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CY1121134T1 - Συνθεσεις και μεθοδοι για την τροποποιηση της smn2 συγκολλησης - Google Patents

Συνθεσεις και μεθοδοι για την τροποποιηση της smn2 συγκολλησης

Info

Publication number
CY1121134T1
CY1121134T1 CY191100060T CY191100060T CY1121134T1 CY 1121134 T1 CY1121134 T1 CY 1121134T1 CY 191100060 T CY191100060 T CY 191100060T CY 191100060 T CY191100060 T CY 191100060T CY 1121134 T1 CY1121134 T1 CY 1121134T1
Authority
CY
Cyprus
Prior art keywords
compositions
methods
smn2
welding
modification
Prior art date
Application number
CY191100060T
Other languages
English (en)
Inventor
Brenda F Baker
Adrian R Krainer
Yimin Hua
Original Assignee
Biogen Ma Inc
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
Application filed by Biogen Ma Inc filed Critical Biogen Ma Inc
Publication of CY1121134T1 publication Critical patent/CY1121134T1/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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N15/111—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
    • 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
    • C12N2310/32—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
    • C12N2310/33—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
    • 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)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Αποκαλύπτονται εδώ ενώσεις, συνθέσεις και μέθοδοι για την τροποποίηση της συγκόλλησης SMN2 mRNA σε ένα κύτταρο, ιστό ή ζώο. Παρέχονται επίσης χρήσεις αποκαλυπτόμενων ενώσεων και συνθέσεων στην παραγωγή ενός φαρμάκου για την αγωγή νόσων και διαταραχών, όπου συμπεριλαμβάνεται η νωτιαία μυϊκή ατροφία.
CY191100060T 2005-06-23 2019-01-17 Συνθεσεις και μεθοδοι για την τροποποιηση της smn2 συγκολλησης CY1121134T1 (el)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US69354205P 2005-06-23 2005-06-23
EP06773838A EP1910395B1 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of SMN2 splicing
EP13165321.4A EP2644700A1 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of SMN2 splicing
EP12188625.3A EP2548560B1 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of SMN2 splicing
PCT/US2006/024469 WO2007002390A2 (en) 2005-06-23 2006-06-23 Compositions and methods for modulation of smn2 splicing
EP17151519.0 2017-01-13

Publications (1)

Publication Number Publication Date
CY1121134T1 true CY1121134T1 (el) 2019-12-11

Family

ID=37595872

Family Applications (2)

Application Number Title Priority Date Filing Date
CY2017040C CY2017040I2 (el) 2005-06-23 2017-11-27 Συνθεσεις και μεθοδοι για διαμορφωση ματισματος smn2
CY191100060T CY1121134T1 (el) 2005-06-23 2019-01-17 Συνθεσεις και μεθοδοι για την τροποποιηση της smn2 συγκολλησης

Family Applications Before (1)

Application Number Title Priority Date Filing Date
CY2017040C CY2017040I2 (el) 2005-06-23 2017-11-27 Συνθεσεις και μεθοδοι για διαμορφωση ματισματος smn2

Country Status (13)

Country Link
US (6) US8361977B2 (el)
EP (5) EP3470072A1 (el)
CY (2) CY2017040I2 (el)
DK (3) DK2548560T3 (el)
ES (3) ES2397113T3 (el)
HK (2) HK1181307A1 (el)
HU (2) HUE027486T2 (el)
LT (2) LT3308788T (el)
LU (1) LUC00046I2 (el)
PL (3) PL2548560T3 (el)
PT (3) PT1910395E (el)
SI (2) SI2548560T1 (el)
WO (1) WO2007002390A2 (el)

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