减弱基因表达调节抗病毒转移载体免疫应答的方法和组合物
JP6867945B2 (ja) 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
KR101968880B1 (ko) 2015-09-15 2019-04-12 니뽄 신야쿠 가부시키가이샤 안티센스 핵산
TW201722439A (zh) 2015-10-09 2017-07-01 波濤生命科學有限公司 寡核苷酸組合物及其方法
WO2017060731A1 (en) 2015-10-09 2017-04-13 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
EA201890908A1 (ru) 2015-10-09 2018-10-31 Сарепта Терапьютикс, Инк. Композиции и способы для лечения мышечной дистрофии дюшенна и сходных нарушений
US12318489B2 (en) 2015-10-26 2025-06-03 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
FR3044926B1 (fr) * 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
AU2016370653A1 (en) 2015-12-14 2018-06-21 Cold Spring Harbor Laboratory Antisense oligomers for treatment of Autosomal Dominant Mental Retardation-5 and Dravet Syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
CN107338250B (zh) * 2016-05-03 2021-01-26 台中荣民总医院 对突变型多巴脱羧酶基因做剪接调整的反义寡核苷酸及其使用方法
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
MA49775A (fr) 2016-05-24 2020-06-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
EP3464306B1 (en) 2016-05-24 2024-03-27 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MA45183A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
TW201811807A (zh) * 2016-06-30 2018-04-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
SG10201609048RA (en) * 2016-10-28 2018-05-30 Agency Science Tech & Res Antisense oligonucleotides
SMT202200440T1 (it) 2016-12-19 2023-01-13 Sarepta Therapeutics Inc Coniugati di oligomeri per salto di esone per distrofia muscolare
EP3554552B1 (en) 2016-12-19 2022-08-17 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
KR102552428B1 (ko) 2016-12-19 2023-07-06 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR20190104381A (ko) * 2017-01-06 2019-09-09 어비디티 바이오사이언시스 엘엘씨 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
AU2017407272B2 (en) * 2017-03-30 2024-06-13 Kyoto University Method for inducing exon skipping by genome editing
WO2019018383A1 (en) 2017-07-18 2019-01-24 Calimmune, Inc. COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
LT3673080T (lt) 2017-08-25 2023-12-27 Stoke Therapeutics, Inc. Priešprasminiai oligomerai, skirti būklėms ir ligoms gydyti
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
EP3687577A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
CN118638788A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
JP2021526796A (ja) * 2018-06-13 2021-10-11 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマー
EP3810150A4 (en) * 2018-06-14 2023-01-25 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
TW202449155A (zh) * 2018-07-27 2024-12-16 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
JP2021533200A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 顔面・肩甲・上腕筋ジストロフィーを処置するための筋標的化複合体およびそれらの使用
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20210308273A1 (en) 2018-08-02 2021-10-07 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
SG11202103938UA (en) * 2018-11-02 2021-05-28 Biomarin Tech Bv Bispecific antisense oligonucleotides for dystrophin exon skipping
MX2021006726A (es) 2018-12-07 2021-09-23 Univ Oxford Innovation Ltd Enlazadores.
CA3122281A1 (en) * 2018-12-13 2020-06-18 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3897745A1 (en) 2018-12-23 2021-10-27 CSL Behring LLC Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome
AU2019417700A1 (en) 2018-12-23 2021-07-08 Csl Behring L.L.C. Donor T-cells with kill switch
CN114206396A (zh) 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
CN114466682A (zh) * 2019-08-02 2022-05-10 全国儿童医院研究所 用于治疗基于肌营养不良蛋白的肌病的靶向外显子44的核酸和包含所述核酸的重组腺相关病毒
TW202136511A (zh) * 2019-12-19 2021-10-01 日商日本新藥股份有限公司 能進行外顯子跳讀之反義核酸
TWI874546B (zh) 2019-12-26 2025-03-01 日商日本新藥股份有限公司 誘導外顯子５０的跳讀的反義核酸
MX2022011499A (es) 2020-03-19 2022-10-07 Avidity Biosciences Inc Composiciones y metodos para tratar la distrofia muscular facioescapulohumeral.
EP4150092A4 (en) 2020-05-11 2024-11-06 Stoke Therapeutics, Inc. OPA1 ANTISENSE OLIGOMERS FOR THE TREATMENT OF DISEASES AND CONDITIONS
AU2021294317A1 (en) 2020-06-26 2023-02-23 Csl Behring Llc Donor T-cells with kill switch
WO2022171972A1 (en) * 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
IL309626A (en) 2021-06-23 2024-02-01 Nippon Shinyaku Co Ltd A combination of antisense oligomers
US11771776B2 (en) * 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
AU2022309028A1 (en) 2021-07-09 2024-01-25 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
CA3244382A1 (en) 2021-12-27 2025-02-26 Nippon Shinyaku Co Ltd PROCESS FOR THE PRODUCTION OF OLIGONUCLEIC ACID COMPOUNDS
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US20250170259A1 (en) 2022-03-03 2025-05-29 Yale University Compositions and methods for delivering therapeutic polynucleotides for exon skipping
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
US12071621B2 (en) 2022-04-05 2024-08-27 Avidity Biosciences, Inc. Anti-transferrin receptor antibody-PMO conjugates for inducing DMD exon 44 skipping
WO2025072530A2 (en) * 2023-09-26 2025-04-03 Regeneron Pharmaceuticals, Inc. Compositions and methods comprising small nuclear rna (snrna) for treating dmd
KR20250054362A (ko) 2023-10-16 2025-04-23 주식회사 엘지에너지솔루션 이차전지
WO2025179121A1 (en) * 2024-02-21 2025-08-28 Research Institute At Nationwide Children's Hospital Exon 17-targeted nucleic acids, compositions, and methods for treatment of dystrophin-based myopathies

Family Cites Families (132)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5138722B1 (enExample) 1970-12-30 1976-10-23
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5190931A (en) 1983-10-20 1993-03-02 The Research Foundation Of State University Of New York Regulation of gene expression by employing translational inhibition of MRNA utilizing interfering complementary MRNA
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
EP0215942B1 (en) 1985-03-15 1995-07-12 Antivirals Inc. Polynucleotide assay reagent and method
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US20020123476A1 (en) 1991-03-19 2002-09-05 Emanuele R. Martin Therapeutic delivery compositions and methods of use thereof
ES2153379T3 (es) 1992-03-31 2001-03-01 Abbott Lab Procedimiento de reaccion de ligasa en cadena multiplex.
US5869252A (en) 1992-03-31 1999-02-09 Abbott Laboratories Method of multiplex ligase chain reaction
US6806084B1 (en) 1992-06-04 2004-10-19 The Regents Of The University Of California Methods for compositions for in vivo gene delivery
EP1251170A3 (en) 1992-07-17 2002-10-30 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of NF-kappaB dependent animal diseases
US5985558A (en) 1997-04-14 1999-11-16 Isis Pharmaceuticals Inc. Antisense oligonucleotide compositions and methods for the inibition of c-Jun and c-Fos
ES2290951T3 (es) 1993-05-11 2008-02-16 The University Of North Carolina At Chapel Hill Oligonucleotidos antisentido que combaten el corte y empalme aberrante y metodos de uso de los mismos.
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US6391636B1 (en) 1994-05-31 2002-05-21 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of raf gene expression
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
GB9510718D0 (en) 1995-05-26 1995-07-19 Sod Conseils Rech Applic Antisense oligonucleotides
PL328563A1 (en) 1996-02-14 1999-02-01 Isis Pharmaceuticals Inc Oligonucleotides of modified sugar residues
WO1997034638A1 (en) 1996-03-20 1997-09-25 The Regents Of The University Of California Antisense approach to gene inhibition
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
US20030114401A1 (en) 2001-12-06 2003-06-19 Isis Pharmaceuticals Inc. Antisense modulation of Ship-1 expression
GB9819999D0 (en) 1998-09-14 1998-11-04 Univ London Treatment of cancer
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
JP2002529499A (ja) 1998-11-13 2002-09-10 イーライ・リリー・アンド・カンパニー 痛みの治療方法
CA2360997A1 (en) 1999-01-29 2000-08-03 Avi Biopharma, Inc. Non-invasive method for detecting target rna
US20020049173A1 (en) 1999-03-26 2002-04-25 Bennett C. Frank Alteration of cellular behavior by antisense modulation of mRNA processing
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
EP1191941A4 (en) 1999-06-21 2006-12-13 Murdoch Childrens Res Inst METHOD OF PROPHYLAXIS AND / OR TREATMENT OF CLINICAL DISORDERS
US7163695B2 (en) 1999-12-29 2007-01-16 Mixson A James Histidine copolymer and methods for using same
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
CA2396068A1 (en) 2000-01-04 2001-07-12 Avi Biopharma, Inc. Antisense antibacterial cell division composition and method
AU4036600A (en) 2000-03-28 2001-10-08 Isis Pharmaceuticals Inc Alteration of cellular behavior by antisense modulation of mrna processing
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
CN100422320C (zh) 2000-04-28 2008-10-01 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
PT1278761E (pt) 2000-05-01 2005-08-31 Hybridon Inc Modulacao da estimulacao imunitaria mediada por oligonucleotidos de cpg, por modificacao posicional de nucleosidos
KR20020097241A (ko) 2000-05-04 2002-12-31 에이브이아이 바이오파마 인코포레이티드 스플라이스-영역 안티센스 조성물 및 방법
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
JP4836366B2 (ja) 2000-08-25 2011-12-14 雅文 松尾 デュシェンヌ型筋ジストロフィー治療剤
AU2001286966B2 (en) 2000-08-30 2007-03-01 Avi Biopharma, Inc. Method for analysis of oligonucleotide analogs
US20070037165A1 (en) 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
US6900016B1 (en) 2000-09-08 2005-05-31 Applera Corporation Polymorphisms in known genes associated with inflammatory autoimmune disease, methods of detection and uses thereof
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
EP1319014A1 (en) 2000-09-20 2003-06-18 Isis Pharmaceuticals, Inc. Antisense modulation of flip-c expression
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
US6689615B1 (en) 2000-10-04 2004-02-10 James Murto Methods and devices for processing blood samples
CN2507125Y (zh) * 2000-10-11 2002-08-21 信一精密株式会社 具有多段时间调节功能的定时器
JP3781687B2 (ja) 2001-02-23 2006-05-31 松下電器産業株式会社 遺伝子診断装置及び遺伝子診断方法
US6656732B1 (en) 2001-05-18 2003-12-02 Isis Pharmaceuticals, Inc. Antisense inhibition of src-c expression
US7499863B2 (en) 2001-07-06 2009-03-03 Dialogic Corporation System and method for constructing phrases for a media server
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
ATE517992T1 (de) 2002-11-14 2011-08-15 Dharmacon Inc Funktionelle und hyperfunktionelle sirna
US7314750B2 (en) 2002-11-20 2008-01-01 Affymetrix, Inc. Addressable oligonucleotide array of the rat genome
US7250289B2 (en) 2002-11-20 2007-07-31 Affymetrix, Inc. Methods of genetic analysis of mouse
EP1568769A4 (en) 2002-11-25 2008-08-06 Masafumi Matsuo ENA-NUCLEIC ACID MEDICAMENTS THAT CHANGE SPLICING IN THE MRNA PRESETTER
AU2003293062A1 (en) 2002-11-25 2004-06-18 Telesector Resources Group, Inc. Methods and systems for automatic forwarding of communications to a preferred device
CA2524255C (en) 2003-03-21 2014-02-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
US7468418B2 (en) 2003-04-29 2008-12-23 Avi Biopharma., Inc. Compositions for enhancing transport of molecules into cells
DK1495769T3 (da) 2003-07-11 2008-06-23 Lbr Medbiotech B V Mannose-6-phosphat-receptormedieret gentransfer til muskelceller
DE602004030583D1 (de) 2003-08-05 2011-01-27 Avi Biopharma Inc Oligonukleotidanalog und verfahren zur behandlung von flavivirusinfektionen
US20050048495A1 (en) 2003-08-29 2005-03-03 Baker Brenda F. Isoform-specific targeting of splice variants
US20050153935A1 (en) 2003-09-12 2005-07-14 Iversen Patrick L. Compound and method for treating androgen-independent prostate cancer
US20050171044A1 (en) 2003-12-24 2005-08-04 Stein David A. Oligonucleotide compound and method for treating nidovirus infections
WO2005115419A1 (en) 2004-05-17 2005-12-08 Board Of Trustees Of The University Of Illinois USES OF BISPECIFIC ANTIBODY (Biab) COATED DENDRITIC CELLS PULSED WITH ANTIGENS
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
EP1766010B1 (en) 2004-06-28 2011-02-16 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
FR2873294B1 (fr) 2004-07-26 2008-05-09 Greenpharma Sa Sa Association de medicaments
FR2874384B1 (fr) * 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
US8129352B2 (en) 2004-09-16 2012-03-06 Avi Biopharma, Inc. Antisense antiviral compound and method for treating ssRNA viral infection
US20060148740A1 (en) 2005-01-05 2006-07-06 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
US20120122801A1 (en) 2005-01-05 2012-05-17 Prosensa B.V. Mannose-6-phosphate receptor mediated gene transfer into muscle cells
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
KR20080031164A (ko) 2005-04-22 2008-04-08 아카데미슈 지켄후이스 라이덴 ＳＲ 단백질의 결합의 방해에 의해 그리고 이차 ＲＮＡ구조의 방해에 의한 ｐｒｅ－ｍＲＮＡ에서 엑손 인식의조절
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007028065A2 (en) 2005-08-30 2007-03-08 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
US8524676B2 (en) 2005-09-08 2013-09-03 Sarepta Therapeutics, Inc. Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
WO2007058894A2 (en) 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
WO2007133812A2 (en) 2005-12-30 2007-11-22 Philadelphia Health & Education Corporation, D/B/A Drexel University College Of Medicine Improved carriers for delivery of nucleic acid agents to cells and tissues
JP2010505741A (ja) 2006-05-10 2010-02-25 エイブイアイ バイオファーマ， インコーポレイテッド カチオン性のサブユニット間結合を有するオリゴヌクレオチドアナログ
US8785407B2 (en) 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20070265215A1 (en) 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
EP1857548A1 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
ES2694726T3 (es) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
EP2119783A1 (en) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
DK3133160T3 (en) 2008-10-24 2019-04-01 Sarepta Therapeutics Inc EXON SKIP COMPOSITIONS FOR DMD
DK2344637T4 (en) 2008-10-27 2018-04-23 Biomarin Tech Bv Methods and means for efficiently skipping exon 45 in Duchenne muscular dystrophy pre-mRNA
WO2010080554A1 (en) 2008-12-17 2010-07-15 Avi Biopharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
PL2417257T3 (pl) 2009-04-10 2016-11-30 Antysensowne oligonukleotydy tricyklo-dna, kompozycje i sposoby leczenia choroby
EP2421971B1 (en) 2009-04-24 2016-07-06 BioMarin Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
EP2258863A1 (en) 2009-05-25 2010-12-08 Universita'Degli Studi di Roma "La Sapienza" miRNA biomarkers for the diagnosis of duchenne muscular dystrophy progression, for monitoring therapeutic interventions, and as therapeutics
WO2010136417A1 (en) 2009-05-25 2010-12-02 Università Degli Studi Di Roma "La Sapienza" miR-31 IN DUCHENNE MUSCULAR DYSTROPHY THERAPY
ITTO20090487A1 (it) 2009-06-26 2010-12-27 Univ Ferrara Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd)
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011024077A2 (en) 2009-08-31 2011-03-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Exon skipping therapy for functional amelioration of semi functional dystrophin in becker and duchenne muscular dystrophy
IT1397011B1 (it) 2009-10-14 2012-12-20 Univ Ferrara Nanoparticella del tipo core-shell idonea per la veicolazione di oligonucleotidi terapeutici in tessuti bersaglio e suo impiego per la preparazione di un medicamento per il trattamento della distrofia muscolare di duchenne.
BR112012011195B1 (pt) 2009-11-12 2024-02-15 The University Of Western Australia Oligonucleotídeo antissentido, composição farmacêutica e uso de uma composição farmacêutica
KR101944119B1 (ko) 2009-11-13 2019-01-30 사렙타 쎄러퓨틱스 인코퍼레이티드 안티센스 항바이러스 화합물 및 인플루엔자 바이러스 감염을 치료하는 방법
CN102934397B (zh) 2010-04-08 2016-01-06 法国电信公司 控制家庭网络的家庭网关的接入点的方法
US9050373B2 (en) 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
US9241929B2 (en) 2010-06-28 2016-01-26 Masatoshi Hagiwara Prophylactic or ameliorating agent for genetic diseases
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
PT2623507T (pt) 2010-09-30 2016-12-23 Nippon Shinyaku Co Ltd Derivado de ácido morfolino-nucleico
WO2012109296A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
KR102183273B1 (ko) 2011-05-05 2020-11-27 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US20140080896A1 (en) 2011-08-30 2014-03-20 The Regents Of The University Of California Identification of small molecules that facilitate therapeutic exon skipping
EP2750715B1 (en) 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
DK2581448T3 (en) 2011-10-13 2015-04-27 Ass Inst De Myologie Tricyclo-DNA phosphorothioate
ES2748868T3 (es) 2011-12-28 2020-03-18 Nippon Shinyaku Co Ltd Acido nucleico antisentido
JP2015509922A (ja) 2012-01-27 2015-04-02 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． デュシェンヌ型及びベッカー型筋ジストロフィーの治療のための改善された特徴を有するｒｎａ調節オリゴヌクレオチド
NZ700399A (en) 2012-03-20 2016-07-29 Sarepta Therapeutics Inc Boronic acid conjugates of oligonucleotide analogues
CN110257379B (zh) 2012-07-03 2023-08-11 马林生物科技有限公司 用于治疗肌肉萎缩症患者的寡核苷酸
EP3885439A1 (en) 2012-12-20 2021-09-29 Sarepta Therapeutics, Inc. Improved exon skipping compositions for treating muscular dystrophy
EP3633035A1 (en) 2013-03-14 2020-04-08 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
KR20220061280A (ko) 2013-03-14 2022-05-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
WO2014172669A1 (en) 2013-04-20 2014-10-23 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
EA201890811A1 (ru) 2015-09-30 2018-09-28 Сарепта Терапьютикс, Инк. Способы лечения мышечной дистрофии
TW201811807A (zh) 2016-06-30 2018-04-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物

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