NO954550L - Antisens oligonukleotider som forhindrer avvikende spleising og fremgangsmåte til å anvende de samme - Google Patents

Antisens oligonukleotider som forhindrer avvikende spleising og fremgangsmåte til å anvende de samme

Info

Publication number
NO954550L
NO954550L NO954550A NO954550A NO954550L NO 954550 L NO954550 L NO 954550L NO 954550 A NO954550 A NO 954550A NO 954550 A NO954550 A NO 954550A NO 954550 L NO954550 L NO 954550L
Authority
NO
Norway
Prior art keywords
mrna
splicing
aberrant
mutation
same
Prior art date
Application number
NO954550A
Other languages
English (en)
Other versions
NO954550D0 (no
Inventor
Ryszard Kole
Zbigniew Dominski
Original Assignee
Univ North Carolina
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 Univ North Carolina filed Critical Univ North Carolina
Publication of NO954550D0 publication Critical patent/NO954550D0/no
Publication of NO954550L publication Critical patent/NO954550L/no

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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
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)

Abstract

Fremgangsmåte til å bekjempe avvikende spleising i et pre-mRNA molekyl som inneholder en mutasjon er beskrevet. Når den er tilstede i pre- mRNA forårsaker mutasjonen at pre-mRNA spleises ukorrekt og produserer et avvikende ! mRNA eller mRNA-fragment forskjellig fra det ; mRNA som ordinært blir kodet ved pre-mRNA. Fremgangsmåten omfatter å hybridisere et antisens oligonukleotid til pre-mRNA molekylet for å danne et dupleks molekyl under betingelser som tillater spleising. Antisens oligonukleotidet er et som ikke aktiverer RNase H og er selektert for å blokkere et medlem av det avvikende sett av spleiseelementer dannet ved mutasjonen slik at det native intron blir fjernet ved spleising og det første mRNA molekylet som koder et nativt protein blir fremstilt. Oligonukleotider som er nyttige til å utføre fremgangsmåten er også beskrevet.
NO954550A 1993-05-11 1995-11-10 Antisens oligonukleotider som forhindrer avvikende spleising og fremgangsmåte til å anvende de samme NO954550L (no)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US6247193A 1993-05-11 1993-05-11
PCT/US1994/005181 WO1994026887A1 (en) 1993-05-11 1994-05-10 Antisense oligonucleotides which combat aberrant splicing and methods of using the same

Publications (2)

Publication Number Publication Date
NO954550D0 NO954550D0 (no) 1995-11-10
NO954550L true NO954550L (no) 1996-01-10

Family

ID=22042714

Family Applications (1)

Application Number Title Priority Date Filing Date
NO954550A NO954550L (no) 1993-05-11 1995-11-10 Antisens oligonukleotider som forhindrer avvikende spleising og fremgangsmåte til å anvende de samme

Country Status (15)

Country Link
US (4) US5665593A (no)
EP (2) EP1897942A1 (no)
JP (3) JPH08510130A (no)
CN (1) CN1123038A (no)
AT (1) ATE368107T1 (no)
AU (1) AU6786594A (no)
CA (1) CA2162361C (no)
DE (1) DE69435005T2 (no)
DK (1) DK0698092T3 (no)
ES (1) ES2290951T3 (no)
FI (1) FI955428A (no)
NO (1) NO954550L (no)
NZ (1) NZ266386A (no)
PT (1) PT698092E (no)
WO (1) WO1994026887A1 (no)

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