RU2018121529A - МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD) - Google Patents

МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD) Download PDF

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RU2018121529A
RU2018121529A RU2018121529A RU2018121529A RU2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A RU 2018121529 A RU2018121529 A RU 2018121529A
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use according
apociii
compound
stranded
interfering rna
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RU2018121529A
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English (en)
Russian (ru)
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Вероника Дж. АЛЕКСАНДЕР
Николас Дж. ВИНЕЙ
Джозеф Л. УИТЗТУМ
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Ионис Фармасьютикалз, Инк.
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Application filed by Ионис Фармасьютикалз, Инк. filed Critical Ионис Фармасьютикалз, Инк.
Publication of RU2018121529A publication Critical patent/RU2018121529A/ru

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    • 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
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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
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    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
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    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • 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
    • C12N2310/3525—MOE, methoxyethoxy
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Steroid Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
RU2018121529A 2013-02-14 2014-02-14 МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD) RU2018121529A (ru)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201361764969P 2013-02-14 2013-02-14
US61/764,969 2013-02-14
US201361880779P 2013-09-20 2013-09-20
US61/880,779 2013-09-20

Related Parent Applications (1)

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RU2015139055A Division RU2661781C2 (ru) 2013-02-14 2014-02-14 Модулирование экспрессии аполипопротеина с-iii (аросiii) у людей с дефицитом липопротеинлипазы (lpld)

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RU2018121529A true RU2018121529A (ru) 2019-03-06

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RU2018121529A RU2018121529A (ru) 2013-02-14 2014-02-14 МОДУЛИРОВАНИЕ ЭКСПРЕССИИ АПОЛИПОПРОТЕИНА C-III (AроCIII) У ЛЮДЕЙ С ДЕФИЦИТОМ ЛИПОПРОТЕИНЛИПАЗЫ (LPLD)
RU2015139055A RU2661781C2 (ru) 2013-02-14 2014-02-14 Модулирование экспрессии аполипопротеина с-iii (аросiii) у людей с дефицитом липопротеинлипазы (lpld)

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US (6) US9593333B2 (cg-RX-API-DMAC10.html)
EP (2) EP3400947B1 (cg-RX-API-DMAC10.html)
JP (3) JP6313789B2 (cg-RX-API-DMAC10.html)
KR (2) KR20200123263A (cg-RX-API-DMAC10.html)
CN (2) CN108743943A (cg-RX-API-DMAC10.html)
AU (3) AU2014216137B2 (cg-RX-API-DMAC10.html)
BR (1) BR112015019515A8 (cg-RX-API-DMAC10.html)
CA (2) CA3170716A1 (cg-RX-API-DMAC10.html)
DK (1) DK2956176T3 (cg-RX-API-DMAC10.html)
ES (2) ES2961686T3 (cg-RX-API-DMAC10.html)
IL (2) IL240338B (cg-RX-API-DMAC10.html)
MX (2) MX368155B (cg-RX-API-DMAC10.html)
NZ (1) NZ631119A (cg-RX-API-DMAC10.html)
RU (2) RU2018121529A (cg-RX-API-DMAC10.html)
WO (1) WO2014127268A2 (cg-RX-API-DMAC10.html)
ZA (1) ZA201505628B (cg-RX-API-DMAC10.html)

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