RU2762747C2 - Генная терапия офтальмологических нарушений - Google Patents

Генная терапия офтальмологических нарушений Download PDF

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RU2762747C2
RU2762747C2 RU2018125468A RU2018125468A RU2762747C2 RU 2762747 C2 RU2762747 C2 RU 2762747C2 RU 2018125468 A RU2018125468 A RU 2018125468A RU 2018125468 A RU2018125468 A RU 2018125468A RU 2762747 C2 RU2762747 C2 RU 2762747C2
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aav
vector
promoter
accordance
composition
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RU2018125468A
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RU2018125468A3 (enExample
RU2018125468A (ru
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Джин БЕННЕТТ
Жаннет БЕННИЧЕЛЛИ
Цзюньвей СУНЬ
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Зе Трастис Оф Зе Юниверсити Оф Пенсильвания
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    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/002—Vectors comprising a special translation-regulating system controllable or inducible
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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RU2018125468A 2015-12-14 2016-12-13 Генная терапия офтальмологических нарушений RU2762747C2 (ru)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562266789P 2015-12-14 2015-12-14
US62/266,789 2015-12-14
PCT/US2016/066402 WO2017106202A2 (en) 2015-12-14 2016-12-13 Gene therapy for ocular disorders

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RU2018125468A RU2018125468A (ru) 2020-01-16
RU2018125468A3 RU2018125468A3 (enExample) 2020-10-22
RU2762747C2 true RU2762747C2 (ru) 2021-12-22

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US (2) US11090392B2 (enExample)
EP (2) EP3795180B1 (enExample)
JP (2) JP7057281B2 (enExample)
KR (1) KR20180099719A (enExample)
CN (1) CN109069668B (enExample)
AU (1) AU2016370487C1 (enExample)
BR (1) BR112018011838A2 (enExample)
CA (1) CA3008264A1 (enExample)
ES (1) ES2826384T3 (enExample)
MX (2) MX2018007230A (enExample)
RU (1) RU2762747C2 (enExample)
WO (1) WO2017106202A2 (enExample)

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