PL2206781T3 - Antysensowne oligonukleotydy do indukowania pomijania eksonów i sposoby ich zastosowania - Google Patents

Antysensowne oligonukleotydy do indukowania pomijania eksonów i sposoby ich zastosowania

Info

Publication number
PL2206781T3
PL2206781T3 PL10004274T PL10004274T PL2206781T3 PL 2206781 T3 PL2206781 T3 PL 2206781T3 PL 10004274 T PL10004274 T PL 10004274T PL 10004274 T PL10004274 T PL 10004274T PL 2206781 T3 PL2206781 T3 PL 2206781T3
Authority
PL
Poland
Prior art keywords
exon skipping
methods
antisense oligonucleotides
inducing exon
inducing
Prior art date
Application number
PL10004274T
Other languages
English (en)
Inventor
Stephen Donald Wilton
Sue Fletcher
Graham Mcclorey
Original Assignee
Univ 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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=35781530&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL2206781(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from AU2004903474A external-priority patent/AU2004903474A0/en
Application filed by Univ Western Australia filed Critical Univ Western Australia
Publication of PL2206781T3 publication Critical patent/PL2206781T3/pl

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—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
    • C12N2310/32—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
    • C12N2310/33—Chemical structure of the base
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/3519—Fusion with another nucleic acid
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
PL10004274T 2004-06-28 2005-06-28 Antysensowne oligonukleotydy do indukowania pomijania eksonów i sposoby ich zastosowania PL2206781T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2004903474A AU2004903474A0 (en) 2004-06-28 Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof
EP10004274.6A EP2206781B1 (en) 2004-06-28 2005-06-28 Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP20050754344 EP1766010B1 (en) 2004-06-28 2005-06-28 Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Publications (1)

Publication Number Publication Date
PL2206781T3 true PL2206781T3 (pl) 2016-06-30

Family

ID=35781530

Family Applications (2)

Application Number Title Priority Date Filing Date
PL05754344T PL1766010T3 (pl) 2004-06-28 2005-06-28 Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania
PL10004274T PL2206781T3 (pl) 2004-06-28 2005-06-28 Antysensowne oligonukleotydy do indukowania pomijania eksonów i sposoby ich zastosowania

Family Applications Before (1)

Application Number Title Priority Date Filing Date
PL05754344T PL1766010T3 (pl) 2004-06-28 2005-06-28 Antysensowne oligonukleotydy do indukcji pominięcia egzonu i sposoby ich zastosowania

Country Status (14)

Country Link
US (48) USRE47769E1 (pl)
EP (9) EP3808845A1 (pl)
AT (1) ATE498685T1 (pl)
CY (2) CY1111447T1 (pl)
DE (1) DE602005026386D1 (pl)
DK (2) DK2206781T3 (pl)
ES (3) ES2627917T3 (pl)
HK (2) HK1216545A1 (pl)
HR (2) HRP20110352T1 (pl)
HU (1) HUE028632T2 (pl)
PL (2) PL1766010T3 (pl)
PT (1) PT1766010E (pl)
SI (2) SI2206781T1 (pl)
WO (1) WO2006000057A1 (pl)

Families Citing this family (194)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
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
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
WO2005072527A2 (en) 2004-01-23 2005-08-11 Avi Biopharma, Inc. Antisense oligomers and methods for inducing immune tolerance and immunosuppression
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
ES2627917T3 (es) 2004-06-28 2017-08-01 The University Of Western Australia Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2006086667A2 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (en) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
JP6047270B2 (ja) * 2006-08-11 2016-12-21 バイオマリン テクノロジーズ ベー．フェー． Ｄｎａリピートの不安定性に関連した遺伝的障害を治療するための方法及び手段
EP2066812A4 (en) * 2006-09-21 2010-04-28 Univ Rochester COMPOSITIONS AND METHODS RELATED TO PROTEIN SHIFT THERAPY FOR MYOTONE DYSTROPHY
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CA2691673A1 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
JP2010533170A (ja) 2007-07-12 2010-10-21 プロセンサ テクノロジーズ ビー．ブイ． 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子
WO2009008727A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
NZ584793A (en) * 2007-10-26 2012-05-25 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
CA2710013A1 (en) 2007-12-28 2009-07-09 Avi Biopharma, Inc. Immunomodulatory agents and methods of use
JP2011510678A (ja) 2008-02-08 2011-04-07 プロセンサ ホールディング ビーブイ Ｄｎａ反復不安定性関連遺伝性障害を治療するための方法及び手段
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
EP3133160B1 (en) * 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
SI2607484T1 (sl) * 2008-10-27 2016-11-30 Biomarin Technologies B.V. Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
CA2758189C (en) 2009-04-10 2020-12-29 Association Institut De Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
CN102459595A (zh) * 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
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)
EP2451461A4 (en) 2009-07-06 2013-05-29 Ontorii Inc NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF
EP2473607A2 (en) * 2009-08-31 2012-07-11 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
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
HUE040445T2 (hu) * 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
MX339050B (es) 2009-12-18 2016-05-09 Novartis Ag Composiciones organicas para tratar las enfermedades relacionadas con hsf1.
AU2014280918B2 (en) * 2009-12-18 2016-11-17 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat HSF1-related diseases
WO2011078797A2 (en) * 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
AU2010335039B2 (en) 2009-12-24 2015-03-26 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Molecule for treating an inflammatory disorder
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
IT1400425B1 (it) * 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
AU2011286539B2 (en) * 2010-08-05 2017-03-02 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
EP3118316A1 (en) 2010-09-02 2017-01-18 Université de Mons Agents useful in treating facioscapulohumeral muscular dystrophy
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
DK2620428T3 (da) 2010-09-24 2019-07-01 Wave Life Sciences Ltd Asymmetrisk hjælpegruppe
EP3702460A1 (en) 2010-11-12 2020-09-02 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
BR112013020273A2 (pt) 2011-02-08 2016-10-18 Charlotte Mecklenburg Hospital oligonucleotídeos antissenso
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2011367230B2 (en) 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
EP2750715B1 (en) 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
EP2766480B1 (en) 2011-10-11 2018-03-14 INSERM - Institut National de la Santé et de la Recherche Médicale Exon skipping therapy for dystrophic epidermolysis bullosa
GB201117880D0 (en) * 2011-10-17 2011-11-30 Ucl Business Plc Antisense oligonucleotides
KR20140097398A (ko) 2011-11-18 2014-08-06 사렙타 쎄러퓨틱스, 인코퍼레이티드 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트
CN108588073A (zh) * 2011-12-28 2018-09-28 日本新药株式会社 反义核酸
CA2862628C (en) * 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
US9920085B2 (en) 2012-03-20 2018-03-20 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
DE102012103041A1 (de) 2012-04-10 2013-10-10 Eberhard-Karls-Universität Tübingen Universitätsklinikum Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden
US9372901B2 (en) * 2012-04-27 2016-06-21 Quixey, Inc. Searching for software applications based on application attributes
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CN104583402A (zh) 2012-05-16 2015-04-29 Rana医疗有限公司 用于调节mecp2表达的组合物和方法
JP2015523854A (ja) 2012-05-16 2015-08-20 ラナ セラピューティクス インコーポレイテッド Ｓｍｎ遺伝子ファミリー発現を調節するための組成物及び方法
CN104603271B (zh) * 2012-07-03 2019-07-19 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
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
DK2872147T3 (da) 2012-07-13 2023-02-20 Wave Life Sciences Ltd Fremgangsmåde til fremstilling af kirale oligonukleotider
EP4678239A3 (en) 2012-08-01 2026-04-08 Ikaika Therapeutics, Inc. Mitigating tissue damage and fibrosis via latent transforming growth factor beta binding protein (ltbp4)
CN106414739A (zh) 2012-12-20 2017-02-15 萨雷普塔医疗公司 用于治疗肌营养不良症的改良的外显子跳跃组合物
EP3760720A1 (en) * 2013-03-14 2021-01-06 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014197748A2 (en) * 2013-06-05 2014-12-11 Duke University Rna-guided gene editing and gene regulation
EP3052632A4 (en) 2013-10-04 2017-03-29 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
BR112016020618B1 (pt) 2014-03-12 2023-04-04 Nippon Shinyaku Co., Ltd. Oligômero antissentido, composição farmacêutica, e, uso de um oligômero antissentido
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
US10724092B2 (en) 2014-06-10 2020-07-28 Erasmus University Medical Center Rotterdam Methods for characterizing alternatively or aberrantly spliced mRNA isoforms
PT3159409T (pt) 2014-06-17 2020-01-21 Nippon Shinyaku Co Ltd Ácido nucleico anti-sentido para utilização no tratamento da distrofia muscular de duchenne
EA201790551A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10676726B2 (en) 2015-02-09 2020-06-09 Duke University Compositions and methods for epigenome editing
US10525076B2 (en) 2015-02-20 2020-01-07 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
GB201503408D0 (en) 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US10273483B2 (en) 2015-04-08 2019-04-30 The University Of Chicago Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
US10324930B2 (en) * 2015-05-27 2019-06-18 Sap Se Database calculation engine with nested multiprovider merging
US11020417B2 (en) 2015-06-04 2021-06-01 Sarepta Therapeutics, Inc Methods and compounds for treatment of lymphocyte-related diseases and conditions
KR20240132120A (ko) 2015-08-25 2024-09-02 듀크 유니버시티 Rna-가이드된 엔도뉴클레아제를 이용하는 게놈 조작에서 특이성을 개선하는 조성물 및 방법
HUE050061T2 (hu) 2015-09-15 2020-12-28 Nippon Shinyaku Co Ltd Antiszensz nukleinsav
WO2017047741A1 (ja) * 2015-09-16 2017-03-23 日本新薬株式会社 筋萎縮症治療用アンチセンス核酸
JP6282620B2 (ja) * 2015-09-28 2018-02-21 東芝テック株式会社 商品販売データ処理装置及びプログラム
JP2018530560A (ja) 2015-10-09 2018-10-18 サレプタ セラピューティクス， インコーポレイテッド デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法
MA46427A (fr) * 2015-10-09 2018-08-15 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
FR3044926B1 (fr) 2015-12-09 2020-01-31 Genethon Outils de therapie genique efficaces pour le saut de l'exon 53 de la dystrophine
KR20250112919A (ko) 2015-12-21 2025-07-24 노파르티스 아게 타우 발현을 감소시키기 위한 조성물 및 방법
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
EP3443081A4 (en) 2016-04-13 2019-10-30 Duke University CRISPR / CAS9-BASED REPRESSORS FOR IN VIVO SHUT-OFF OF GEN-TARGETS AND METHOD OF USE
MA45158A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Composition pharmaceutique comprenant de l'éteplirsen
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
MX384727B (es) 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros
US10947533B2 (en) 2016-05-24 2021-03-16 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
TWI737736B (zh) 2016-05-24 2021-09-01 美商薩羅塔治療公司 製備磷醯二胺嗎啉代寡聚物之方法
MX390906B (es) 2016-05-24 2025-03-21 Sarepta Therapeutics Inc Procesos para preparar oligómeros de morfolino fosforodiamidato.
KR20190024977A (ko) 2016-06-30 2019-03-08 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머
US11655470B2 (en) 2016-07-05 2023-05-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing COL6-related disorders and methods for treating same
AU2017291960B2 (en) * 2016-07-05 2024-03-14 Biomarin Technologies B.V. Pre-mRNA splice switching or modulating oligonucleotides comprising bicyclic scaffold moieties, with improved characteristics for the treatment of genetic disorders
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
JOP20190104A1 (ar) * 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
PT4122497T (pt) 2016-12-19 2024-06-12 Sarepta Therapeutics Inc Conjugados oligoméricos de salto de exão para a distrofia muscular
ES3051834T3 (en) 2016-12-19 2025-12-30 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
NZ755416A (en) 2016-12-19 2023-05-26 Sarepta Therapeutics Inc Exon skipping oligomer conjugates for muscular dystrophy
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
WO2018150196A1 (en) 2017-02-17 2018-08-23 Oxford University Innovation Limited Cell penetrating peptides modified by glycosylation
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CA3065523A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CA3070242A1 (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
WO2019055460A1 (en) * 2017-09-13 2019-03-21 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR THE TREATMENT OF TRANSPOSON-ASSOCIATED DISEASES
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
JP2020536060A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
US11180541B2 (en) * 2017-09-28 2021-11-23 Geltor, Inc. Recombinant collagen and elastin molecules and uses thereof
WO2019075360A1 (en) 2017-10-13 2019-04-18 Selecta Biosciences, Inc. METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES
US11555189B2 (en) 2017-10-18 2023-01-17 Sarepta Therapeutics, Inc. Antisense oligomer compounds
CN118638787A (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
JP2021521140A (ja) * 2018-04-12 2021-08-26 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
WO2019209764A2 (en) * 2018-04-26 2019-10-31 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
CA3098624A1 (en) 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
US20220251551A1 (en) * 2018-06-13 2022-08-11 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
JP7579147B2 (ja) * 2018-06-14 2024-11-07 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およびオリゴマーコンジュゲート
JP7345466B2 (ja) 2018-06-26 2023-09-15 日本新薬株式会社 アンチセンスオリゴヌクレオチドを含有する組成物およびデュシェンヌ型筋ジストロフィーの治療へのその使用
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020028832A1 (en) 2018-08-02 2020-02-06 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
GB201812980D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
GB201812972D0 (en) 2018-08-09 2018-09-26 Univ Oxford Innovation Ltd Cell-penetrating peptides
EP3844278A4 (en) * 2018-08-27 2022-05-25 North Carolina State University TARGETING KIT WITH SPLICE-SWITCHING OLIGONUCLEOTIDES FOR INDUCING APOPTOSIS OF MASTS CELLS
US12331293B2 (en) * 2018-11-02 2025-06-17 Biomarin Technologies B.V. Bispecific antisense oligonucleotides for dystrophin exon skipping
AU2019394177A1 (en) 2018-12-07 2021-06-10 Oxford University Innovation Limited Linkers
CN113518825A (zh) 2018-12-23 2021-10-19 美国杰特贝林生物制品有限公司 Wiskott-aldrich综合征的造血干细胞基因疗法
EP3897831A1 (en) 2018-12-23 2021-10-27 CSL Behring LLC Donor t-cells with kill switch
US20220098578A1 (en) 2019-01-31 2022-03-31 Bar Ilan University Neoantigens created by aberrant-induced splicing and uses thereof in enhancing immunotherapy
MX2019001389A (es) 2019-01-31 2020-02-20 Atso Corp Affairs S A De C V Oligonucleotidos antisentido de las glutation s transferasa para el tratamiento del cancer.
GB2597416A (en) 2019-04-12 2022-01-26 Geltor Inc Recombinant elastin and production thereof
TWI887234B (zh) 2019-04-14 2025-06-21 美國公爵大學 治療杜興氏（ｄｕｃｈｅｎｎｅ）肌肉失養症之大型突變熱點之ａａｖ載體介導之刪除
WO2020219820A1 (en) 2019-04-25 2020-10-29 Avidity Biosciences, Inc. Nucleic acid compositions and methods of multi-exon skipping
SG11202112499VA (en) 2019-05-15 2021-12-30 Univ Miami Treatment of heart disease by disruption of the anchoring of pp2a
CA3141863A1 (en) 2019-05-28 2020-12-03 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
EP4054639A4 (en) * 2019-11-08 2025-04-30 North Carolina State University EXON SKIPPING OF FC-EPSILON-RI-BETA AND MS4A6A IN COMBINATION FOR THE TREATMENT OF ALLERGIC DISEASES
EP4079329A4 (en) 2019-12-19 2024-04-17 Nippon Shinyaku Co., Ltd. ANTISENSE NUCLEIC ACID ENABLING EXON SKIPPING
IL294271A (en) 2019-12-26 2022-08-01 Nippon Shinyaku Co Ltd Antisense nucleic acid that induces skipping of exon 50
PE20230237A1 (es) 2020-02-28 2023-02-07 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido que inducen la omision del exon 51
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
WO2021263070A1 (en) 2020-06-26 2021-12-30 Csl Behring Llc Donor t-cells with kill switch
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11987795B2 (en) 2020-11-24 2024-05-21 The Broad Institute, Inc. Methods of modulating SLC7A11 pre-mRNA transcripts for diseases and conditions associated with expression of SLC7A11
KR20230117182A (ko) 2020-12-04 2023-08-07 다인 세라퓨틱스, 인크. 항체-올리고뉴클레오티드 복합체 및 그의 용도
WO2022173811A1 (en) * 2021-02-12 2022-08-18 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting genes associated with cystic fibrosis
CA3211038A1 (en) 2021-02-12 2022-08-18 Oxford University Innovation Limited Cell-penetrating peptide conjugates and methods of their use
AU2022262420A1 (en) * 2021-04-23 2023-11-16 Research Institute At Nationwide Children's Hospital Products and methods for treating muscular dystrophy
JPWO2022270585A1 (pl) 2021-06-23 2022-12-29
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
JP2024525613A (ja) * 2021-07-09 2024-07-12 ダイン セラピューティクス，インコーポレーテッド 筋標的化複合体およびジストロフィノパチーを処置するためのその使用
WO2023026994A1 (ja) 2021-08-21 2023-03-02 武田薬品工業株式会社 ヒトトランスフェリンレセプター結合ペプチド－薬物コンジュゲート
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
KR20240129006A (ko) 2021-12-27 2024-08-27 니뽄 신야쿠 가부시키가이샤 올리고 핵산 화합물의 제조 방법
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
WO2023168427A1 (en) 2022-03-03 2023-09-07 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
CN115820642B (zh) * 2022-11-11 2023-10-10 昆明理工大学 一种用于治疗杜氏肌营养不良症的CRISPR-Cas9系统
JP2026513831A (ja) 2023-04-03 2026-05-01 ジャズ ファーマシューティカルズ アイルランド リミテッド ナノ粒子取り込みの調節剤
EP4702144A1 (en) 2023-04-27 2026-03-04 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of chronic kidney disease
US11792125B1 (en) * 2023-05-16 2023-10-17 Citibank, N.A. Reducing network traffic by filtering network requests based on network request-related information systems and methods
WO2025085810A2 (en) 2023-10-18 2025-04-24 Sarepta Therapeutics, Inc. Antisense oligomers for treatment of centronuclear myopathies
WO2025165891A1 (en) 2024-01-29 2025-08-07 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof
WO2026151958A1 (en) 2025-01-10 2026-07-16 Entrada Therapeutics, Inc. Compounds and methods for skipping exon 51 in duchenne muscular dystrophy

Family Cites Families (130)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5138722B1 (pl) 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
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
US5521063A (en) 1985-03-15 1996-05-28 Antivirals Inc. Polynucleotide reagent containing chiral subunits and methods of use
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
WO1993020227A1 (en) * 1992-03-31 1993-10-14 Abbott Laboratories Method of multiplex ligase chain reaction
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
JPH07509133A (ja) 1992-07-17 1995-10-12 リボザイム・ファーマシューティカルズ・インコーポレイテッド 動物疾患の処置のための方法および剤
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
ATE368107T1 (de) 1993-05-11 2007-08-15 Univ North Carolina Antisense oligonukleotide die anomales splicing verhindern und deren verwendung
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
DK0882061T3 (da) 1996-02-14 2004-09-27 Isis Pharmaceuticals Inc Sukkermodificerede gapped oligonukleotider
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
ES2194536T3 (es) 1998-11-13 2003-11-16 Lilly Co Eli Combinacion de duloxetina con farmacos antiinflamatorios no esteroideos.
KR20010102992A (ko) 1999-01-29 2001-11-17 추후보정 표적 ｒｎａ를 검출하는 비-침입적 방법
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
AU768904B2 (en) 1999-06-21 2004-01-08 Murdoch Childrens Research Institute, The A method for the prophylaxis and/or treatment of medical disorders
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
CA2394758A1 (en) 1999-12-29 2001-07-05 A. James Mixson Histidine-containing copolymers enhance pharmaceutical agent delivery
WO2001049775A2 (en) 2000-01-04 2001-07-12 Avi Biopharma, Inc. Antisense antibacterial cell division composition and method
CA2400573A1 (en) 2000-03-28 2001-10-04 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
WO2001083695A2 (en) 2000-04-28 2001-11-08 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
AU2001257366B2 (en) 2000-05-01 2006-03-16 Idera Pharmaceuticals, Inc. Modulation of oligonucleotide CpG-mediated immune stimulation by positional modification of nucleosides
AU5752601A (en) 2000-05-04 2001-11-12 Avi Biopharma Inc Splice-region antisense composition and method
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
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
WO2002018656A2 (en) 2000-08-30 2002-03-07 Avi Biopharma, Inc. Method for analysis of oligonucleotide analogs
US6559279B1 (en) 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
US20070037165A1 (en) 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
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
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
US20030224353A1 (en) 2001-10-16 2003-12-04 Stein David A. Antisense antiviral agent and method for treating ssRNA viral infection
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
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
EP2530155B1 (en) * 2002-11-25 2016-03-16 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
EP1566047A4 (en) 2002-11-25 2012-10-03 Telesector Resources Group Inc METHODS AND SYSTEMS FOR NOTIFYING CALL TO A DEVICE
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
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
ES2302898T3 (es) 2003-07-11 2008-08-01 Lbr Medbiotech B.V. Transferencia de genes a celulas musculares mediada por el receptor de manosa-6-fosfato.
KR101032853B1 (ko) 2003-08-05 2011-05-06 에이브이아이 바이오파마 인코포레이티드 웨스트 나일 바이러스 감염을 치료하기 위한 약학적 조성물
US20050048495A1 (en) 2003-08-29 2005-03-03 Baker Brenda F. Isoform-specific targeting of splice variants
WO2005027833A2 (en) 2003-09-12 2005-03-31 Avi Biopharma, Inc. 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
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
ES2627917T3 (es) 2004-06-28 2017-08-01 The University Of Western Australia Oligonucleótidos no codificantes para la inducción de la omisión exónica y métodos de uso de los mismos
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
WO2006086667A2 (en) 2005-02-09 2006-08-17 Avi Bio Pharma, Inc. Antisense composition and method for treating muscle atrophy
US20090312532A1 (en) 2005-04-22 2009-12-17 Van Deutekom Judith Christina Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secodary rna structure
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
WO2007030691A2 (en) 2005-09-08 2007-03-15 Avi Biopharma, Inc. Antisense antiviral compound and method for treating picornavirus infection
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
US20090011004A1 (en) 2005-12-30 2009-01-08 Philadelphia Health & Education Corp., D/B/A/ Drexel University Of College Of Medicine Improved carriers for delivery of nucleic acid agents to cells and tissues
US8785407B2 (en) 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
LT2735568T (lt) 2006-05-10 2017-11-27 Sarepta Therapeutics, Inc. Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų
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
CA2691673A1 (en) 2007-06-29 2009-01-08 Avi Biopharma, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
NZ584793A (en) 2007-10-26 2012-05-25 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
EP3133160B1 (en) 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
SI2607484T1 (sl) 2008-10-27 2016-11-30 Biomarin Technologies B.V. Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije
US8592386B2 (en) 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
CA2758189C (en) 2009-04-10 2020-12-29 Association Institut De Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
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
US9034838B2 (en) 2009-05-25 2015-05-19 Universita 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
EP2473607A2 (en) 2009-08-31 2012-07-11 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.
HUE040445T2 (hu) 2009-11-12 2019-03-28 Univ Western Australia Antiszensz molekulák és eljárások betegségek kezelésére
AU2010319314C1 (en) 2009-11-13 2016-09-01 Sarepta Therapeutics, Inc. Antisense antiviral compound and method for treating influenza viral infection
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
WO2012001941A1 (ja) 2010-06-28 2012-01-05 Hagiwara Masatoshi 遺伝性疾患の予防・改善剤
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
CN103154009B (zh) 2010-09-30 2015-06-10 日本新药株式会社 吗啉基核酸衍生物
BR112013020273A2 (pt) 2011-02-08 2016-10-18 Charlotte Mecklenburg Hospital oligonucleotídeos antissenso
JP2012226016A (ja) 2011-04-15 2012-11-15 Toyota Industries Corp センサ用光ファイバ
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2011367230B2 (en) 2011-05-05 2017-08-10 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP2750715B1 (en) 2011-08-30 2018-10-31 The Regents of The University of California Identification of small molecules that enhance therapeutic exon skipping
US20140080896A1 (en) 2011-08-30 2014-03-20 The Regents Of The University Of California Identification of small molecules that facilitate therapeutic exon skipping
DK2581448T3 (en) 2011-10-13 2015-04-27 Ass Inst De Myologie Tricyclo-DNA phosphorothioate
CN108588073A (zh) 2011-12-28 2018-09-28 日本新药株式会社 反义核酸
CA2862628C (en) 2012-01-27 2021-08-24 Prosensa Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
DE102012203443A1 (de) 2012-03-05 2013-09-05 Robert Bosch Gmbh Verfahren zum Bilden eines Kontakts auf einem Halbleitersubstrat und Halbleitervorrichtung
US9920085B2 (en) 2012-03-20 2018-03-20 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
CN104603271B (zh) 2012-07-03 2019-07-19 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
CN106414739A (zh) 2012-12-20 2017-02-15 萨雷普塔医疗公司 用于治疗肌营养不良症的改良的外显子跳跃组合物
EP3760720A1 (en) 2013-03-14 2021-01-06 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
US9217148B2 (en) 2013-03-14 2015-12-22 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
HK1220154A1 (zh) 2013-03-15 2017-04-28 Sarepta Therapeutics, Inc. 改进的用於治疗肌营养不良的组合物
US20140314998A1 (en) 2013-04-18 2014-10-23 Brigham Young University Porous material for thermal and/or electrical isolation and methods of manufacture
US9862945B2 (en) 2013-04-20 2018-01-09 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
US20190054113A1 (en) 2015-09-30 2019-02-21 Sarepta Therapeutics, Inc. Methods for treating muscular dystrophy
US20200195590A1 (en) 2018-12-18 2020-06-18 Zipwhip, Inc. System and method for generating and transmitting automatic reply messages

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