ATE498685T1 - Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon - Google Patents

Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon

Info

Publication number
ATE498685T1
ATE498685T1 AT05754344T AT05754344T ATE498685T1 AT E498685 T1 ATE498685 T1 AT E498685T1 AT 05754344 T AT05754344 T AT 05754344T AT 05754344 T AT05754344 T AT 05754344T AT E498685 T1 ATE498685 T1 AT E498685T1
Authority
AT
Austria
Prior art keywords
exon skipping
antisense oligonucleotides
inducing exon
inducing
seq
Prior art date
Application number
AT05754344T
Other languages
English (en)
Inventor
Stephen 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.)
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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=ATE498685(T1) "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
Application granted granted Critical
Publication of ATE498685T1 publication Critical patent/ATE498685T1/de

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    • C—CHEMISTRY; METALLURGY
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    • 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
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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
    • 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
    • C12N2310/30—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)
AT05754344T 2004-06-28 2005-06-28 Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon ATE498685T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
AU2004903474A AU2004903474A0 (en) 2004-06-28 Antisense Oligonucleotides for Inducing Exon Skipping and Methods of Use Thereof
PCT/AU2005/000943 WO2006000057A1 (en) 2004-06-28 2005-06-28 Antisense oligonucleotides for inducing exon skipping and methods of use thereof

Publications (1)

Publication Number Publication Date
ATE498685T1 true ATE498685T1 (de) 2011-03-15

Family

ID=35781530

Family Applications (1)

Application Number Title Priority Date Filing Date
AT05754344T ATE498685T1 (de) 2004-06-28 2005-06-28 Antisense-oligonukleotide zur induktion von exon- skipping sowie verfahren zur verwendung davon

Country Status (14)

Country Link
US (47) USRE47769E1 (de)
EP (9) EP2206781B1 (de)
AT (1) ATE498685T1 (de)
CY (2) CY1111447T1 (de)
DE (1) DE602005026386D1 (de)
DK (2) DK1766010T3 (de)
ES (3) ES2361325T3 (de)
HK (2) HK1216545A1 (de)
HR (2) HRP20110352T1 (de)
HU (1) HUE028632T2 (de)
PL (2) PL2206781T3 (de)
PT (1) PT1766010E (de)
SI (2) SI1766010T1 (de)
WO (1) WO2006000057A1 (de)

Families Citing this family (172)

* 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 (de) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induktion von "exon-skipping" in eukaryotischen Zellen
WO2004083432A1 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
WO2004097017A2 (en) 2003-04-29 2004-11-11 Avi Biopharma, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
CA2553104A1 (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
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
EP2206781B1 (de) 2004-06-28 2015-12-02 The University Of Western Australia Antisense-Oligonukleotide zur Induktion von Exon-Skipping sowie Verfahren zur Verwendung davon
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 (de) * 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Mittel und Verfahren zur Induktion von Exon-skipping
NZ574807A (en) 2006-08-11 2011-01-28 Prosensa Technologies Bv Methods and means for treating dna repeat instability associated genetic disorders
CA2981308C (en) * 2006-09-21 2020-12-22 University Of Rochester Compositions and methods related to protein displacement therapy for myotonic dystrophy
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
EP2170363B1 (de) 2007-06-29 2018-08-08 Sarepta Therapeutics, Inc. Gewebespezifische peptid-konjugate und verfahren
EP2167135A2 (de) 2007-07-12 2010-03-31 Prosensa Technologies B.V. Moleküle für targeting von verbindungen auf verschiedene ausgewählte organe, gewebe oder tumorzellen
CN103212085A (zh) 2007-07-12 2013-07-24 普罗森那技术公司 用于使化合物靶向多种选定器官或组织的分子
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
US7989608B2 (en) * 2007-12-28 2011-08-02 Avi Biopharma Inc. Immunomodulatory agents and methods of use
WO2009099326A1 (en) 2008-02-08 2009-08-13 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 (de) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
SI3133160T1 (sl) * 2008-10-24 2019-05-31 Sarepta Therapeutics, Inc. Sestavki, ki preskakujejo ekson za DMD
DK2607484T3 (en) * 2008-10-27 2016-03-07 Biomarin Technologies B V Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-MRNA
CA2744987C (en) 2008-12-02 2018-01-16 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
ES2593836T3 (es) * 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
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
AU2010270714B2 (en) 2009-07-06 2015-08-13 Wave Life Sciences Ltd. Novel nucleic acid prodrugs and methods use thereof
US20120172415A1 (en) * 2009-08-31 2012-07-05 Thomas Voit Exon Skipping Therapy for Functional Amelioration of Semifunctional 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
ES2693459T3 (es) * 2009-11-12 2018-12-11 The University Of Western Australia Moléculas antisentido y métodos para el tratamiento de patologías
AU2014280918B2 (en) * 2009-12-18 2016-11-17 Arrowhead Pharmaceuticals, Inc. Organic compositions to treat HSF1-related diseases
KR101605932B1 (ko) 2009-12-18 2016-03-24 노파르티스 아게 Ｈｓｆ1-관련 질환을 치료하기 위한 유기 조성물
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
TWI620756B (zh) 2010-05-28 2018-04-11 薩羅塔治療公司 具有經修飾之單元間連結及／或末端基團之寡核苷酸類似物
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 (de) 2010-09-02 2017-01-18 Université de Mons Zur behandlung von fazioskapulohumeraler muskeldystrophie nützliche wirkstoffe
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
EP2620428B1 (de) 2010-09-24 2019-05-22 Wave Life Sciences Ltd. Asymmetrische hilfsgruppe
EP3702460A1 (de) 2010-11-12 2020-09-02 The General Hospital Corporation Polycombassoziierte nichtcodierende rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012109296A1 (en) * 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
KR102339196B1 (ko) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
SG10201700554VA (en) 2011-07-19 2017-03-30 Wave Life Sciences Pte Ltd Methods for the synthesis of functionalized nucleic acids
EP2750715B1 (de) 2011-08-30 2018-10-31 The Regents of The University of California Identifikation von kleinen molekülen zur verstärkung eines therapeutischen exon-skippings
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
ES2670495T3 (es) * 2011-10-11 2018-05-30 Inserm - Institut National De La Santé Et De La Recherche Médicale Tratamiento de salto de exones para epidermólisis ampollosa distrófica
GB201117880D0 (en) * 2011-10-17 2011-11-30 Ucl Business Plc Antisense oligonucleotides
CA2854907C (en) 2011-11-18 2020-03-10 Sarepta Therapeutics, Inc. Functionally-modified oligonucleotides and subunits thereof
EP2799548B1 (de) * 2011-12-28 2019-08-21 Nippon Shinyaku Co., Ltd. Antisense-nucleinsäure
EP4043039A1 (de) * 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna-modulierende oligonukleotide mit verbesserten eigenschaften zur behandlung der duchenne- und becker-muskeldystrophie
KR102079284B1 (ko) 2012-03-20 2020-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 올리고뉴클레오티드 유사체의 보론산 접합체
DE102012103041A1 (de) 2012-04-10 2013-10-10 Eberhard-Karls-Universität Tübingen Universitätsklinikum Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9372901B2 (en) * 2012-04-27 2016-06-21 Quixey, Inc. Searching for software applications based on application attributes
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
EP2850190B1 (de) 2012-05-16 2020-07-08 Translate Bio MA, Inc. Zusammensetzungen und verfahren zur modulation der mecp2-expression
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CA2877644A1 (en) * 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
WO2014012081A2 (en) 2012-07-13 2014-01-16 Ontorii, Inc. Chiral control
SI2880053T1 (sl) 2012-08-01 2020-08-31 Ikaika Therapeutics, Llc Blažitev poškodbe tkiva in fibroze s protitelesom proti-LTBP4
EP3885439A1 (de) 2012-12-20 2021-09-29 Sarepta Therapeutics, Inc. Verbesserte exon-skipping-zusammensetzungen zur behandlung von muskeldystrophie
EP3633035A1 (de) 2013-03-14 2020-04-08 Sarepta Therapeutics, Inc. Verbesserte exon-skipping-zusammensetzungen zur behandlung von muskeldystrophie
MX366274B (es) 2013-03-14 2019-07-04 Sarepta Therapeutics Inc Composiciones para el salto del exón para el tratamiento de distrofia muscular.
BR112015022998A2 (pt) * 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
EP3004370B1 (de) * 2013-06-05 2024-08-21 Duke University Rna-geführte genmanipulation und genregulierung
US10174328B2 (en) 2013-10-04 2019-01-08 Translate Bio Ma, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
US10149905B2 (en) 2014-01-15 2018-12-11 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
EP3095461A4 (de) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chirales nukleinsäure-adjuvans mit immunitätsinduktionswirkung und immunitätsinduktionsaktivator
MX2016009290A (es) 2014-01-16 2017-02-28 Wave Life Sciences Ltd Diseño quiral.
TR201901939T4 (tr) * 2014-03-12 2019-03-21 Nat Center Neurology & Psychiatry Antisens nükleik asit.
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
MX2016016526A (es) * 2014-06-17 2017-04-04 Nippon Shinyaku Co Ltd Acidos nucleicos antisentido.
MX2017002931A (es) 2014-09-07 2017-05-30 Selecta Biosciences Inc Metodos y composiciones para atenuar respuestas inmunes anti-vector de transferencia viral.
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
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
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The 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
ES2846902T3 (es) 2015-04-08 2021-07-30 Univ Chicago Composiciones y procedimientos para corregir la distrofia muscular de cinturas tipo 2C mediante omisión de exones
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
EP3341727B1 (de) 2015-08-25 2022-08-10 Duke University Zusammensetzungen und verfahren zur verbesserung der spezifität von genomischem engineering mit rna-geführten endonukleasen
WO2017047707A1 (ja) 2015-09-15 2017-03-23 日本新薬株式会社 アンチセンス核酸
US10563199B2 (en) * 2015-09-16 2020-02-18 Nippon Shinyaku Co., Ltd. Antisense nucleic acid for treating amyotrophy
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
CN108699555A (zh) 2015-10-09 2018-10-23 萨勒普塔医疗公司 用于治疗杜兴肌营养不良和相关病症的组合物和方法
AU2016334232B2 (en) * 2015-10-09 2022-05-26 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
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
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
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
KR20190009343A (ko) 2016-05-24 2019-01-28 사렙타 쎄러퓨틱스 인코퍼레이티드 에테플리센을 포함하는 약제학적 조성물
FI3464306T3 (fi) 2016-05-24 2024-05-16 Sarepta Therapeutics Inc Menetelmiä fosforodiamidaattimorfolino-oligomeerien valmistamiseksi
SG10202101830WA (en) 2016-05-24 2021-04-29 Sarepta Therapeutics Inc Processes for preparing oligomers
MA45362A (fr) 2016-05-24 2019-04-10 Sarepta Therapeutics Inc Procédés de préparation d'oligomères morpholino de phosphorodiamidate
US11472824B2 (en) 2016-05-24 2022-10-18 Sarepta Therapeutics, Inc. Processes for preparing phosphorodiamidate morpholino oligomers
WO2018005805A1 (en) 2016-06-30 2018-01-04 Sarepta Therapeutics, Inc. Exon skipping oligomers for muscular dystrophy
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
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
JOP20190104A1 (ar) * 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
PL3554552T3 (pl) 2016-12-19 2022-11-21 Sarepta Therapeutics, Inc. Koniugaty oligomerów do pomijania egzonów dla dystrofii mięśniowej
MX2019006989A (es) 2016-12-19 2019-08-16 Sarepta Therapeutics Inc Conjugados de oligomeros de omision de exon para distrofia muscular.
DK3554553T3 (da) 2016-12-19 2022-09-19 Sarepta Therapeutics Inc Exon-overspringnings-oligomerkonjugat til muskeldystrofi
SG11201906200WA (en) 2017-01-06 2019-08-27 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
JP7557941B2 (ja) 2017-06-02 2024-09-30 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその使用方法
WO2018223056A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US10961537B2 (en) 2017-07-18 2021-03-30 Csl Behring Gene Therapy, 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
US11180541B2 (en) * 2017-09-28 2021-11-23 Geltor, Inc. Recombinant collagen and elastin molecules and uses thereof
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
WO2019075360A1 (en) 2017-10-13 2019-04-18 Selecta Biosciences, Inc. METHODS AND COMPOSITIONS FOR MITIGATING ANTI-VECTOR VIRAL TRANSFER IGM RESPONSES
JP7394753B2 (ja) 2017-10-18 2023-12-08 サレプタ セラピューティクス， インコーポレイテッド アンチセンスオリゴマー化合物
AU2018378812A1 (en) 2017-12-06 2020-07-09 Avidity Biosciences, Inc. Compositions and methods of treating muscle atrophy and myotonic dystrophy
AU2019252680A1 (en) * 2018-04-12 2020-10-22 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
JP2021521794A (ja) * 2018-04-26 2021-08-30 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
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
JP2021526807A (ja) * 2018-06-14 2021-10-11 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーに対するエクソンスキッピングオリゴマーおよびオリゴマーコンジュゲート
US20210261963A1 (en) 2018-06-26 2021-08-26 Nippon Shinyaku Co., Ltd. Composition comprising antisense oligonucleotide and use thereof for treatment of duchenne muscular dystrophy
EP3830259A4 (de) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. Auf muskel abzielende komplexe und ihre verwendungen zur behandlung von fazioskapulohumeraler muskeldystrophie
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3829595A4 (de) 2018-08-02 2022-08-24 Dyne Therapeutics, Inc. Auf muskeln gerichtete komplexe und verwendungen davon zur behandlung von dystrophinopathien
WO2020046985A1 (en) * 2018-08-27 2020-03-05 North Carolina State University Targeting kit with splice switching oligonucleotides to induce apoptosis of mast cells
EP3874044A1 (de) * 2018-11-02 2021-09-08 BioMarin Technologies B.V. Bispezifische antisense-oligonukleotide für dystrophin-exon-skipping
JP2022514955A (ja) 2018-12-23 2022-02-16 シーエスエル・ベーリング・エルエルシー ウィスコット・アルドリッチ症候群の造血幹細胞遺伝子治療
EP3897831A1 (de) 2018-12-23 2021-10-27 CSL Behring LLC Spender-t-zellen mit 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.
TW202104254A (zh) 2019-04-12 2021-02-01 美商格爾托公司 重組彈性蛋白及其生產
AU2020276156A1 (en) 2019-05-15 2021-12-16 The Board Of Trustees Of The Leland Stanford Junior University 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
AU2020378207A1 (en) * 2019-11-08 2022-06-23 North Carolina State University Exon skipping of Fc-epsilon-RI-beta and MS4A6A in combination for the treatment of allergic diseases
AU2020408773A1 (en) 2019-12-19 2022-07-14 National Center Of Neurology And Psychiatry Antisense nucleic acid enabling exon skipping
CA3165961A1 (en) 2019-12-26 2021-07-01 Nippon Shinyaku Co., Ltd. Antisense nucleic acid that induces skipping of exon 50
US20230140736A1 (en) 2020-02-28 2023-05-04 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
CA3172111A1 (en) 2020-03-19 2021-09-23 Barbora MALECOVA 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
AU2021294317A1 (en) 2020-06-26 2023-02-23 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
TW202307208A (zh) 2021-06-23 2023-02-16 日商日本新藥股份有限公司 反義寡聚物之組合
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
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
AU2022345098A1 (en) 2021-09-16 2024-04-04 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
WO2023127918A1 (ja) 2021-12-27 2023-07-06 日本新薬株式会社 オリゴ核酸化合物の製造方法
EP4215614A1 (de) 2022-01-24 2023-07-26 Dynacure Kombinationstherapie für dystrophin-verwandte krankheiten
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
WO2024209267A2 (en) 2023-04-03 2024-10-10 Jazz Pharmaceuticals Ireland Limited Modulators of nanoparticle uptake
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

Family Cites Families (131)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5138722B1 (de) 1970-12-30 1976-10-23
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5190931A (en) 1983-10-20 1993-03-02 The Research Foundation Of State University Of New York Regulation of gene expression by employing translational inhibition of MRNA utilizing interfering complementary MRNA
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
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
US5217866A (en) 1985-03-15 1993-06-08 Anti-Gene Development Group Polynucleotide assay reagent and method
ATE171185T1 (de) 1985-03-15 1998-10-15 Antivirals Inc Immunotestmittel für polynukleotid und verfahren
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
JP2652732B2 (ja) 1991-09-30 1997-09-10 三光総業株式会社 ブロー成型法
EP0633944B1 (de) * 1992-03-31 2000-11-08 Abbott Laboratories Verfahren zur mehrfachen ligase-kettenreaktion
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
AU4769893A (en) 1992-07-17 1994-02-14 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
PT698092E (pt) 1993-05-11 2007-10-29 Univ North Carolina Oligonucleótidos complementares de uma cadeia codificadora que combatem splicing aberrante e métodos para a sua utilização
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
US5820873A (en) 1994-09-30 1998-10-13 The University Of British Columbia Polyethylene glycol modified ceramide lipids and liposome uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
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
EP1135121B1 (de) 1998-11-13 2003-03-12 Eli Lilly And Company Kombination von duloxetin mit nichtsteroid-entzündungshemmern für die behandlung von schmerz
JP2002535015A (ja) 1999-01-29 2002-10-22 エイブイアイ バイオファーマ， インコーポレイテッド 標的ｒｎａを検出するための非侵襲性方法
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
NZ515964A (en) 1999-06-21 2004-03-26 Murdoch Childrens Res Inst A method for the prophylaxis and/or treatment of medical disorders
US7163695B2 (en) 1999-12-29 2007-01-16 Mixson A James Histidine copolymer and methods for using same
US7070807B2 (en) 1999-12-29 2006-07-04 Mixson A James Branched histidine copolymers and methods for using same
WO2001049775A2 (en) 2000-01-04 2001-07-12 Avi Biopharma, Inc. Antisense antibacterial cell division composition and method
AU2000240366B2 (en) 2000-03-28 2005-08-04 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
EP1287125B1 (de) 2000-04-28 2009-07-29 Asklepios Biopharmaceutical, Inc. Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung
AU5736601A (en) 2000-05-01 2001-11-12 Hybridon Inc Modulation of oligonucleotide cpg-mediated immune stimulation by positional modification of nucleosides
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
ES2263651T3 (es) 2000-08-30 2006-12-16 Avi Biopharma, Inc. Metodo para analidis de analogos de oligonucleotidos.
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
JP2004509619A (ja) 2000-09-20 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 Ｆｌｉｐ−ｃ発現のアンチセンスモジュレーション
EP1191097A1 (de) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induktion von "exon-skipping" in eukaryotischen Zellen
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
DK2284266T3 (da) 2002-11-14 2014-01-13 Thermo Fisher Scient Biosciences Inc sIRNA-MOLEKYLE MOD TP53
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
JP2006507780A (ja) 2002-11-25 2006-03-02 テレセクター リソーシーズ グループ インコーポレイテッド 装置への呼出しの通知のための方法及び装置
ES2566632T3 (es) 2002-11-25 2016-04-14 Masafumi Matsuo Fármacos de ácido nucleico ENA que modifican el corte y empalme en precursores de ARNm
WO2004083432A1 (en) 2003-03-21 2004-09-30 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
WO2004097017A2 (en) 2003-04-29 2004-11-11 Avi Biopharma, 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.
AU2004276226B2 (en) 2003-08-05 2009-07-30 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
CA2538729A1 (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
EP2206781B1 (de) 2004-06-28 2015-12-02 The University Of Western Australia Antisense-Oligonukleotide zur Induktion von Exon-Skipping sowie Verfahren zur Verwendung davon
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
WO2006112705A2 (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
EP1937312B1 (de) 2005-08-30 2016-06-29 Ionis Pharmaceuticals, Inc. Chimere oligomere verbindungen zur modulation von splicing
US8524676B2 (en) 2005-09-08 2013-09-03 Sarepta Therapeutics, Inc. Method for treating enterovirus or rhinovirus infection using antisense antiviral compounds
US8501704B2 (en) 2005-11-08 2013-08-06 Sarepta Therapeutics, Inc. Immunosuppression compound and treatment method
CA2629323A1 (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
LT2024499T (lt) 2006-05-10 2018-02-26 Sarepta Therapeutics, Inc. Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų
US8785407B2 (en) 2006-05-10 2014-07-22 Sarepta Therapeutics, Inc. Antisense antiviral agent and method for treating ssRNA viral infection
US20070265215A1 (en) 2006-05-11 2007-11-15 Iversen Patrick L Antisense restenosis composition and method
EP1857548A1 (de) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Mittel und Verfahren zur Induktion von Exon-skipping
EP2170363B1 (de) 2007-06-29 2018-08-08 Sarepta Therapeutics, Inc. Gewebespezifische peptid-konjugate und verfahren
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
WO2009101399A1 (en) 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)
EP2119783A1 (de) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
SI3133160T1 (sl) 2008-10-24 2019-05-31 Sarepta Therapeutics, Inc. Sestavki, ki preskakujejo ekson za DMD
DK2607484T3 (en) 2008-10-27 2016-03-07 Biomarin Technologies B V Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-MRNA
EP2376633A1 (de) 2008-12-17 2011-10-19 AVI BioPharma, Inc. Antisense-zusammensetzungen und verfahren zur modulation von kontaktüberempfindlichkeit oder kontaktdermatitis
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
ES2593836T3 (es) 2009-04-24 2016-12-13 Biomarin Technologies B.V. Oligonucleótido que comprende una inosina para tratar la DMD
US9034838B2 (en) 2009-05-25 2015-05-19 Universita Degli Studi Di Roma “La Sapienza” miR-31 in duchenne muscular dystrophy therapy
EP2258863A1 (de) 2009-05-25 2010-12-08 Universita'Degli Studi di Roma "La Sapienza" miRNA biomarkers für die Diagnose und Weiterentwicklung der Duchenne Dystrophie, für die Überwachung therapeutischer Interventionen und als Therapeutik
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)
US20120172415A1 (en) 2009-08-31 2012-07-05 Thomas Voit Exon Skipping Therapy for Functional Amelioration of Semifunctional 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.
ES2693459T3 (es) 2009-11-12 2018-12-11 The University Of Western Australia Moléculas antisentido y métodos para el tratamiento de patologías
JP5991922B2 (ja) 2009-11-13 2016-09-14 サレプタ セラピューティクス， インコーポレイテッド アンチセンス抗ウイルス性化合物およびインフルエンザウイルス感染を処置するための方法
US9050373B2 (en) 2010-05-13 2015-06-09 The Charlotte-Mecklenburg Hospital Authority Pharmaceutical compositions comprising antisense oligonucleotides and methods of using same
TWI620756B (zh) 2010-05-28 2018-04-11 薩羅塔治療公司 具有經修飾之單元間連結及／或末端基團之寡核苷酸類似物
JP5950818B2 (ja) 2010-06-28 2016-07-13 正敏 萩原 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
WO2012109296A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
JP2012226016A (ja) 2011-04-15 2012-11-15 Toyota Industries Corp センサ用光ファイバ
KR102339196B1 (ko) 2011-05-05 2021-12-15 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
US20140080896A1 (en) 2011-08-30 2014-03-20 The Regents Of The University Of California Identification of small molecules that facilitate therapeutic exon skipping
EP2750715B1 (de) 2011-08-30 2018-10-31 The Regents of The University of California Identifikation von kleinen molekülen zur verstärkung eines therapeutischen exon-skippings
ES2535654T3 (es) 2011-10-13 2015-05-13 Association Institut De Myologie ADN triciclo-fosforotioato
EP2799548B1 (de) 2011-12-28 2019-08-21 Nippon Shinyaku Co., Ltd. Antisense-nucleinsäure
EP4043039A1 (de) 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna-modulierende oligonukleotide mit verbesserten eigenschaften zur behandlung der duchenne- und becker-muskeldystrophie
DE102012203443A1 (de) 2012-03-05 2013-09-05 Robert Bosch Gmbh Verfahren zum Bilden eines Kontakts auf einem Halbleitersubstrat und Halbleitervorrichtung
KR102079284B1 (ko) 2012-03-20 2020-02-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 올리고뉴클레오티드 유사체의 보론산 접합체
CA2877644A1 (en) 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
EP3885439A1 (de) 2012-12-20 2021-09-29 Sarepta Therapeutics, Inc. Verbesserte exon-skipping-zusammensetzungen zur behandlung von muskeldystrophie
EP3633035A1 (de) 2013-03-14 2020-04-08 Sarepta Therapeutics, Inc. Verbesserte exon-skipping-zusammensetzungen zur behandlung von muskeldystrophie
MX366274B (es) 2013-03-14 2019-07-04 Sarepta Therapeutics Inc Composiciones para el salto del exón para el tratamiento de distrofia muscular.
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
US20140314998A1 (en) 2013-04-18 2014-10-23 Brigham Young University Porous material for thermal and/or electrical isolation and methods of manufacture
CA2909807C (en) 2013-04-20 2023-08-08 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
BR112018006445A2 (pt) 2015-09-30 2018-12-11 Sarepta Therapeutics Inc métodos para tratar distrofia muscular
US20200195590A1 (en) 2018-12-18 2020-06-18 Zipwhip, Inc. System and method for generating and transmitting automatic reply messages

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