DK2203173T3 - Midler og fremgangsmåder til modvirkning af muskellidelser - Google Patents

Midler og fremgangsmåder til modvirkning af muskellidelser Download PDF

Info

Publication number
DK2203173T3
DK2203173T3 DK08842559.0T DK08842559T DK2203173T3 DK 2203173 T3 DK2203173 T3 DK 2203173T3 DK 08842559 T DK08842559 T DK 08842559T DK 2203173 T3 DK2203173 T3 DK 2203173T3
Authority
DK
Denmark
Prior art keywords
dystrophin
exon
muscle
compound
protein
Prior art date
Application number
DK08842559.0T
Other languages
English (en)
Inventor
Deutekom Judith Christina Theodora Van
Annemieke Aartsma-Rus
Ommen Garrit-Jan Boudewijn Van
Gerardus Johannes Platenburg
Kimpe Josephus Johannes De
Original Assignee
Academisch Ziekenhuis Leiden
Biomarin Technologies B V
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=39045623&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=DK2203173(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Academisch Ziekenhuis Leiden, Biomarin Technologies B V filed Critical Academisch Ziekenhuis Leiden
Application granted granted Critical
Publication of DK2203173T3 publication Critical patent/DK2203173T3/da

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
    • A61K31/52—Purines, e.g. adenine
    • A61K31/522—Purines, e.g. adenine having oxo groups directly attached to the heterocyclic ring, e.g. hypoxanthine, guanine, acyclovir
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/58—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids containing heterocyclic rings, e.g. danazol, stanozolol, pancuronium or digitogenin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/12—Drugs for disorders of the metabolism for electrolyte homeostasis
    • A61P3/14—Drugs for disorders of the metabolism for electrolyte homeostasis for calcium homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00—General protective or antinoxious agents
    • A61P39/06—Free radical scavengers or antioxidants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/313—Phosphorodithioates
    • 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
    • C12N2310/314—Phosphoramidates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Endocrinology (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)

Claims (7)

1. Kombination af: - et antisenseoligonukleotid, omfattende en sekvens, der er komplementær til en del af human dystrofin-præ-mRNA exon 51, og hvilket oligonukleotid er gengivet ved SEQ ID NO:204 og - en tilsætningsforbindelse til reduktion af inflammation, hvor denne forbindelse omfatter en steroid, hvilken kombination er til anvendelse som et medikament til lindring af et eller flere symptom(er) på Duchennes muskeldystrofi hos individet.
2. Kombination til anvendelse ifølge krav 1, hvor fraværet af exonet fra mRNA fremstillet ud fra dystrofin-præ-mRNA'et genererer en kodende region til et funktionelt dystrofinprotein.
3. Kombination til anvendelse ifølge krav 1 eller 2, hvor oligonukleotidet omfatter RNA.
4. Kombination til anvendelse ifølge krav 3, hvor RNA'et indeholder en 2'-0-methyl-modificeret ribose (RNA).
5. Kombination til anvendelse ifølge krav 3 eller 4, hvor oligonukleotidet er et 2'-0-methylphosphorothioat-oligoribonukleotid.
6. Kombination til anvendelse ifølge et hvilket som helst af kravene 1 til 5, hvor oligonukleotidet er en peptidnukleinsyre, låst nukleinsyre, morpho-linophosphorodiamidat eller en hvilken som helst kombination deraf.
7. Farmaceutisk præparat omfattende - en kombination som defineret i et hvilket som helst af kravene 1 til 6 og - en farmaceutisk acceptabel bærer, adjuvans, fortyndingsmiddel og/eller excipiens.
DK08842559.0T 2007-10-26 2008-10-27 Midler og fremgangsmåder til modvirkning af muskellidelser DK2203173T3 (da)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US67007P 2007-10-26 2007-10-26
EP07119351 2007-10-26
PCT/NL2008/050673 WO2009054725A2 (en) 2007-10-26 2008-10-27 Means and methods for counteracting muscle disorders

Publications (1)

Publication Number Publication Date
DK2203173T3 true DK2203173T3 (da) 2016-02-29

Family

ID=39045623

Family Applications (1)

Application Number Title Priority Date Filing Date
DK08842559.0T DK2203173T3 (da) 2007-10-26 2008-10-27 Midler og fremgangsmåder til modvirkning af muskellidelser

Country Status (17)

Country Link
US (18) US9243245B2 (da)
EP (4) EP3238737B1 (da)
JP (6) JP5600064B2 (da)
CN (5) CN101896186A (da)
AU (1) AU2008317566B2 (da)
CA (1) CA2704049A1 (da)
CY (2) CY1117286T1 (da)
DK (1) DK2203173T3 (da)
ES (5) ES2639852T3 (da)
HK (2) HK1185098A1 (da)
HR (1) HRP20160025T1 (da)
HU (2) HUE028662T2 (da)
IL (4) IL205322A (da)
NZ (2) NZ584793A (da)
PL (1) PL2203173T3 (da)
PT (1) PT2203173E (da)
WO (1) WO2009054725A2 (da)

Families Citing this family (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1191097A1 (en) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
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
EP3345895B1 (en) 2003-04-11 2019-12-04 PTC Therapeutics, Inc. 1,2,4-oxadiazole benzoic acid compound and its use for nonsense suppression and the treatment of disease
EP2500430B1 (en) 2004-06-28 2017-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
CA2596506C (en) 2005-02-09 2021-04-06 Avi Biopharma, Inc. Antisense composition and method for treating muscle atrophy
CA2660523C (en) 2006-08-11 2019-03-19 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
EP3238737B1 (en) 2007-10-26 2022-04-06 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
EP2119783A1 (en) * 2008-05-14 2009-11-18 Prosensa Technologies B.V. Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
TR201902952T4 (tr) 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
PT2344637E (pt) * 2008-10-27 2015-03-23 Academisch Ziekenhuis Leiden Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne
JP2012524540A (ja) 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
CN105838714B (zh) 2009-11-12 2020-07-17 西澳大利亚大学 反义分子和治疗疾病的方法
US9611471B2 (en) * 2010-08-05 2017-04-04 Academisch Ziekenhuis Leiden 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
CA2826836A1 (en) * 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
WO2012138223A2 (en) 2011-04-05 2012-10-11 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Compounds and methods for altering activin receptor-like kinase signalling
JP5850519B2 (ja) * 2011-05-09 2016-02-03 ネッパジーン株式会社 モルフォリノ搭載バブルリポソームを有効成分としてなる筋ジストロフィー治療薬
WO2013016595A1 (en) * 2011-07-28 2013-01-31 The Regents Of The University Of California Exonic splicing enhancers and exonic splicing silencers
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CN110079525A (zh) 2011-12-28 2019-08-02 日本新药株式会社 反义核酸
EP4043039A1 (en) * 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
DE102012103041A1 (de) 2012-04-10 2013-10-10 Eberhard-Karls-Universität Tübingen Universitätsklinikum Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden
EP3597741A1 (en) 2012-04-27 2020-01-22 Duke University Genetic correction of mutated genes
WO2014007620A2 (en) * 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
BR122020016865B1 (pt) 2013-03-14 2022-12-27 Sarepta Therapeutics, Inc. Oligonucleotídeo antisenso, composição farmacêutica compreendendo o mesmo e uso da dita composição para o tratamento de distrofia muscular de duchenne (dmd)
BR112015022998A2 (pt) 2013-03-15 2017-11-14 Sarepta Therapeutics Inc composições melhoradas para o tratamento de distrofia muscular
US9849066B2 (en) 2013-04-24 2017-12-26 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
US10704060B2 (en) 2013-06-05 2020-07-07 Duke University RNA-guided gene editing and gene regulation
TWI695717B (zh) 2014-03-06 2020-06-11 美商 Ptc 治療公司 １,２,４-二唑苯甲酸之鹽及醫藥組合物
AU2015227733B2 (en) * 2014-03-12 2019-10-31 National Center Of Neurology And Psychiatry Antisense nucleic acids
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015277924B2 (en) 2014-06-17 2021-02-25 National Center Of Neurology And Psychiatry Antisense nucleic acids
CA2959130A1 (en) * 2014-08-11 2016-02-18 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
SG11201702682PA (en) 2014-10-03 2017-04-27 Cold Spring Harbor Lab Targeted augmentation of nuclear gene output
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
US10370660B2 (en) * 2015-05-21 2019-08-06 Wings Therapeutics, Inc. Antisense oligonucleotides to treat Dystrophic Epidermolysis Bullosa
WO2016198676A1 (en) * 2015-06-10 2016-12-15 Association Institut De Myologie Combined therapy for duchenne muscular dystrophy
EP3325018A4 (en) 2015-07-22 2019-04-24 Duke University HIGH EFFICIENCY SCREENING OF REGULATORY ELEMENT FUNCTION USING EPIGENOUS EDITING TECHNOLOGIES
EP4345454A2 (en) 2015-08-25 2024-04-03 Duke University Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases
HUE050061T2 (hu) 2015-09-15 2020-12-28 Nippon Shinyaku Co Ltd Antiszensz nukleinsav
SG11201802870RA (en) 2015-10-09 2018-05-30 Univ Southampton Modulation of gene expression and screening for deregulated protein expression
IL258230B (en) * 2015-10-09 2022-09-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods thereof
BR112018007066A2 (pt) 2015-10-09 2018-10-23 Sarepta Therapeutics Inc composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados
EP3362571A4 (en) 2015-10-13 2019-07-10 Duke University GENOMIC ENGINEERING WITH TYPE I CRISPRISMS IN EUKARYOTIC CELLS
KR20180081516A (ko) 2015-10-30 2018-07-16 피티씨 테라퓨틱스, 인크. 간질 치료 방법
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
GB2545898B (en) 2015-12-21 2019-10-09 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
GB2574525B (en) * 2015-12-21 2020-09-02 Sutura Therapeutics Ltd Improved drug delivery by conjugating oligonucleotides to stitched/stapled peptides
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MY195439A (en) 2016-04-15 2023-01-21 Res Inst Nationwide Childrens Hospital Adeno-Associated Virus Vector Delivery of B-Sarcoglycan and Microrna-29 and the Treatment of Muscular Dystrophy
MA45290A (fr) * 2016-05-04 2019-03-13 Wave Life Sciences Ltd Procédés et compositions d'agents biologiquement actifs
MX2018016052A (es) * 2016-06-30 2019-05-02 Sarepta Therapeutics Inc Oligomeros de omision de exon para distrofia muscular.
EP3554552B1 (en) 2016-12-19 2022-08-17 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
PL3554553T3 (pl) 2016-12-19 2022-11-07 Sarepta Therapeutics, Inc. Koniugaty oligomerów z pominięciem egzonu w dystrofii mięśniowej
EP4122497B1 (en) 2016-12-19 2024-04-10 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
EP3565577A4 (en) 2017-01-06 2020-10-07 Avidity Biosciences, Inc. NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCTION OF EXON SKIP
CA3056638A1 (en) * 2017-03-17 2018-09-20 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
EP3612546B1 (en) * 2017-04-20 2022-07-13 Synthena AG Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
CN111499615B (zh) 2017-08-04 2024-02-02 斯基霍克疗法公司 用于调节剪接的方法和组合物
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
EP3684376A4 (en) * 2017-09-22 2021-10-20 Avidity Biosciences, Inc. NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND EXON BREAK INDUCTION METHODS
EP3687519A1 (en) 2017-09-28 2020-08-05 Sarepta Therapeutics, Inc. Combination therapies for treating muscular dystrophy
WO2019067975A1 (en) 2017-09-28 2019-04-04 Sarepta Therapeutics, Inc. POLYTHERAPIES FOR TREATING MUSCLE DYSTROPHY
US20210145852A1 (en) 2017-09-28 2021-05-20 Sarepta Therapeutics, Inc. Combination Therapies for Treating Muscular Dystrophy
MA51103A (fr) 2017-12-06 2020-10-14 Avidity Biosciences Inc Compositions et procédés de traitement de l'atrophie musculaire et de la dystrophie myotonique
WO2019164852A1 (en) * 2018-02-20 2019-08-29 Edgewise Therapeutics, Inc. Methods and compositions for treating movement disorders
CN112004928A (zh) * 2018-04-12 2020-11-27 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
US10765760B2 (en) 2018-05-29 2020-09-08 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
TW202020153A (zh) * 2018-07-27 2020-06-01 美商薩羅塔治療公司 用於肌肉萎縮症之外顯子跳躍寡聚物
JP2021532831A (ja) 2018-08-02 2021-12-02 ダイン セラピューティクス, インコーポレーテッドDyne Therapeutics, Inc. 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それらの使用
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
CN109486813B (zh) * 2018-10-10 2022-01-18 广州医科大学附属第二医院 一种用于修复TPP1基因Pre-mRNA异常剪接的U1-snRNA及其应用
SG11202105626TA (en) * 2018-12-06 2021-06-29 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
JP2022521467A (ja) 2019-02-05 2022-04-08 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
CN113677344A (zh) 2019-02-06 2021-11-19 斯基霍克疗法公司 用于调节剪接的方法和组合物
MX2021012428A (es) * 2019-04-10 2022-01-19 Ptc Therapeutics Inc Metodo para el tratamiento de distrofia muscular de duchenne mediada por mutacion sin sentido en pacientes pediatricos.
CN110288555B (zh) * 2019-07-02 2022-08-02 桂林电子科技大学 一种基于改进的胶囊网络的低照度增强方法
KR20220038771A (ko) * 2019-08-02 2022-03-29 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 엑손 44-표적화된 핵산 및 상기 핵산을 포함하는 디스트로핀-기반 근병증 치료용 재조합 아데노-관련 바이러스
CA3151789A1 (en) * 2019-09-19 2021-03-25 Arnay Sciences, Llc Compounds and methods useful for modulating gene splicing
CA3173049A1 (en) 2020-02-28 2021-09-02 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
US11555190B2 (en) 2020-03-19 2023-01-17 Avidity Biosciences, Inc. Compositions and methods of treating Facioscapulohumeral muscular dystrophy
US20230287410A1 (en) 2020-05-11 2023-09-14 Stoke Therapeutics, Inc. Opa1 antisense oligomers for treatment of conditions and diseases
EP4314016A1 (en) 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Cyclic cell penetrating peptides
EP4337264A1 (en) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Compositions and methods for modulating tissue distribution of intracellular therapeutics
EP4088722A1 (en) * 2021-05-12 2022-11-16 Justus-Liebig-Universität Gießen Pharmaceutical compositions, uses thereof and methods for treatment of genetic diseases caused by intronic splice-acceptor site mutations
AU2022298774A1 (en) 2021-06-23 2023-12-14 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
IL309909A (en) * 2021-07-09 2024-03-01 Dyne Therapeutics Inc Muscle targeting complexes and their uses for the treatment of dystrophinopathy
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
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
WO2024069229A2 (en) 2022-08-03 2024-04-04 Sutura Therapeutics Ltd Biologically active compounds

Family Cites Families (220)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE2752581C2 (de) 1977-11-25 1979-12-13 Wolfgang 4044 Kaarst Keil Vorrichtung zur Vollentsalzung von Wasser
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5541308A (en) 1986-11-24 1996-07-30 Gen-Probe Incorporated Nucleic acid probes for detection and/or quantitation of non-viral organisms
DE3834636A1 (de) 1988-10-11 1990-04-19 Max Planck Gesellschaft Verfahren zur analyse von laengenpolymorphismen in dna-bereichen
US5766847A (en) 1988-10-11 1998-06-16 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Process for analyzing length polymorphisms in DNA regions
US6867195B1 (en) 1989-03-21 2005-03-15 Vical Incorporated Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
FR2675803B1 (fr) 1991-04-25 1996-09-06 Genset Sa Oligonucleotides fermes, antisens et sens et leurs applications.
WO1993001286A2 (en) 1991-06-28 1993-01-21 Massachusetts Institute Of Technology Localized oligonucleotide therapy
CA2082411A1 (en) 1991-06-28 1992-12-29 Robert D. Rosenberg Localized oligonucleotide therapy
US6200747B1 (en) 1992-01-28 2001-03-13 North Shore University Hospital Research Corp. Method and kits for detection of fragile X specific, GC-rich DNA sequences
US5869252A (en) 1992-03-31 1999-02-09 Abbott Laboratories Method of multiplex ligase chain reaction
US6172208B1 (en) 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
US5418139A (en) 1993-02-10 1995-05-23 University Of Iowa Research Foundation Method for screening for cardiomyopathy
CA2116280A1 (en) 1993-03-05 1994-09-06 Marcy E. Macdonald Huntingtin dna, protein and uses thereof
WO1994026887A1 (en) 1993-05-11 1994-11-24 The University Of North Carolina At Chapel Hill Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US5695933A (en) 1993-05-28 1997-12-09 Massachusetts Institute Of Technology Direct detection of expanded nucleotide repeats in the human genome
US5741645A (en) 1993-06-29 1998-04-21 Regents Of The University Of Minnesota Gene sequence for spinocerebellar ataxia type 1 and method for diagnosis
US5624803A (en) 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US5627263A (en) 1993-11-24 1997-05-06 La Jolla Cancer Research Foundation Integrin-binding peptides
DE4342605A1 (de) 1993-12-14 1995-06-22 Buna Gmbh Funktionalisierte Olefinhomo- und -copolymere
US5962332A (en) 1994-03-17 1999-10-05 University Of Massachusetts Detection of trinucleotide repeats by in situ hybridization
WO1995030774A1 (en) 1994-05-05 1995-11-16 Beckman Instruments, Inc. Oligonucleotide repeat arrays
US5968909A (en) 1995-08-04 1999-10-19 Hybridon, Inc. Method of modulating gene expression with reduced immunostimulatory response
US5854223A (en) 1995-10-06 1998-12-29 The Trustees Of Columbia University In The City Of New York S-DC28 as an antirestenosis agent after balloon injury
US20070173465A9 (en) 1995-10-11 2007-07-26 Monahan Sean D Expression of zeta negative and zeta positive nucleic acids using a dystrophin gene
US7034009B2 (en) 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US6300060B1 (en) 1995-11-09 2001-10-09 Dana-Farber Cancer Institute, Inc. Method for predicting the risk of prostate cancer morbidity and mortality
KR19990082476A (ko) 1996-02-14 1999-11-25 하우스 한스 루돌프, 긴터 베아트리체 당-변형되어 갭이 형성된 올리고뉴클레오티드
JP3950480B2 (ja) 1996-07-18 2007-08-01 株式会社エスアールエル 脊髄小脳変性症２型の原因遺伝子の検出方法及びそのためのプライマー
AU4725597A (en) 1996-10-30 1998-05-22 Srl Inc. Cdna fragments of gene causative of spinocerebellar ataxia type
US5853995A (en) 1997-01-07 1998-12-29 Research Development Foundation Large scale genotyping of diseases and a diagnostic test for spinocerebellar ataxia type 6
AU6591798A (en) 1997-03-31 1998-10-22 Yale University Nucleic acid catalysts
US20020137890A1 (en) 1997-03-31 2002-09-26 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
AU7265298A (en) 1997-04-29 1998-11-24 Trustees Of Boston University Methods and compositions for targeted dna differential display
US6329501B1 (en) 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
US6280938B1 (en) 1997-08-19 2001-08-28 Regents Of The University Of Minnesota SCA7 gene and method of use
US6514755B1 (en) 1998-08-18 2003-02-04 Regents Of The University Of Minnesota SCA7 gene and methods of use
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6127173A (en) 1997-09-22 2000-10-03 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts with endonuclease activity
US6130207A (en) 1997-11-05 2000-10-10 South Alabama Medical Science Foundation Cell-specific molecule and method for importing DNA into a nucleus
JP3012923B2 (ja) 1998-01-26 2000-02-28 新潟大学長 Ｃａｇリピート病の治療薬
KR100280219B1 (ko) 1998-02-26 2001-04-02 이수빈 삼핵산 반복 서열을 이용한 신경정신 질환의 진단 방법 및 진단 시약
US6322978B1 (en) 1998-04-20 2001-11-27 Joslin Diabetes Center, Inc. Repeat polymorphism in the frataxin gene and uses therefore
AU751480B2 (en) 1998-04-29 2002-08-15 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into ribozymes
WO1999063975A2 (en) 1998-06-10 1999-12-16 Biognostik Gesellschaft für Biomolekulare Diagnostik mbH A method for stimulating the immune system
US6924355B2 (en) 1998-09-01 2005-08-02 Genentech, Inc. PRO1343 polypeptides
EP1115847A1 (en) 1998-09-25 2001-07-18 Children's Medical Center Corporation Short peptides which selectively modulate the activity of protein kinases
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
US6210892B1 (en) 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
JP4777515B2 (ja) * 1998-10-26 2011-09-21 エイブイアイ バイオファーマ， インコーポレイテッド ｐ５３モルホリノに基づくアンチセンス
US6399575B1 (en) 1998-11-10 2002-06-04 Auburn University Methods and compositions for targeting compounds to the central nervous system
US6133031A (en) 1999-08-19 2000-10-17 Isis Pharmaceuticals Inc. Antisense inhibition of focal adhesion kinase expression
US20040226056A1 (en) 1998-12-22 2004-11-11 Myriad Genetics, Incorporated Compositions and methods for treating neurological disorders and diseases
US20020049173A1 (en) 1999-03-26 2002-04-25 Bennett C. Frank Alteration of cellular behavior by antisense modulation of mRNA processing
US6379698B1 (en) 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
IL145586A0 (en) 1999-04-08 2002-06-30 Oasis Biosciences Inc Antisense oligonucleotides comprising universal and/or degenerate bases
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
US20030236214A1 (en) 1999-06-09 2003-12-25 Wolff Jon A. Charge reversal of polyion complexes and treatment of peripheral occlusive disease
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
WO2000078813A2 (en) 1999-06-18 2000-12-28 Emory University Huntington disease cellular model: stably transfected pc12 cells expressing mutant huntingtin
JP2004512810A (ja) 1999-08-31 2004-04-30 サーナ・セラピューティクス・インコーポレイテッド 核酸に基づく遺伝子発現の調節剤
DE60042338D1 (de) 1999-10-04 2009-07-16 Univ New Jersey Med TAR RNA bindende Peptide
US6165786A (en) 1999-11-03 2000-12-26 Isis Pharmaceuticals, Inc. Antisense modulation of nucleolin expression
WO2001059102A2 (en) 2000-02-08 2001-08-16 Ribozyme Pharmaceuticals, Inc. Nucleozymes with endonuclease activity
EP1133993A1 (en) 2000-03-10 2001-09-19 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Substances for the treatment of spinal muscular atrophy
AU4819101A (en) 2000-04-13 2001-10-30 University Of British Columbia, The Modulating cell survival by modulating huntingtin function
US6653467B1 (en) * 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
DE60139394D1 (de) 2000-04-28 2009-09-10 Asklepios Biopharmaceutical In Für das dystrophin-minigen kodierende dna-sequenzen und verfahren zu deren verwendung
KR100875003B1 (ko) 2000-05-01 2008-12-19 이데라 파마슈티칼즈, 인코포레이티드 누클레오시드의 위치 변형에 의한 올리고누클레오티드 ＣｐＧ 매개성 면역 자극의 조절
US20020013287A1 (en) 2000-05-09 2002-01-31 Reliable Biopharmaceuticals, Inc. St Louis Missouri Polymeric compounds useful as prodrugs
ATE464378T1 (de) 2000-05-31 2010-04-15 Serono Genetics Inst Sa Verwendung des globulären kopfes von acrp30 zur verstärkung der zunahme von muskelmasse und - differenzierung
CN1326990A (zh) 2000-06-07 2001-12-19 上海博德基因开发有限公司 一种新的多肽——人类dna cgg重复结合蛋白16.17和编码这种多肽的多核苷酸
US20030124523A1 (en) 2000-06-22 2003-07-03 Asselbergs Fredericus Alphonsus Maria Organic compounds
US6794192B2 (en) 2000-06-29 2004-09-21 Pfizer Inc. Target
RU2165149C1 (ru) 2000-07-03 2001-04-20 Шапошников Валерий Геннадьевич Система формования и упаковки изделий из сахарной ваты
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
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
AU3922802A (en) 2000-10-02 2002-05-27 Keck Graduate Inst Methods for identifying nucleotides at defined positions in target nucleic acidsusing fluorescence polarization
EP1366160B1 (en) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
US6623927B1 (en) 2000-11-08 2003-09-23 Council Of Scientific And Industrial Research Method of detection of allelic variants of SCA2 gene
AU2002236499A1 (en) 2000-11-30 2002-06-11 Uab Research Foundation Receptor-mediated uptake of peptides that bind the human transferrin receptor
US7001994B2 (en) 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
TW200526779A (en) 2001-02-08 2005-08-16 Wyeth Corp Modified and stabilized GDF propeptides and uses thereof
KR100408053B1 (ko) 2001-02-13 2003-12-01 엘지전자 주식회사 에스알엠의 토크리플 저감방법
AU2002256359A1 (en) * 2001-04-24 2002-11-05 Epigenesis Pharmaceuticals, Inc. Antisense and anti-inflammatory based compositions to treat respiratory disorders
WO2002092763A2 (en) 2001-05-11 2002-11-21 Regents Of The University Of Minnesota Intron associated with myotonic dystrophy type 2 and methods of use
US20050282188A1 (en) 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20050277133A1 (en) 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
AU2004266311B2 (en) 2001-05-18 2009-07-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20050014172A1 (en) 2002-02-20 2005-01-20 Ivan Richards RNA interference mediated inhibition of muscarinic cholinergic receptor gene expression using short interfering nucleic acid (siNA)
IL143379A (en) 2001-05-24 2013-11-28 Yissum Res Dev Co Oligonucleotide against human ache isoform r and its uses
HUP0401711A3 (en) 2001-06-26 2009-07-28 Bristol Myers Squibb Co N-heterocyclic inhibitors of tnf-alpha and pharmaceutical compositions containing them
CA2451738C (en) 2001-07-06 2013-09-17 Topigen Pharmaceutique Inc. Methods for increasing in vivo efficacy of oligonucleotides and inhibiting inflammation in mammals using 2-amino-2'-deoxyadenosine
US20030109476A1 (en) 2001-08-07 2003-06-12 Kmiec Eric B. Compositions and methods for the prevention and treatment of Huntington's disease
JP2005507244A (ja) 2001-08-10 2005-03-17 ノバルティス アクチエンゲゼルシャフト アテローム硬化性病変に結合するペプチド
US20060074034A1 (en) 2001-09-17 2006-04-06 Collins Douglas A Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof
KR20030035047A (ko) 2001-10-29 2003-05-09 (주)바이오코돈 Bmp-4 유전자 발현을 이용한 편평태선 질환의 치료 및진단방법
AU2002363253A1 (en) 2001-11-01 2003-05-12 Gpc Biotech Inc. Endothelial-cell binding peptides for diagnosis and therapy
US20030134790A1 (en) 2002-01-11 2003-07-17 University Of Medicine And Dentistry Of New Jersey Bone Morphogenetic Protein-2 And Bone Morphogenetic Protein-4 In The Treatment And Diagnosis Of Cancer
WO2003062258A1 (en) 2002-01-22 2003-07-31 The Cleveland Clinic Foundation Rnase l activator-antisense complexes
WO2003069330A1 (en) 2002-02-11 2003-08-21 The Trustees Of Columbia University In The City Of New York System and method for identifying proteins involved in force-initiated signal transduction
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
ES2339934T3 (es) 2002-03-01 2010-05-27 The Administrators Of The Tulane Educational Fund Conjugados de agentes citotoxicos y peptidos biologicamente activos.
US20040101852A1 (en) 2002-11-21 2004-05-27 Isis Pharmaceuticals Inc. Modulation of CGG triplet repeat binding protein 1 expression
ITRM20020253A1 (it) 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
US20040102395A1 (en) 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of IAP-like expression
EP1380644A1 (en) 2002-07-08 2004-01-14 Kylix B.V. The use of specified TCF target genes to identify drugs for the treatment of cancer, in particular colorectal cancer, in which TCF/beta-catenin/WNT signalling plays a central role
US7589059B2 (en) 2002-07-26 2009-09-15 Roche Madison Inc. Delivery of molecules and complexes to mammalian cells in vivo
US20050255086A1 (en) 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
JP2005535318A (ja) 2002-08-12 2005-11-24 ユニヴェルシテ ドゥ シェルブルック プレメッセンジャーｒｎａのスプライス部位選択の再プログラミング方法
GB0219143D0 (en) 2002-08-16 2002-09-25 Univ Leicester Modified tailed oligonucleotides
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
ES2283608T3 (es) 2002-10-23 2007-11-01 Centre For Research And Technology Hellas/Institute Of Agrobiotechnology In.A Secuencias peptidicas de union a la proteina prionica.
US7892793B2 (en) 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
US7250496B2 (en) 2002-11-14 2007-07-31 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory genes and uses thereof
PT2284266E (pt) 2002-11-14 2013-12-17 Thermo Fisher Scient Biosciences Inc Siarn contra tp53
US7655785B1 (en) 2002-11-14 2010-02-02 Rosetta Genomics Ltd. Bioinformatically detectable group of novel regulatory oligonucleotides and uses thereof
JP4777777B2 (ja) 2002-11-25 2011-09-21 雅文 松尾 ｍＲＮＡ前駆体のスプライシングを修飾するＥＮＡ核酸医薬
GB0228079D0 (en) 2002-12-02 2003-01-08 Laxdale Ltd Huntington's Disease
CA2518475C (en) 2003-03-07 2014-12-23 Alnylam Pharmaceuticals, Inc. Irna agents comprising asymmetrical modifications
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
US7514551B2 (en) 2003-04-03 2009-04-07 Enzo Life Sciences, Inc. Multisignal labeling reagents, and processes and uses therefor
AU2004239114B2 (en) 2003-05-14 2008-03-13 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
MXPA05012965A (es) 2003-06-02 2006-03-09 Wyeth Corp Uso de inhibidores de miostatina (gdf8) en conjuncion con corticoesteroides para el tratamiento de desordenes neuromusculares y composiciones farmaceuticas para ello.
DK1495769T3 (da) 2003-07-11 2008-06-23 Lbr Medbiotech B V Mannose-6-phosphat-receptormedieret gentransfer til muskelceller
US20050048495A1 (en) 2003-08-29 2005-03-03 Baker Brenda F. Isoform-specific targeting of splice variants
US20050054752A1 (en) 2003-09-08 2005-03-10 O'brien John P. Peptide-based diblock and triblock dispersants and diblock polymers
WO2005033134A2 (en) 2003-09-30 2005-04-14 Regeneron Pharmaceuticals, Inc. Secreted protein therapeutics and uses thereof
EP1680439A2 (en) 2003-10-14 2006-07-19 Kernel Biopharma Inc. Dual phase-pna conjugates for the delivery of pna through the blood brain barrier
US20050191636A1 (en) 2004-03-01 2005-09-01 Biocept, Inc. Detection of STRP, such as fragile X syndrome
US20080207538A1 (en) 2004-03-11 2008-08-28 Lawrence David S Enhanced Production of Functional Proteins From Defective Genes
EP1735443A2 (en) 2004-04-14 2006-12-27 Sirna Therapeutics, Inc. RNA INTERFERENCE MEDIATED TREATMENT OF POLYGLUTAMINE (POLYQ) REPEAT EXPANSION DISEASES USING SHORT INTERFERING NUCLEIC ACID (siNA)
JPWO2005116204A1 (ja) 2004-05-11 2008-06-19 株式会社アルファジェン Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
US20050288246A1 (en) 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
US20100183695A1 (en) 2004-05-27 2010-07-22 John Knopf Cerberus/coco derivatives and uses thereof
EP2500430B1 (en) 2004-06-28 2017-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
US7361468B2 (en) 2004-07-02 2008-04-22 Affymetrix, Inc. Methods for genotyping polymorphisms in humans
EP1618881A1 (en) * 2004-07-20 2006-01-25 Santhera Pharmaceuticals (Schweiz) GmbH Use of non-glucocorticoid steroids for the treatment of muscular dystrophy
WO2006017522A2 (en) 2004-08-03 2006-02-16 University Of Utah Research Foundation Use of antisense oligonucleotides to effect translation modulation
US20060099612A1 (en) * 2004-09-02 2006-05-11 Suntory Limited Method for analyzing genes of industrial yeasts
CA2582137A1 (en) 2004-10-05 2007-02-15 Wyeth Probe arrays for detecting multiple strains of different species
ITRM20040568A1 (it) 2004-11-18 2005-02-18 Uni Degli Studi Di Roma Tor Vergata Uso della tecnica "phage display" per l'identificazione di peptidi con capacita' di legame a cellule staminali/progenitore, peptidi cosi' ottenuti e loro usi.
US7838657B2 (en) 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
KR100663277B1 (ko) 2004-12-20 2007-01-02 삼성전자주식회사 휴대단말기의 시스템 관련 이벤트 처리 장치 및 방법
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
WO2006083800A2 (en) 2005-01-31 2006-08-10 University Of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
EP1877099B1 (en) 2005-04-06 2012-09-19 Genzyme Corporation Therapeutic conjugates comprising a lysosomal enzyme, polysialic acid and a targeting moiety
US20090312532A1 (en) 2005-04-22 2009-12-17 Van Deutekom Judith Christina Modulation of exon recognition in pre-mrna by interfering with the binding of sr proteins and by interfering with secodary rna structure
US7902352B2 (en) 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
WO2006121960A2 (en) 2005-05-06 2006-11-16 Medtronic, Inc. Methods and sequences to suppress primate huntington gene expression
WO2006121277A1 (en) 2005-05-09 2006-11-16 Korea Research Institute Of Standards And Science Fret probes for fluorescent detection of the epsps gene
EP2548560B1 (en) 2005-06-23 2015-06-03 Isis Pharmaceuticals, Inc. Compositions and methods for modulation of SMN2 splicing
ATE522626T1 (de) 2005-06-28 2011-09-15 Medtronic Inc Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken
EP2179737B1 (en) * 2005-07-01 2013-08-14 Index Pharmaceuticals AB Modulating responsiveness to steroids
CN101365801B (zh) 2005-10-28 2013-03-27 阿尔尼拉姆医药品有限公司 抑制亨廷顿基因表达的组合物和方法
US7906617B2 (en) 2005-12-15 2011-03-15 E. I. Du Pont De Nemours And Company Polyethylene binding peptides and methods of use
AU2007210038B2 (en) 2006-01-26 2013-01-10 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
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
US7855053B2 (en) 2006-07-19 2010-12-21 The Regents Of The University Of California Methods for detecting the presence of expanded CGG repeats in the FMR1 gene 5′ untranslated region
CN101501193B (zh) 2006-08-11 2013-07-03 普罗森那技术公司 用于治疗与dna重复不稳定性相关的遗传病的方法和手段
CA2660523C (en) 2006-08-11 2019-03-19 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
WO2008039418A2 (en) 2006-09-27 2008-04-03 Merck & Co., Inc. Acylated piperidine derivatives as melanocortin-4 receptor modulators
WO2008043561A2 (en) 2006-10-11 2008-04-17 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Influenza targets
WO2008103755A1 (en) 2007-02-20 2008-08-28 Mayo Foundation For Medical Education And Research Treating cancer with viral nucleic acid
PL381824A1 (pl) 2007-02-22 2008-09-01 Instytut Chemii Bioorganicznej Pan W Poznaniu Sekwencja siRNA, wektor, cel molekularny dla reagentów siRNA i wektorów wprowadzanych do komórek i tkanek, sposób oceny specyficzności wyciszania zmutowanego transkryptu, sposób badania oddziaływań enzymów szlaku interferencji RNA z transkryptami oraz zastosowanie sekwencji siRNA i wektora
ES2694726T3 (es) 2007-06-29 2018-12-26 Sarepta Therapeutics, Inc. Conjugados peptídicos específicos de tejido y métodos
WO2009008727A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
WO2009008725A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
WO2009015384A1 (en) 2007-07-26 2009-01-29 University Of Rochester Nucleic acid binding compounds and methods of use
AU2008286771B2 (en) 2007-08-15 2013-08-15 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
EP3238737B1 (en) 2007-10-26 2022-04-06 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
NZ587178A (en) 2008-02-08 2011-11-25 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)
EP2105145A1 (en) 2008-03-27 2009-09-30 ETH Zürich Method for muscle-specific delivery lipid-conjugated oligonucleotides
CA2726866A1 (en) 2008-05-09 2009-11-12 The University Of British Columbia Methods and compositions for the treatment of huntington's disease
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
US20110130346A1 (en) 2008-05-30 2011-06-02 Isis Innovation Limited Peptide conjugates for delvery of biologically active compounds
WO2009151600A2 (en) 2008-06-10 2009-12-17 Tufts University Smad proteins control drosha-mediated mirna maturation
GB2457965B8 (en) 2008-07-01 2011-02-16 Renovo Ltd Methods and systems for determining efficacy of medicaments.
WO2010006237A2 (en) 2008-07-11 2010-01-14 Alnylam Pharmaceuticals, Inc. Phosphorothioate oligonucleotides non-nucleosidic phosphorothiotes as delivery agents for irna agents
CA2732343C (en) 2008-07-29 2017-05-09 The Board Of Regents Of The University Of Texas System Selective inhibition of polyglutamine protein expression
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
CA2777486A1 (en) 2008-10-15 2010-04-22 4S3 Bioscience Inc. Methods and compositions for treatment of myotonic dystrophy
TR201902952T4 (tr) * 2008-10-24 2019-03-21 Sarepta Therapeutics Inc Dmd için ekson atlama bileşimleri.
PT2344637E (pt) 2008-10-27 2015-03-23 Academisch Ziekenhuis Leiden Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne
US20100248239A1 (en) 2009-03-24 2010-09-30 Mayo Foundation For Medical Education And Research Methods and materials for detecting fragile x mutations
AU2010229334B2 (en) 2009-03-24 2015-08-20 Asuragen, Inc. PCR methods for characterizing the 5' untranslated region of the FMR1 and FMR2 genes
CN109576268A (zh) 2009-04-10 2019-04-05 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
JP2012524540A (ja) 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド
KR20120047892A (ko) 2009-06-08 2012-05-14 미라젠 세러퓨틱스 ｍｉＲＮＡ 억제제 및 유사체를 위한 화학 변형 모티프
NZ599032A (en) 2009-09-11 2014-09-26 Isis Pharmaceuticals Inc Modulation of huntingtin expression
CN105838714B (zh) 2009-11-12 2020-07-17 西澳大利亚大学 反义分子和治疗疾病的方法
US20110166081A1 (en) 2009-12-03 2011-07-07 University Of Iowa Research Foundation Alpha-dystroglycan as a Protein Therapeutic
WO2011078797A2 (en) 2009-12-22 2011-06-30 Singapore Health Services Pte. Ltd Antisense oligonucleotides and uses threreof
JP6141018B2 (ja) 2009-12-24 2017-06-07 バイオマリン テクノロジーズ ベー．フェー． 炎症性障害を治療するための分子
US20130059902A1 (en) 2010-02-08 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
WO2011097614A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Mehods and compositions useful in diseases or conditions related to repeat expansion
ES2756326T3 (es) 2010-07-19 2020-04-27 Ionis Pharmaceuticals Inc Modulación de la expresión de distrofia miotónica-proteína quinasa (DMPK)
EP2605794B1 (en) 2010-08-20 2016-07-20 Replicor Inc. Oligonucleotide chelate complexes
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
CA2826836A1 (en) 2011-02-08 2012-08-16 The Charlotte-Mecklenburg Hospital Authority D/B/A Carolinas Medical Center Antisense oligonucleotides
EP2699269A1 (en) 2011-04-22 2014-02-26 Prosensa Technologies B.V. New compounds for treating, delaying and/or preventing a human genetic disorder such as myotonic dystrophy type 1 (dm1)
KR102229650B1 (ko) 2011-05-05 2021-03-19 사렙타 쎄러퓨틱스, 인코퍼레이티드 펩타이드 올리고뉴클레오타이드 접합체
US20140298496A1 (en) 2011-06-23 2014-10-02 Cold Spring Harbor Laboratory Phenocopy model of disease
ES2832531T3 (es) 2011-11-30 2021-06-10 Sarepta Therapeutics Inc Oligonucleótidos para el tratamiento de enfermedades por expansión de repeticiones
US9203291B2 (en) 2011-12-03 2015-12-01 Thomas Krupenkin Method and apparatus for mechanical energy harvesting using combined magnetic and microfluidic energy generation
JP2015502365A (ja) 2011-12-12 2015-01-22 オンコイミューニン，インコーポレイティド オリゴヌクレオチドのイン−ビボ送達
CN110079525A (zh) 2011-12-28 2019-08-02 日本新药株式会社 反义核酸
EP4043039A1 (en) 2012-01-27 2022-08-17 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
EP3330278A1 (en) 2012-02-08 2018-06-06 Ionis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
PT2841578T (pt) 2012-04-23 2017-08-10 Biomarin Tech Bv Resumo
AR091065A1 (es) 2012-05-18 2014-12-30 Replicor Inc Una formulacion farmaceutica que comprende un quelato de oligonucleotidos antiviral para el tratamiento de una infeccion antiviral
WO2014007620A2 (en) 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
EA201591694A1 (ru) * 2013-03-14 2016-06-30 Сарепта Терапьютикс, Инк. Композиции для пропуска экзонов для лечения мышечной дистрофии

Also Published As

Publication number Publication date
CY1117454T1 (el) 2017-04-26
JP2017141296A (ja) 2017-08-17
EP3238737B1 (en) 2022-04-06
EP2614827A3 (en) 2013-09-25
IL205322A (en) 2016-08-31
JP2021113229A (ja) 2021-08-05
US20140113955A1 (en) 2014-04-24
ES2639852T3 (es) 2017-10-30
US20140221458A1 (en) 2014-08-07
ES2692886T3 (es) 2018-12-05
EP4183399A1 (en) 2023-05-24
CN102264903A (zh) 2011-11-30
US20210139904A1 (en) 2021-05-13
EP2614827B1 (en) 2017-06-28
JP5879374B2 (ja) 2016-03-08
EP2614827A2 (en) 2013-07-17
US20170107512A1 (en) 2017-04-20
CN102264903B (zh) 2015-01-07
US20200239886A1 (en) 2020-07-30
US9499818B2 (en) 2016-11-22
CA2704049A1 (en) 2009-04-30
US20190100754A1 (en) 2019-04-04
JP2011502118A (ja) 2011-01-20
US20170044534A1 (en) 2017-02-16
US20110263682A1 (en) 2011-10-27
PL2203173T3 (pl) 2016-06-30
AU2008317566B2 (en) 2014-05-01
HUE027124T2 (en) 2016-08-29
US11427820B2 (en) 2022-08-30
EP2203173A2 (en) 2010-07-07
IL241928A0 (en) 2015-11-30
US20230151362A1 (en) 2023-05-18
IL261127B (en) 2020-07-30
HK1185098A1 (zh) 2014-02-07
WO2009054725A2 (en) 2009-04-30
ES2914775T3 (es) 2022-06-16
CN102256606A (zh) 2011-11-23
ES2564563T3 (es) 2016-03-23
IL241928B (en) 2018-08-30
US9528109B2 (en) 2016-12-27
US20160304864A1 (en) 2016-10-20
US20120022134A1 (en) 2012-01-26
AU2008317566A1 (en) 2009-04-30
JP2014111638A (ja) 2014-06-19
US20140128592A1 (en) 2014-05-08
US20190112604A1 (en) 2019-04-18
JP2019142942A (ja) 2019-08-29
IL261127A (en) 2018-10-31
HRP20160025T1 (hr) 2016-02-12
JP2016033140A (ja) 2016-03-10
US20190119679A1 (en) 2019-04-25
JP6885620B2 (ja) 2021-06-16
EP2203173B1 (en) 2015-12-23
US20150166996A1 (en) 2015-06-18
CY1117286T1 (el) 2017-04-26
US20190177725A1 (en) 2019-06-13
CN105641700B (zh) 2021-01-01
IL205322A0 (en) 2010-12-30
PT2203173E (pt) 2016-03-15
US9926557B2 (en) 2018-03-27
IL284321B (en) 2022-04-01
CN105641700A (zh) 2016-06-08
US20150073037A1 (en) 2015-03-12
HUE028662T2 (en) 2016-12-28
NZ592446A (en) 2012-03-30
US20110294753A1 (en) 2011-12-01
ES2936464T3 (es) 2023-03-17
JP6579629B2 (ja) 2019-09-25
IL284321A (en) 2021-07-29
WO2009054725A3 (en) 2009-10-15
JP7107622B2 (ja) 2022-07-27
CN105647921A (zh) 2016-06-08
US9243245B2 (en) 2016-01-26
HK1245670A1 (zh) 2018-08-31
JP5600064B2 (ja) 2014-10-01
NZ584793A (en) 2012-05-25
US10876114B2 (en) 2020-12-29
CN101896186A (zh) 2010-11-24
EP3238737A1 (en) 2017-11-01

Similar Documents

Publication Publication Date Title
JP7107622B2 (ja) 筋障害を相殺するための手段と方法
US11634714B2 (en) Oligonucleotide comprising an inosine for treating DMD
USRE48468E1 (en) Means and methods for counteracting muscle disorders