KR102139203B1 - 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터 - Google Patents

범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터 Download PDF

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KR102139203B1
KR102139203B1 KR1020137024782A KR20137024782A KR102139203B1 KR 102139203 B1 KR102139203 B1 KR 102139203B1 KR 1020137024782 A KR1020137024782 A KR 1020137024782A KR 20137024782 A KR20137024782 A KR 20137024782A KR 102139203 B1 KR102139203 B1 KR 102139203B1
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vector
aav
composition
seq
sequence
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KR20140044793A (ko
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로버트 맥클라렌
미구엘 시브라
매튜 존 두링
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옥스포드 유니버시티 이노베이션 리미티드
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    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • 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
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/12—Viral antigens
    • 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
    • A61K48/0008—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 '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
    • A61K48/0025—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 '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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1085—Transferases (2.) transferring alkyl or aryl groups other than methyl groups (2.5)
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020137024782A 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터 Expired - Fee Related KR102139203B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020207021515A KR102299430B1 (ko) 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터

Applications Claiming Priority (3)

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GBGB1103062.4A GB201103062D0 (en) 2011-02-22 2011-02-22 Method
GB1103062.4 2011-02-22
PCT/GB2012/050376 WO2012114090A1 (en) 2011-02-22 2012-02-21 Aav -vectors for use in gene therapy of choroideremia

Related Child Applications (1)

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KR1020207021515A Division KR102299430B1 (ko) 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터

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KR20140044793A KR20140044793A (ko) 2014-04-15
KR102139203B1 true KR102139203B1 (ko) 2020-07-29

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KR1020137024782A Expired - Fee Related KR102139203B1 (ko) 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터
KR1020207021515A Expired - Fee Related KR102299430B1 (ko) 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터
KR1020217028082A Ceased KR20210112407A (ko) 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터

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KR1020207021515A Expired - Fee Related KR102299430B1 (ko) 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터
KR1020217028082A Ceased KR20210112407A (ko) 2011-02-22 2012-02-21 범맥락막위축의 유전자 치료에서 사용하기 위한 ａａｖ－벡터

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US (4) US9834788B2 (https=)
EP (2) EP3428283A1 (https=)
JP (4) JP2014512171A (https=)
KR (3) KR102139203B1 (https=)
CN (2) CN103562396A (https=)
AU (2) AU2012220404B2 (https=)
BR (1) BR112013021318B1 (https=)
CA (1) CA2827975C (https=)
DK (1) DK2678435T3 (https=)
ES (1) ES2676550T5 (https=)
GB (1) GB201103062D0 (https=)
HK (2) HK1258917A1 (https=)
IL (1) IL228031B (https=)
MX (2) MX356525B (https=)
PL (1) PL2678435T3 (https=)
PT (1) PT2678435T (https=)
SG (1) SG192911A1 (https=)
WO (1) WO2012114090A1 (https=)

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KR20140044793A (ko) 2014-04-15
BR112013021318B1 (pt) 2021-12-07
JP2014512171A (ja) 2014-05-22
PT2678435T (pt) 2018-07-17
GB201103062D0 (en) 2011-04-06
EP3428283A1 (en) 2019-01-16
MX356525B (es) 2018-06-01
AU2012220404A1 (en) 2013-09-12
US20180135078A1 (en) 2018-05-17
ES2676550T5 (es) 2024-06-27
JP2020054345A (ja) 2020-04-09
CN103562396A (zh) 2014-02-05
JP2023036618A (ja) 2023-03-14
AU2012220404B2 (en) 2017-04-20
MX2013009604A (es) 2013-09-16
US20210032656A1 (en) 2021-02-04
CN107881198A (zh) 2018-04-06
US9834788B2 (en) 2017-12-05
KR102299430B1 (ko) 2021-09-07
HK1249548A1 (zh) 2018-11-02
WO2012114090A1 (en) 2012-08-30
US20230332176A1 (en) 2023-10-19
IL228031B (en) 2018-02-28
PL2678435T3 (pl) 2018-11-30
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MX388146B (es) 2025-03-19
BR112013021318A2 (pt) 2016-10-25
CA2827975A1 (en) 2012-08-30
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