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
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    • 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
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    • 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
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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
EP20050754344 EP1766010B1 (en) 2004-06-28 2005-06-28 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

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) EP2933332A1 (pl)
AT (1) ATE498685T1 (pl)
CY (2) CY1111447T1 (pl)
DE (1) DE602005026386D1 (pl)
DK (2) DK1766010T3 (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 (190)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6784291B2 (en) * 2000-05-04 2004-08-31 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
ES2351976T3 (es) 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
EP1713332A4 (en) 2004-01-23 2010-08-18 Avi Biopharma Inc ANTISENSE OLIGOMERS AND METHOD FOR INDUCTION OF IMMUNOTHERAPY AND IMMUNOSUPPRESSION
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
SI2206781T1 (sl) 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
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
CA2664189C (en) * 2006-09-21 2017-11-21 University Of Rochester Compositions and methods related to protein displacement therapy for myotonic dystrophy
AU2008271050B2 (en) 2007-06-29 2014-11-06 Sarepta Therapeutics, 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
WO2009008725A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
JP5706157B2 (ja) 2007-07-12 2015-04-22 プロセンサ テクノロジーズ ビー．ブイ．Ｐｒｏｓｅｎｓａ Ｔｅｃｈｎｏｌｏｇｉｅｓ Ｂ．Ｖ． 化合物を種々の選択臓器又は組織に標的化するための分子
DK2203173T3 (en) 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
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
EP2249874A1 (en) 2008-02-08 2010-11-17 ProSensa Holding BV Methods and means for treating dna repeat instability associated genetic 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
EP2762567B1 (en) * 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
CA2741629C (en) * 2008-10-27 2022-07-05 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
CN102625840A (zh) 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
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)
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
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
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
SMT201800579T1 (it) * 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
CA2784783C (en) 2009-12-18 2021-07-20 Novartis Ag Organic compositions to treat hsf1-related diseases
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
WO2011078672A1 (en) 2009-12-24 2011-06-30 Prosensa Technologies B.V. 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
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
IT1400425B1 (it) * 2010-06-08 2013-05-31 Amsterdam Molecular Therapeutics Bv Modified snrnas for use in therapy.
DK2601294T3 (en) * 2010-08-05 2019-02-18 Academisch Ziekenhuis Leiden ANTISENSE OLIGONUCLEOTIDE CALCULATED FOR THE REMOVAL OF PROTEOLYTIC DIVISION PLACES FROM PROTEINS
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
EP2426203B1 (en) 2010-09-02 2016-07-13 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
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
EP2672977A1 (en) * 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
WO2012150960A1 (en) 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
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
EP3279324B1 (en) 2011-10-11 2021-02-17 Institut National De La Sante Et De La Recherche Medicale (Inserm) Exon skipping therapy for dystrophic epidermolysis bullosa
GB201117880D0 (en) * 2011-10-17 2011-11-30 Ucl Business Plc Antisense oligonucleotides
BR112014011875B1 (pt) 2011-11-18 2022-01-04 Sarepta Therapeutics, Inc Oligonucleotídeos funcionalmente modificados e subunidades dos mesmos
CN108611349A (zh) * 2011-12-28 2018-10-02 日本新药株式会社 反义核酸
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
CA2868174A1 (en) 2012-03-20 2013-09-26 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
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
AU2013285698A1 (en) * 2012-07-03 2015-02-19 Biomarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
EP2880053B8 (en) 2012-08-01 2020-04-15 Ikaika Therapeutics, LLC Mitigating tissue damage and fibrosis via anti-ltbp4 antibody
KR20150099804A (ko) 2012-12-20 2015-09-01 사렙타 쎄러퓨틱스 인코퍼레이티드 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
KR20230074606A (ko) * 2013-03-14 2023-05-30 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
KR102307280B1 (ko) * 2013-06-05 2021-10-01 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
WO2015051283A1 (en) 2013-10-04 2015-04-09 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
RU2702424C2 (ru) * 2014-03-12 2019-10-08 Ниппон Синяку Ко., Лтд. Антисмысловые нуклеиновые кислоты
WO2015171918A2 (en) * 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
AU2015272127A1 (en) 2014-06-10 2017-01-19 Erasmus University Medical Center Rotterdam Methods for characterizing alternatively or aberrantly spliced mRNA isoforms
WO2015194520A1 (ja) * 2014-06-17 2015-12-23 日本新薬株式会社 アンチセンス核酸
WO2016037161A2 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
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
EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation RNA INTERACTOM 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
SI3283500T1 (sl) * 2015-04-08 2020-12-31 The University Of Chicago Sestavki in postopki za popravilo medenično ramenske distrofije tipa 2C z uporabo preskakovanja eksonov
US10324930B2 (en) * 2015-05-27 2019-06-18 Sap Se Database calculation engine with nested multiprovider merging
WO2016196897A1 (en) 2015-06-04 2016-12-08 Sarepta Therapeutics, Inc. Methods and compounds for treatment of lymphocyte-related diseases and conditions
CN108351350B (zh) 2015-08-25 2022-02-18 杜克大学 使用rna指导型内切核酸酶改善基因组工程特异性的组合物和方法
CN113913426B (zh) 2015-09-15 2024-08-02 日本新药株式会社 反义核酸
JP6875684B2 (ja) * 2015-09-16 2021-05-26 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
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
SG10201912902TA (en) 2015-10-09 2020-02-27 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
US11970710B2 (en) 2015-10-13 2024-04-30 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
JOP20200228A1 (ar) 2015-12-21 2017-06-16 Novartis Ag تركيبات وطرق لخفض تعبير البروتين tau
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
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
SG10202101834QA (en) 2016-05-24 2021-04-29 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
SG11201809497RA (en) 2016-05-24 2018-12-28 Sarepta Therapeutics Inc Processes for preparing phosphorodiamidate morpholino oligomers
MX383656B (es) 2016-05-24 2025-03-14 Sarepta Therapeutics Inc Procesos para preparar oligómeros de morfolino fosforodiamidato.
CN109563114B (zh) 2016-05-24 2022-08-12 萨勒普塔医疗公司 用于制备寡聚物的方法
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
SG11201809494VA (en) 2016-05-24 2018-12-28 Sarepta Therapeutics Inc Pharmaceutical composition comprising eteplirsen
US20190262375A1 (en) 2016-06-30 2019-08-29 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
WO2018009547A1 (en) 2016-07-05 2018-01-11 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing col6-related disorders and methods for treating same
CN109757108A (zh) * 2016-07-05 2019-05-14 比奥马林技术公司 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
JOP20190104A1 (ar) * 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
KR102810425B1 (ko) 2016-12-19 2025-05-21 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
KR102646318B1 (ko) 2016-12-19 2024-03-12 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머 결합체
HRP20240705T1 (hr) 2016-12-19 2024-08-30 Sarepta Therapeutics, Inc. Oligomerni konjugati koji preskaču egzon za mišićnu distrofiju
SG10202107429WA (en) 2017-01-06 2021-08-30 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
US11597927B2 (en) 2017-06-02 2023-03-07 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
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
US11999953B2 (en) * 2017-09-13 2024-06-04 The Children's Medical Center Corporation Compositions and methods for treating 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
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
EP3697910A4 (en) 2017-10-18 2021-07-14 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
BR112020020670A2 (pt) * 2018-04-12 2021-03-02 Wave Life Sciences Ltd. composição de oligonucleotídeo, composição farmacêutica, método para alterar o splicing de uma transcrição alvo, método para tratar distrofia muscular, método para preparar um oligonucleotídeo ou uma composição de oligonucleotídeo do mesmo e oligonucleotídeo
EP3784248A4 (en) * 2018-04-26 2022-08-10 Sarepta Therapeutics, Inc. EXON-SKIPPING OLIGOMERS AND OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
TWI844541B (zh) 2018-05-11 2024-06-11 新加坡商波濤生命科學有限公司 寡核苷酸組成物及其使用方法
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2019241385A2 (en) * 2018-06-13 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystropy
WO2019241470A2 (en) * 2018-06-14 2019-12-19 Sarepta Therapeutics, Inc. Exon skipping oligomers and oligomer conjugates for muscular dystrophy
CN112399849A (zh) 2018-06-26 2021-02-23 日本新药株式会社 含有反义寡核苷酸的组合物及其在迪谢内肌营养不良症的治疗中的用途
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
AU2019316103B2 (en) 2018-08-02 2026-02-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
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
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
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
EP3874044A1 (en) * 2018-11-02 2021-09-08 BioMarin Technologies B.V. Bispecific antisense oligonucleotides for dystrophin exon skipping
EP3890783A1 (en) 2018-12-07 2021-10-13 Oxford University Innovation Limited Linkers
CA3123045A1 (en) 2018-12-23 2020-07-02 Csl Behring L.L.C. Donor t-cells with kill switch
BR112021012240A2 (pt) 2018-12-23 2022-01-18 Csl Behring Llc Terapia de gene-célula-tronco hematopoiética para síndrome wiskott-aldrich
EP3918069A1 (en) 2019-01-31 2021-12-08 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
EP3959319A4 (en) 2019-04-25 2023-06-07 Avidity Biosciences, Inc. NUCLEIC ACID COMPOSITIONS AND METHODS FOR MULTI-EXON SKIPPING
SG11202112499VA (en) 2019-05-15 2021-12-30 Univ Miami Treatment of heart disease by disruption of the anchoring of pp2a
WO2020243261A1 (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
MX2022007491A (es) 2019-12-19 2022-08-15 Nippon Shinyaku Co Ltd Acido nucleico antisentido que permite la omision de exones.
AU2020411964A1 (en) 2019-12-26 2022-06-16 National Center Of Neurology And Psychiatry 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
AU2021237465A1 (en) 2020-03-19 2022-10-13 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
WO2021263070A1 (en) 2020-06-26 2021-12-30 Csl Behring Llc Donor t-cells with kill switch
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
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
EP4326752A1 (en) * 2021-04-23 2024-02-28 Research Institute at Nationwide Children's Hospital Products and methods for treating muscular dystrophy
AU2022298028A1 (en) 2021-06-23 2023-12-21 National Center Of Neurology And Psychiatry Combination of antisense oligomers
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
JP2024525608A (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 ジストロフィン異常症を処置するための筋標的化複合体および製剤
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US20240390508A1 (en) 2021-08-21 2024-11-28 Takeda Pharmaceutical Company Limited Human transferrin receptor binding peptide-drug conjugate
CN118265544A (zh) 2021-09-16 2024-06-28 艾维迪提生物科学公司 治疗面肩肱型肌营养不良的组合物和方法
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
AU2022424485A1 (en) 2021-12-27 2024-07-11 Nippon Shinyaku Co., Ltd. Method for producing oligonucleic acid compound
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
WO2023172624A1 (en) 2022-03-09 2023-09-14 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
WO2024209267A2 (en) 2023-04-03 2024-10-10 Jazz Pharmaceuticals Ireland Limited Modulators of nanoparticle uptake
WO2024227040A1 (en) 2023-04-27 2024-10-31 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
EP4671372A3 (en) 2024-01-29 2026-03-11 Arnatar Therapeutics, Inc Translation enhancing nucleic acid compounds: aso coupled translation - upregulation 1 (act-up1) and uses thereof

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
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
AU5661386A (en) 1985-03-15 1986-10-13 Stirchak, E. Stereoregular polynucleotide-binding polymers
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group 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
EP1018649A3 (en) 1992-03-31 2000-11-29 Abbott Laboratories Immunochromatographic device and method for multiplex detection of multiple analytes
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
WO1994002595A1 (en) 1992-07-17 1994-02-03 Ribozyme Pharmaceuticals, Inc. Method and reagent for treatment of 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
DE69435005T2 (de) 1993-05-11 2008-04-17 The University Of North Carolina At Chapel Hill 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
BR9707529A (pt) 1996-02-14 2000-01-04 Isis Pharmaceuticals Inc Oligunucleotìdeo especificamente hibridizável com dna ou rna.
AU2585197A (en) 1996-03-20 1997-10-10 Regents Of The University Of California, The 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
DE69905938T2 (de) 1998-11-13 2003-11-13 Eli Lilly And Co., Indianapolis Kombination von duloxetin mit nichtsteroid-entzündungshemmern für die behandlung von schmerz
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
AU2281201A (en) 1999-12-29 2001-07-09 A. James Mixson Histidine copolymer and methods for using same
KR20020079768A (ko) 2000-01-04 2002-10-19 에이브이아이 바이오파마 인코포레이티드 안티센스 항박테리아 세포분열 조성물 및 방법
JP2004509604A (ja) 2000-03-28 2004-04-02 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 ｍＲＮＡプロセッシングのアンチセンスモジュレーションによる、細胞行動の改変
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
JP2003531915A (ja) 2000-05-01 2003-10-28 ハイブリドン・インコーポレイテッド ヌクレオシドの位置的修飾によるオリゴヌクレオチドＣｐＧ誘導性免疫刺激の調節
US6784291B2 (en) 2000-05-04 2004-08-31 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
DE60119173T2 (de) 2000-08-30 2007-01-18 Avi Biopharma, Inc., Corvallis Methode zur analyse von oligonukleotidanaloga
US20070037165A1 (en) 2000-09-08 2007-02-15 Applera Corporation Polymorphisms in known genes associated with human 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
WO2002024717A1 (en) 2000-09-20 2002-03-28 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
US8090542B2 (en) 2002-11-14 2012-01-03 Dharmacon Inc. Functional 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
ES2351976T3 (es) 2003-04-29 2011-02-14 Avi Biopharma, Inc. Composiciones para mejorar el transporte y la eficacia antisentido de análogos de ácidos nucleicos en células.
DK1495769T3 (da) 2003-07-11 2008-06-23 Lbr Medbiotech B V Mannose-6-phosphat-receptormedieret gentransfer til muskelceller
WO2005030800A2 (en) 2003-08-05 2005-04-07 Avi Biopharma, Inc. Oligonucleotide analog and method for treating flavivirus infections
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
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
SI2206781T1 (sl) 2004-06-28 2016-05-31 The University Of Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksona in postopki njihove uporabe
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
ES2852549T3 (es) 2005-02-09 2021-09-13 Sarepta Therapeutics Inc Composición antisentido para tratamiento de la atrofia muscular
CA2605512A1 (en) 2005-04-22 2006-10-26 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secondary 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
US20070105807A1 (en) 2005-11-10 2007-05-10 Sazani Peter L Splice switch 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
ES2657400T3 (es) 2006-05-10 2018-03-05 Sarepta Therapeutics, Inc. Análogos de oligonucleótidos que tienen enlaces intersubunitarios catiónicos
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
AU2008271050B2 (en) 2007-06-29 2014-11-06 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
DK2203173T3 (en) 2007-10-26 2016-02-29 Academisch Ziekenhuis Leiden Materials and methods for prevention of muscle diseases
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
EP2762567B1 (en) 2008-10-24 2016-07-13 Sarepta Therapeutics, Inc. Multiple exon skipping compositions for DMD
CA2741629C (en) 2008-10-27 2022-07-05 Academisch Ziekenhuis Leiden Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna
US8592386B2 (en) 2008-12-17 2013-11-26 Sarepta Therapeutics, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
CN102625840A (zh) 2009-04-10 2012-08-01 肌肉学研究协会 用于治疗疾病的三环dna反义寡核苷酸、组合物和方法
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la 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
US9034838B2 (en) 2009-05-25 2015-05-19 Universita Degli Studi Di Roma “La Sapienza” miR-31 in duchenne muscular dystrophy therapy
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)
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.
SMT201800579T1 (it) 2009-11-12 2019-01-11 Univ Western Australia Molecole antisenso e metodi per trattare patologie
CA2779830C (en) 2009-11-13 2020-07-21 Avi Biopharma, 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
JP2013530154A (ja) 2010-05-28 2013-07-25 サレプタ セラピューティクス， インコーポレイテッド 修飾されたサブユニット間結合および／または末端基を有するオリゴヌクレオチドアナログ
EP2586462A4 (en) 2010-06-28 2013-12-11 Masatoshi Hagiwara PROPHYLACTIC OR ALTERNATIVE 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
PL2623507T3 (pl) 2010-09-30 2017-03-31 Nippon Shinyaku Co., Ltd. Pochodna kwasu morfolinonukleinowego
EP2672977A1 (en) 2011-02-08 2013-12-18 The Charlotte-Mecklenburg Hospital Authority d/b/a Carolina Healthcare System Antisense oligonucleotides
JP2012226016A (ja) 2011-04-15 2012-11-15 Toyota Industries Corp センサ用光ファイバ
WO2012150960A1 (en) 2011-05-05 2012-11-08 Avi Biopharma, Inc. Peptide oligonucleotide conjugates
US9161948B2 (en) 2011-05-05 2015-10-20 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
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
CN108611349A (zh) 2011-12-28 2018-10-02 日本新药株式会社 反义核酸
ES2907250T3 (es) 2012-01-27 2022-04-22 Biomarin Tech Bv Oligonucleótidos de modulación de ARN con características mejoradas para el tratamiento de la distrofia muscular de Duchenne y de Becker
DE102012203443A1 (de) 2012-03-05 2013-09-05 Robert Bosch Gmbh Verfahren zum Bilden eines Kontakts auf einem Halbleitersubstrat und Halbleitervorrichtung
CA2868174A1 (en) 2012-03-20 2013-09-26 Sarepta Therapeutics, Inc. Boronic acid conjugates of oligonucleotide analogues
AU2013285698A1 (en) 2012-07-03 2015-02-19 Biomarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
KR20150099804A (ko) 2012-12-20 2015-09-01 사렙타 쎄러퓨틱스 인코퍼레이티드 근위축증을 치료하기 위한 개선된 엑손 스키핑 조성물
KR20230074606A (ko) 2013-03-14 2023-05-30 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이영양증의 치료를 위한 엑손 스키핑 조성물
AU2014236140B2 (en) 2013-03-14 2019-10-03 Sarepta Therapeutics, Inc. Exon skipping compositions for treating muscular dystrophy
NZ732507A (en) 2013-03-15 2018-08-31 Sarepta Therapeutics Inc Improved compositions for treating muscular dystrophy
WO2014172660A1 (en) 2013-04-18 2014-10-23 Brigham Young University Porous material for thermal and/or electrical isolation and methods of manufacture
EP2986632B1 (en) 2013-04-20 2018-09-05 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
WO2017059131A1 (en) 2015-09-30 2017-04-06 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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