CY1116305T1 - Μεθοδοι και μεσα για την αποτελεσματικη παραλειψη του εξονιου 45 εις το προ-mrna της μυϊκης δυστροφιας τυπου duchenne - Google Patents

Μεθοδοι και μεσα για την αποτελεσματικη παραλειψη του εξονιου 45 εις το προ-mrna της μυϊκης δυστροφιας τυπου duchenne

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Publication number
CY1116305T1
CY1116305T1 CY20151100228T CY151100228T CY1116305T1 CY 1116305 T1 CY1116305 T1 CY 1116305T1 CY 20151100228 T CY20151100228 T CY 20151100228T CY 151100228 T CY151100228 T CY 151100228T CY 1116305 T1 CY1116305 T1 CY 1116305T1
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Cyprus
Prior art keywords
preservation
muscle
instruments
extension
methods
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CY20151100228T
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English (en)
Inventor
Kimpe Josephus Johannes De
Gerardus Johannes Platenburg
Deutekom Judith Christina Theodora Van
Annemieke Aartsma-Rus
Ommen Garrit-Jan Boudewijn Van
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Prosensa Technologies B.V.
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=40873329&utm_source=***_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1116305(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from PCT/NL2008/050673 external-priority patent/WO2009054725A2/en
Application filed by Prosensa Technologies B.V. filed Critical Prosensa Technologies B.V.
Publication of CY1116305T1 publication Critical patent/CY1116305T1/el

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Neurology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Biotechnology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Psychiatry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Hospice & Palliative Care (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

Η εφεύρεση αφορά μέθοδο, όπου χρησιμοποιείται μόριο για την επαγωγή και/ή προώθηση της παράλειψης τουλάχιστον ενός από τα εξόνιο 43, εξόνιο 46, εξόνια 50-53 του προ-mRNA DMD εις ασθενή, κατά προτίμηση εις απομονωμένο κύτταρο ασθενή, όπου η μέθοδος περιλαμβάνει παροχή μορίου εις το εν λόγω κύτταρο και/ή τον εν λόγω ασθενή. Η εφεύρεση αφορά επίσης το εν λόγω μόριο καθαυτό.
CY20151100228T 2008-10-27 2015-03-04 Μεθοδοι και μεσα για την αποτελεσματικη παραλειψη του εξονιου 45 εις το προ-mrna της μυϊκης δυστροφιας τυπου duchenne CY1116305T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
PCT/NL2008/050673 WO2009054725A2 (en) 2007-10-26 2008-10-27 Means and methods for counteracting muscle disorders
EP09788160.1A EP2344637B2 (en) 2008-10-27 2009-01-13 Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mrna

Publications (1)

Publication Number Publication Date
CY1116305T1 true CY1116305T1 (el) 2017-02-08

Family

ID=40873329

Family Applications (1)

Application Number Title Priority Date Filing Date
CY20151100228T CY1116305T1 (el) 2008-10-27 2015-03-04 Μεθοδοι και μεσα για την αποτελεσματικη παραλειψη του εξονιου 45 εις το προ-mrna της μυϊκης δυστροφιας τυπου duchenne

Country Status (15)

Country Link
EP (4) EP2607484B1 (el)
JP (2) JP5786109B2 (el)
AU (1) AU2009310557B2 (el)
CA (3) CA2741629C (el)
CY (1) CY1116305T1 (el)
DK (2) DK2607484T3 (el)
ES (2) ES2562658T3 (el)
HK (1) HK1160169A1 (el)
HR (1) HRP20160078T1 (el)
IL (2) IL212509A (el)
NZ (1) NZ592498A (el)
PL (1) PL2607484T3 (el)
PT (2) PT2344637E (el)
SI (1) SI2607484T1 (el)
WO (2) WO2010050801A1 (el)

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Publication number Publication date
PT2344637E (pt) 2015-03-23
PL2607484T3 (pl) 2016-06-30
SI2607484T1 (sl) 2016-11-30
CA3017539A1 (en) 2010-05-06
EP2349287B1 (en) 2018-08-15
EP2349287A2 (en) 2011-08-03
IL212508A0 (en) 2011-06-30
AU2009310558A1 (en) 2010-05-06
IL212508A (en) 2013-11-28
HK1160169A1 (en) 2012-08-10
JP5905260B2 (ja) 2016-04-20
JP5786109B2 (ja) 2015-09-30
ES2532634T3 (es) 2015-03-30
DK2607484T3 (en) 2016-03-07
EP2344637A1 (en) 2011-07-20
IL212509A (en) 2016-04-21
AU2009310558A8 (en) 2015-01-22
AU2009310557A1 (en) 2010-05-06
CA2741793A1 (en) 2010-05-06
WO2010050801A1 (en) 2010-05-06
WO2010050802A2 (en) 2010-05-06
NZ592498A (en) 2012-05-25
AU2009310558B2 (en) 2014-09-04
CA2741629A1 (en) 2010-05-06
EP2607484A1 (en) 2013-06-26
PT2607484E (pt) 2016-03-09
ES2532634T5 (es) 2018-04-30
WO2010050802A3 (en) 2010-08-12
CA2741793C (en) 2018-10-30
DK2344637T4 (en) 2018-04-23
AU2009310557A8 (en) 2011-09-29
EP2344637B1 (en) 2014-12-24
HRP20160078T1 (hr) 2016-04-08
EP3400948B1 (en) 2022-11-02
JP2012506697A (ja) 2012-03-22
DK2344637T3 (en) 2015-02-02
IL212509A0 (en) 2011-06-30
EP2607484B1 (en) 2016-01-06
CA2741629C (en) 2022-07-05
ES2562658T3 (es) 2016-03-07
EP3400948A1 (en) 2018-11-14
EP2344637B2 (en) 2018-02-28
AU2009310557B2 (en) 2014-09-11
JP2012506698A (ja) 2012-03-22

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