CY1121198T1 - Αντινοημaτικα μορια και μεθοδοι για την θεραπεια παθολογιων - Google Patents

Αντινοημaτικα μορια και μεθοδοι για την θεραπεια παθολογιων

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Publication number
CY1121198T1
CY1121198T1 CY20181101163T CY181101163T CY1121198T1 CY 1121198 T1 CY1121198 T1 CY 1121198T1 CY 20181101163 T CY20181101163 T CY 20181101163T CY 181101163 T CY181101163 T CY 181101163T CY 1121198 T1 CY1121198 T1 CY 1121198T1
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CY
Cyprus
Prior art keywords
pathologies
treatment
methods
terminal molecules
molecules
Prior art date
Application number
CY20181101163T
Other languages
English (en)
Inventor
Stephen Wilton
Sue Fletcher
Abbie Adams
Penny Meloni
Original Assignee
The University Of Western Australia
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from AU2009905549A external-priority patent/AU2009905549A0/en
Application filed by The University Of Western Australia filed Critical The University Of Western Australia
Publication of CY1121198T1 publication Critical patent/CY1121198T1/el

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • A61K31/70—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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/351—Conjugate
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • C12N2320/33—Alteration of splicing

Abstract

Ένα αντινοηματικό μόριο ικανό να δεσμεύεται σε μια επιλεγμένη θέση στόχο ώστε να επάγει παράλειψη εξονίου στο γονίδιο της δυστροφίνης, όπως εκτίθεται στην SEQ ID NO: 1 έως 59.
CY20181101163T 2009-11-12 2018-11-07 Αντινοημaτικα μορια και μεθοδοι για την θεραπεια παθολογιων CY1121198T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
AU2009905549A AU2009905549A0 (en) 2009-11-12 Antisense Molecules and Methods for Treating Pathologies
PCT/AU2010/001520 WO2011057350A1 (en) 2009-11-12 2010-11-12 Antisense molecules and methods for treating pathologies

Publications (1)

Publication Number Publication Date
CY1121198T1 true CY1121198T1 (el) 2020-05-29

Family

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CY20181101163T CY1121198T1 (el) 2009-11-12 2018-11-07 Αντινοημaτικα μορια και μεθοδοι για την θεραπεια παθολογιων

Country Status (22)

Country Link
US (7) US8637483B2 (el)
EP (2) EP3431603A1 (el)
JP (6) JP5963678B2 (el)
KR (8) KR102113306B1 (el)
CN (2) CN105838714B (el)
AU (5) AU2010317599B2 (el)
BR (2) BR112012011195B1 (el)
CA (1) CA2780563A1 (el)
CY (1) CY1121198T1 (el)
DK (1) DK2499249T3 (el)
ES (1) ES2693459T3 (el)
HR (1) HRP20181824T1 (el)
HU (1) HUE040445T2 (el)
IL (5) IL297299A (el)
LT (1) LT2499249T (el)
NZ (2) NZ716534A (el)
PL (1) PL2499249T3 (el)
PT (1) PT2499249T (el)
RS (1) RS58079B1 (el)
SI (1) SI2499249T1 (el)
TR (1) TR201816523T4 (el)
WO (1) WO2011057350A1 (el)

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KR20130084595A (ko) 2013-07-25
EP3431603A1 (en) 2019-01-23
TR201816523T4 (tr) 2018-11-21
KR102366851B1 (ko) 2022-02-23
US9758783B2 (en) 2017-09-12
AU2018202105A1 (en) 2018-04-19
KR20230010814A (ko) 2023-01-19
KR101958491B1 (ko) 2019-03-15
AU2023203103A1 (en) 2023-06-08
NZ716534A (en) 2018-06-29
US10287586B2 (en) 2019-05-14
JP6936830B2 (ja) 2021-09-22
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US9228187B2 (en) 2016-01-05

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