KR20180113990A - 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 - Google Patents

가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 Download PDF

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KR20180113990A
KR20180113990A KR1020187019657A KR20187019657A KR20180113990A KR 20180113990 A KR20180113990 A KR 20180113990A KR 1020187019657 A KR1020187019657 A KR 1020187019657A KR 20187019657 A KR20187019657 A KR 20187019657A KR 20180113990 A KR20180113990 A KR 20180113990A
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제임스 엠. 윌슨
다니엘 제이. 레이더
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더 트러스티스 오브 더 유니버시티 오브 펜실바니아
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    • A—HUMAN NECESSITIES
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    • 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
    • A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/63—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
    • 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
    • A61K48/0041—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 the non-active part being polymeric
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    • 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
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Immunology (AREA)
  • Hematology (AREA)
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  • Manufacturing & Machinery (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020187019657A 2015-12-11 2016-12-09 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법 Ceased KR20180113990A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562266383P 2015-12-11 2015-12-11
US62/266,383 2015-12-11
US201562269440P 2015-12-18 2015-12-18
US62/269,440 2015-12-18
PCT/US2016/065984 WO2017100682A1 (en) 2015-12-11 2016-12-09 Gene therapy for treating familial hypercholesterolemia

Publications (1)

Publication Number Publication Date
KR20180113990A true KR20180113990A (ko) 2018-10-17

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KR1020187019657A Ceased KR20180113990A (ko) 2015-12-11 2016-12-09 가족성 고콜레스테롤혈증을 치료하기 위한 유전자 요법

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Country Link
US (2) US10889832B2 (enExample)
EP (2) EP3400304B1 (enExample)
JP (2) JP7328760B2 (enExample)
KR (1) KR20180113990A (enExample)
CN (2) CN115957350A (enExample)
AU (2) AU2016366560A1 (enExample)
BR (1) BR112018011687A2 (enExample)
CA (1) CA3008142A1 (enExample)
CO (1) CO2018007165A2 (enExample)
ES (1) ES2921450T3 (enExample)
IL (2) IL259850B2 (enExample)
MA (1) MA43570A (enExample)
MX (1) MX2018007080A (enExample)
WO (1) WO2017100682A1 (enExample)

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CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
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CA3008142A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia
CA3051011A1 (en) 2017-02-20 2018-08-23 The Trustees Of The University Of Pennsylvania Gene therapy for treating familial hypercholesterolemia

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CN109121421A (zh) 2019-01-01
US20190002917A1 (en) 2019-01-03
EP3400304B1 (en) 2022-04-06
MX2018007080A (es) 2018-11-12
JP7328760B2 (ja) 2023-08-17
CN115957350A (zh) 2023-04-14
WO2017100682A1 (en) 2017-06-15
JP2019504003A (ja) 2019-02-14
CO2018007165A2 (es) 2018-07-19
AU2023201657A1 (en) 2023-06-15
EP4085934A1 (en) 2022-11-09
IL259850A (en) 2018-07-31
MA43570A (fr) 2018-11-14
EP3400304A1 (en) 2018-11-14
ES2921450T3 (es) 2022-08-25
IL259850B2 (en) 2023-11-01
US20210285013A1 (en) 2021-09-16
IL259850B1 (en) 2023-07-01
JP2022050575A (ja) 2022-03-30
AU2016366560A1 (en) 2018-06-28
IL303850A (en) 2023-08-01
CN109121421B (zh) 2022-11-01
US10889832B2 (en) 2021-01-12
CA3008142A1 (en) 2017-06-15
BR112018011687A2 (pt) 2018-12-04

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