JP2020511521A - ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 - Google Patents

ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 Download PDF

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JP2020511521A
JP2020511521A JP2019552611A JP2019552611A JP2020511521A JP 2020511521 A JP2020511521 A JP 2020511521A JP 2019552611 A JP2019552611 A JP 2019552611A JP 2019552611 A JP2019552611 A JP 2019552611A JP 2020511521 A JP2020511521 A JP 2020511521A
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composition
nucleic acid
therapeutic
expression
mab
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JP2020511521A5 (enExample
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ジェームズ デブズ，ロバート
ジェームズ デブズ，ロバート
ディー． ヒース，ティモシー
ディー． ヒース，ティモシー
ハンダムロンクル，チャクラポン
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ディーエヌエーアールエックス
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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
    • A61K48/005—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
    • A61K31/56—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids
    • A61K31/57—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone
    • A61K31/573—Compounds containing cyclopenta[a]hydrophenanthrene ring systems; Derivatives thereof, e.g. steroids substituted in position 17 beta by a chain of two carbon atoms, e.g. pregnane or progesterone substituted in position 21, e.g. cortisone, dexamethasone, prednisone or aldosterone
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    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
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    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
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    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
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    • C07K16/10—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from RNA viruses
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    • C07K16/24—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against cytokines, lymphokines or interferons
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2019552611A 2017-03-23 2018-03-23 ｉｎ ｖｉｖｏでの核酸発現のためのシステム及び方法 Pending JP2020511521A (ja)

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US201762475477P 2017-03-23 2017-03-23
US62/475,477 2017-03-23
PCT/US2018/024096 WO2018175932A1 (en) 2017-03-23 2018-03-23 Systems and methods for nucleic acid expression in vivo

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US (2) US11541130B2 (enExample)
EP (1) EP3600428A4 (enExample)
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CN (2) CN110678202B (enExample)
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WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
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FR3111913A1 (fr) * 2020-06-30 2021-12-31 Eyevensys Construction d’adn pour le traitement de pathologies oculaires
CN111863118B (zh) * 2020-07-20 2023-09-05 湖南莱博赛医用机器人有限公司 基于tct制片进行tct和dna倍体分析的方法
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CN115992211B (zh) * 2022-08-10 2025-07-29 南京鼓楼医院 一种用于狼疮性肾炎诊断的血清外泌体tsRNA标志物、探针及其应用
KR20250107834A (ko) * 2022-11-11 2025-07-14 빔 테라퓨틱스, 인크. 트랜스티레틴 유전자의 염기 편집
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