JP7213238B2 - 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 - Google Patents

筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 Download PDF

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JP7213238B2
JP7213238B2 JP2020522046A JP2020522046A JP7213238B2 JP 7213238 B2 JP7213238 B2 JP 7213238B2 JP 2020522046 A JP2020522046 A JP 2020522046A JP 2020522046 A JP2020522046 A JP 2020522046A JP 7213238 B2 JP7213238 B2 JP 7213238B2
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muscle
seq
microdystrophin
muscular dystrophy
nucleotide sequence
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JP2021500352A (ja
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ルイーズ ロディノ－カラパック，
ジェリー アール． メンデル，
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リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル
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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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C—CHEMISTRY; METALLURGY
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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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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    • C12N15/09—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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • C—CHEMISTRY; METALLURGY
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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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  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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JP2020522046A 2017-10-18 2018-03-16 筋ジストロフィーを治療するための筋特異的マイクロジストロフィンのアデノ随伴ウイルスベクター送達 Active JP7213238B2 (ja)

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US201762573955P 2017-10-18 2017-10-18
US62/573,955 2017-10-18
PCT/US2018/022853 WO2019078916A1 (en) 2017-10-18 2018-03-16 ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY

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JP2021500352A JP2021500352A (ja) 2021-01-07
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US (2) US11534501B2 (enExample)
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EA202091739A1 (ru) 2018-01-31 2021-01-14 Рисёч Инститьют Эт Нэйшенвайд Чилдрен'С Хоспитал Генная терапия против тазово-плечевой мышечной дистрофии типа 2c
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CN113766935A (zh) 2019-02-26 2021-12-07 全国儿童医院研究所 β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
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IL299094A (en) * 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
RU2767335C1 (ru) * 2021-03-02 2022-03-17 Общество с ограниченной ответственностью «Марлин Биотех» Химерные белки на основе утрофина и дистрофина человека и их применение для лечения миодистрофии Дюшенна
EP4108263A3 (en) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP4219726A1 (en) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用

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WO2019078916A1 (en) 2019-04-25
US20230302157A1 (en) 2023-09-28
MA50836A (fr) 2020-08-26
EP3697915A1 (en) 2020-08-26
WO2019078916A9 (en) 2020-07-23
US20210128749A1 (en) 2021-05-06
EP3697915A4 (en) 2021-12-08
JP2021500352A (ja) 2021-01-07
US11534501B2 (en) 2022-12-27

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