EA201791417A1 - Супрессия гена гентингтина, индуцированная рнк-интерференцией - Google Patents

Супрессия гена гентингтина, индуцированная рнк-интерференцией

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Publication number
EA201791417A1
EA201791417A1 EA201791417A EA201791417A EA201791417A1 EA 201791417 A1 EA201791417 A1 EA 201791417A1 EA 201791417 A EA201791417 A EA 201791417A EA 201791417 A EA201791417 A EA 201791417A EA 201791417 A1 EA201791417 A1 EA 201791417A1
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Eurasian Patent Office
Prior art keywords
rna
induced
sequence
gentingin
rnc
Prior art date
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EA201791417A
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English (en)
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EA037696B1 (ru
Inventor
Павлина Стефанова Константинова
Яна Миниарикова
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ЮНИКЕР АйПи Б.В.
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Publication of EA201791417A1 publication Critical patent/EA201791417A1/ru
Publication of EA037696B1 publication Critical patent/EA037696B1/ru

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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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
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    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
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  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Настоящее изобретение обеспечивает двухцепочечную РНК, содержащую первую последовательность РНК и вторую последовательность РНК, в которой первая и вторая последовательности РНК являются, по существу, комплементарными, причем первая последовательность РНК имеет длину последовательности, равную по меньшей мере 19 нуклеотидам, и является, по существу, комплементарной последовательности SEQ ID NO: 1. Двухцепочечная РНК предназначена для применения в индукции РНК-интерференции против последовательностей экзона 1 гена белка гентингтина. Двухцепочечная РНК по настоящему изобретению была способна снижать гибель нейрональных клеток и образование агрегатов белка гентингтина в экспериментальной модели на животных.
EA201791417A 2014-12-24 2015-12-23 Супрессия гена гентингтина, индуцированная рнк-интерференцией EA037696B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP14200308 2014-12-24
PCT/EP2015/081157 WO2016102664A1 (en) 2014-12-24 2015-12-23 Rnai induced huntingtin gene suppression

Publications (2)

Publication Number Publication Date
EA201791417A1 true EA201791417A1 (ru) 2017-12-29
EA037696B1 EA037696B1 (ru) 2021-05-12

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US (4) US10174321B2 (ru)
EP (2) EP3540063A1 (ru)
JP (2) JP6839094B2 (ru)
KR (1) KR102636633B1 (ru)
CN (1) CN108064292B (ru)
AU (1) AU2015370903B2 (ru)
CA (1) CA2971920C (ru)
CO (1) CO2017007335A2 (ru)
CY (1) CY1121733T1 (ru)
DK (1) DK3237618T3 (ru)
EA (1) EA037696B1 (ru)
ES (1) ES2732023T3 (ru)
HK (1) HK1246344B (ru)
HR (1) HRP20190992T1 (ru)
HU (1) HUE043842T2 (ru)
IL (1) IL252990B (ru)
LT (1) LT3237618T (ru)
MX (1) MX2017008500A (ru)
PL (1) PL3237618T3 (ru)
PT (1) PT3237618T (ru)
RS (1) RS58862B1 (ru)
SI (1) SI3237618T1 (ru)
TR (1) TR201908969T4 (ru)
WO (1) WO2016102664A1 (ru)

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US20230119344A1 (en) 2023-04-20
HUE043842T2 (hu) 2019-09-30
HRP20190992T1 (hr) 2019-09-20
SI3237618T1 (sl) 2019-09-30
PL3237618T3 (pl) 2019-09-30
CA2971920A1 (en) 2016-06-30
PT3237618T (pt) 2019-07-04
EP3237618A1 (en) 2017-11-01
KR102636633B1 (ko) 2024-02-13
AU2015370903B2 (en) 2021-06-17
CN108064292B (zh) 2021-05-04
EA037696B1 (ru) 2021-05-12
EP3540063A1 (en) 2019-09-18
CA2971920C (en) 2024-05-07
HUE043842T4 (hu) 2019-08-28
RS58862B1 (sr) 2019-07-31
DK3237618T3 (da) 2019-06-24
WO2016102664A1 (en) 2016-06-30
US20190144860A1 (en) 2019-05-16
CY1121733T1 (el) 2020-07-31
US20170355989A1 (en) 2017-12-14
EP3237618B1 (en) 2019-05-22
JP6839094B2 (ja) 2021-03-03
IL252990A0 (en) 2017-08-31
BR112017013573A2 (pt) 2018-03-06
TR201908969T4 (tr) 2019-07-22
HK1246344B (zh) 2020-01-10
MX2017008500A (es) 2018-02-01
KR20170120572A (ko) 2017-10-31
US10767180B2 (en) 2020-09-08
LT3237618T (lt) 2019-07-10
US20200339992A1 (en) 2020-10-29
US11371044B2 (en) 2022-06-28
AU2015370903A1 (en) 2017-07-20
IL252990B (en) 2021-09-30
CO2017007335A2 (es) 2018-01-05
NZ733296A (en) 2021-10-29
CN108064292A (zh) 2018-05-22
US10174321B2 (en) 2019-01-08
JP2021000136A (ja) 2021-01-07
ES2732023T3 (es) 2019-11-20
JP2018502601A (ja) 2018-02-01

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