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BRPI0811170B8 - oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila - Google Patents

oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila

Info

Publication number
BRPI0811170B8
BRPI0811170B8 BRPI0811170A BRPI0811170A BRPI0811170B8 BR PI0811170 B8 BRPI0811170 B8 BR PI0811170B8 BR PI0811170 A BRPI0811170 A BR PI0811170A BR PI0811170 A BRPI0811170 A BR PI0811170A BR PI0811170 B8 BRPI0811170 B8 BR PI0811170B8
Authority
BR
Brazil
Prior art keywords
rna
complexes
hydroxymethyl
group
substituted
Prior art date
Application number
BRPI0811170A
Other languages
English (en)
Inventor
Wengel Jesper
Original Assignee
Arcturus Therapeutics Inc
Marina Biotech Inc
Mdrna 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 Arcturus Therapeutics Inc, Marina Biotech Inc, Mdrna Inc filed Critical Arcturus Therapeutics Inc
Publication of BRPI0811170A2 publication Critical patent/BRPI0811170A2/pt
Publication of BRPI0811170B1 publication Critical patent/BRPI0811170B1/pt
Publication of BRPI0811170B8 publication Critical patent/BRPI0811170B8/pt

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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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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
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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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

oligonucleotídeos de rna e complecos de rna substituídos por hidroximetila a presente invenção é dirigida a oligonucleotídeos de rna ou complexos de oligonucleotídeos de rna, conjuntamente denominados no presente como complexos de rna, contando pelo menos um, porém opcionalmente mais que um, monômeros(s) de nucleotídeo substituídos por hidroximetila. por um monômero de nucleotídeo substituído por hidroximetila, entende-se um monômero de nucleotídeo, contendo uma grupo hidroximetila (que pode ser não substituído, o-substituído, por exemplo, por um grupo de conjugação, ou convertido em um grupo aminometila opcionalmente substituído ou conjugado). este grupo hidroximetila não participa na formação de uma ligação internucleotídica e não é o grupo hidroximetila (contendo o grupo 5'-hidroxila) de um monômero natural do rna. os complexos de rna da invenção podem ser úteis para aplicações terapêuticas, aplicação diagnósticas ou aplicações de pesquisa. os complexos incluem complexos curtos de rna interferente (sirna duplos) compreendendo um filamento antissenso e um filamento passageiro contínuo ou interrompido ("filamento senso") capaz de regular a expressão dos genes. pelo menos um desses filamentos, possivelmente mais que um desses filamentos, são modificadas por um ou mais monômero(s) de nucleotídeo substituído por hidroximetila desta invenção, que podem ser posicionados na extremidade 3', na extremidade 5' ou internamente. os complexos de rna da invenção também podem ser oligonucleotídeos de rna de filamento simples ("filamentos de rna") que são modificados por pelo menos um, porém opcionalmente, mais que um monômero(s) de nucleotídeo substituído por hidroximetila. tais filamentos de rna de filamento único devem ser considerados incluídos sempre que o termo complexos de rna for usado neste pedido de patente. os complexos da invenção exibem melhor estabilidade em relação à degradação nucleolítica em relação aos complexos correspondentes abrangidos inteiramente a partir dos monômeros naturais do rna.
BRPI0811170A 2007-05-22 2008-05-21 oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila BRPI0811170B8 (pt)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
DKPA200700751 2007-05-22
DKPA200700751 2007-05-22
DKPA200701718 2007-11-30
DKPA200701718 2007-11-30
DKPA200701785 2007-12-14
DKPA200701785 2007-12-14
DKPA200800534 2008-04-11
DKPA200800534 2008-04-11
PCT/US2008/064417 WO2008147824A2 (en) 2007-05-22 2008-05-21 Hydroxymethyl substituted rna oligonucleotides and rna complexes

Publications (3)

Publication Number Publication Date
BRPI0811170A2 BRPI0811170A2 (pt) 2014-10-07
BRPI0811170B1 BRPI0811170B1 (pt) 2020-12-01
BRPI0811170B8 true BRPI0811170B8 (pt) 2021-05-25

Family

ID=40075730

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0811170A BRPI0811170B8 (pt) 2007-05-22 2008-05-21 oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila

Country Status (17)

Country Link
US (6) US8314227B2 (pt)
EP (2) EP2162538B1 (pt)
JP (2) JP5726520B2 (pt)
KR (2) KR101629017B1 (pt)
CN (2) CN101679978B (pt)
AU (1) AU2008256871B2 (pt)
BR (1) BRPI0811170B8 (pt)
CA (1) CA2687850C (pt)
DK (1) DK2162538T3 (pt)
ES (1) ES2573936T3 (pt)
IL (1) IL202040A (pt)
MX (1) MX2009012568A (pt)
MY (1) MY153691A (pt)
NZ (1) NZ580712A (pt)
PL (1) PL2162538T3 (pt)
WO (1) WO2008147824A2 (pt)
ZA (1) ZA200907529B (pt)

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