BR112018015164A2 - oligonucleotídeo de fita simples - Google Patents

oligonucleotídeo de fita simples

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Publication number
BR112018015164A2
BR112018015164A2 BR112018015164A BR112018015164A BR112018015164A2 BR 112018015164 A2 BR112018015164 A2 BR 112018015164A2 BR 112018015164 A BR112018015164 A BR 112018015164A BR 112018015164 A BR112018015164 A BR 112018015164A BR 112018015164 A2 BR112018015164 A2 BR 112018015164A2
Authority
BR
Brazil
Prior art keywords
nucleotide sequence
oligonucleotide
single stranded
hybridization
stranded oligonucleotide
Prior art date
Application number
BR112018015164A
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English (en)
Inventor
Nakajima Hiroyuki
KANAKI Tatsuro
Iriyama Yusuke
Original Assignee
Nissan Chemical Corp
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Publication date
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Application filed by Nissan Chemical Corp filed Critical Nissan Chemical Corp
Publication of BR112018015164A2 publication Critical patent/BR112018015164A2/pt

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    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
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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
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    • 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
    • C12N15/1137—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 against enzymes
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    • C12P19/26—Preparation of nitrogen-containing carbohydrates
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
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    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03067—Phosphatidylinositol-3,4,5-trisphosphate 3-phosphatase (3.1.3.67)
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/35—Nature of the modification
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    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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Abstract

a presente invenção refere-se a um oligonucleotídeo de fita simples que é capaz de controlar um gene alvo com alta eficiência e pode ser facilmente produzido. o oligonucleotídeo de fita simples é representado pela fórmula x-l-y onde x e y hibridizam através de uma primeira porção de sequência de nucleotídeos e uma segunda porção de sequência de nucleotídeos. x é composto por 7 a 100 nucleotídeos, contém pelo menos um nucleotídeo modificado, e tem uma primeira sequência de nucleotídeos que é capaz de hibridização com um segundo oligonucleotídeo e contém pelo menos quatro nucleotídeos contíguos reconhecidos por rnase h. y é composto por 4 a 100 nucleotídeos, e tem uma segunda sequência de nucleotídeos que é capaz de hibridização com um segundo oligonucleotídeo e contém pelo menos um ribonucleotídeo. pelo menos uma de sequência de nucleotídeos x e sequência de nucleotídeos y tem uma sequência antissentido capaz de hibridização com um rna alvo. l é um grupo derivado de um terceiro oligonucleotídeo que é degradado sob condições fisiológicas.
BR112018015164A 2016-01-26 2017-01-26 oligonucleotídeo de fita simples BR112018015164A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2016012804 2016-01-26
JP2016158833 2016-08-12
PCT/JP2017/002831 WO2017131124A1 (ja) 2016-01-26 2017-01-26 一本鎖オリゴヌクレオチド

Publications (1)

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BR112018015164A2 true BR112018015164A2 (pt) 2018-12-26

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Family Applications (1)

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BR112018015164A BR112018015164A2 (pt) 2016-01-26 2017-01-26 oligonucleotídeo de fita simples

Country Status (10)

Country Link
US (2) US11530409B2 (pt)
EP (1) EP3409779A4 (pt)
JP (3) JP6988481B2 (pt)
CN (2) CN116064544A (pt)
AU (1) AU2017211461B2 (pt)
BR (1) BR112018015164A2 (pt)
CA (1) CA3023222A1 (pt)
MX (2) MX2018009090A (pt)
TW (2) TWI803455B (pt)
WO (1) WO2017131124A1 (pt)

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