RU2764587C2 - Способы и композиции для лечения хореи гентингтона - Google Patents

Способы и композиции для лечения хореи гентингтона Download PDF

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RU2764587C2
RU2764587C2 RU2018140499A RU2018140499A RU2764587C2 RU 2764587 C2 RU2764587 C2 RU 2764587C2 RU 2018140499 A RU2018140499 A RU 2018140499A RU 2018140499 A RU2018140499 A RU 2018140499A RU 2764587 C2 RU2764587 C2 RU 2764587C2
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sequence
seq
paragraphs
modulating
nucleotide sequence
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RU2018140499A3 (OSRAM
RU2018140499A (ru
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Динах Вен-Йи Сах
Цзиньчжао ХОУ
Пэнчэн ЧЖОУ
Синь Ван
Йохен Деккерт
Маркус ХОССБАХ
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Вояджер Терапьютикс, Инк.
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
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    • A61K48/0008Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025Medicinal 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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
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    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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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    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Virology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
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RU2018140499A 2016-05-18 2017-05-18 Способы и композиции для лечения хореи гентингтона RU2764587C2 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662338113P 2016-05-18 2016-05-18
US62/338,113 2016-05-18
US201762485049P 2017-04-13 2017-04-13
US62/485,049 2017-04-13
PCT/US2017/033281 WO2017201258A1 (en) 2016-05-18 2017-05-18 Compositions and methods of treating huntington's disease

Publications (3)

Publication Number Publication Date
RU2018140499A RU2018140499A (ru) 2020-06-18
RU2018140499A3 RU2018140499A3 (OSRAM) 2021-01-25
RU2764587C2 true RU2764587C2 (ru) 2022-01-18

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US (2) US11951121B2 (OSRAM)
EP (1) EP3458589A4 (OSRAM)
JP (1) JP7220080B2 (OSRAM)
KR (2) KR102427379B1 (OSRAM)
CN (1) CN109831916B (OSRAM)
AU (1) AU2017268382B2 (OSRAM)
BR (1) BR112018073472A2 (OSRAM)
CA (1) CA3024449A1 (OSRAM)
IL (2) IL297576B2 (OSRAM)
MX (1) MX2018014152A (OSRAM)
RU (1) RU2764587C2 (OSRAM)
SG (1) SG11201809643UA (OSRAM)
WO (1) WO2017201258A1 (OSRAM)

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