JP7676111B2 - 筋ジストロフィーを治療するための筋肉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肉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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JP7676111B2
JP7676111B2 JP2019550767A JP2019550767A JP7676111B2 JP 7676111 B2 JP7676111 B2 JP 7676111B2 JP 2019550767 A JP2019550767 A JP 2019550767A JP 2019550767 A JP2019550767 A JP 2019550767A JP 7676111 B2 JP7676111 B2 JP 7676111B2
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muscle
microdystrophin
aav
mhck7
vector
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JP2020513811A5 (enExample
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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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • 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
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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
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    • 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
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    • 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
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    • C12N15/86—Viral vectors
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
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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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JP2019550767A 2017-03-17 2018-03-16 筋ジストロフィーを治療するための筋肉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 JP7676111B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023019888A JP2023053254A (ja) 2017-03-17 2023-02-13 筋ジストロフィーを治療するための筋肉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
JP2024229759A JP2025061676A (ja) 2017-03-17 2024-12-26 筋ジストロフィーを治療するための筋肉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

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762473148P 2017-03-17 2017-03-17
US62/473,148 2017-03-17
PCT/US2018/022881 WO2018170408A1 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

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JP2023019888A Division JP2023053254A (ja) 2017-03-17 2023-02-13 筋ジストロフィーを治療するための筋肉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
JP2024229759A Division JP2025061676A (ja) 2017-03-17 2024-12-26 筋ジストロフィーを治療するための筋肉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

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JP2020513811A JP2020513811A (ja) 2020-05-21
JP2020513811A5 JP2020513811A5 (enExample) 2021-04-22
JP7676111B2 true JP7676111B2 (ja) 2025-05-14

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JP2019550767A Active JP7676111B2 (ja) 2017-03-17 2018-03-16 筋ジストロフィーを治療するための筋肉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
JP2023019888A Pending JP2023053254A (ja) 2017-03-17 2023-02-13 筋ジストロフィーを治療するための筋肉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
JP2024229759A Pending JP2025061676A (ja) 2017-03-17 2024-12-26 筋ジストロフィーを治療するための筋肉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

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JP2023019888A Pending JP2023053254A (ja) 2017-03-17 2023-02-13 筋ジストロフィーを治療するための筋肉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
JP2024229759A Pending JP2025061676A (ja) 2017-03-17 2024-12-26 筋ジストロフィーを治療するための筋肉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

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CN (2) CN110997923B (enExample)
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US11338045B2 (en) 2017-03-17 2022-05-24 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
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AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
IL324506A (en) 2018-06-18 2026-01-01 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector for delivery of muscle-specific microdystrophin for the treatment of muscular dystrophy
IL279789B2 (en) 2018-06-29 2026-04-01 Res Inst Nationwide Childrens Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
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US12630840B2 (en) * 2019-02-02 2026-05-19 University of Pittsburgh—of the Commonwealth System of Higher Education One-step gene therapy for Duchenne muscular dystrophy via gene replacement and anti-inflammation
KR20210133242A (ko) 2019-02-26 2021-11-05 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 B-사르코글리칸의 아데노 관련 바이러스 벡터 전달 및 근이영양증의 치료
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CN119061198A (zh) * 2019-08-29 2024-12-03 美国西门子医学诊断股份有限公司 用于检测aav脱落的试剂和方法
AU2020392243A1 (en) * 2019-11-28 2022-06-16 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
US20230040544A1 (en) * 2019-12-16 2023-02-09 Research Institute At Nationwide Children's Hospital Compositions and methods for restoring and maintaining the dystrophin-associated protein complex (dapc)
US11535868B2 (en) 2020-04-29 2022-12-27 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
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KR20250099412A (ko) * 2020-11-12 2025-07-01 프리시젼 바이오사이언시스 인코포레이티드 디스트로핀 유전자 내의 인식 서열에 대해 특이성을 갖는 조작된 메가뉴클레아제
EP4086276A1 (en) 2021-05-03 2022-11-09 Université d'Aix-Marseille Composition for treating dysferlinopathy
BR112023024078A2 (pt) * 2021-05-17 2024-01-30 Sarepta Therapeutics Inc Produção de vetores de aav recombinantes para o tratamento de distrofia muscular
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
WO2023015304A1 (en) * 2021-08-05 2023-02-09 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
JP2024531138A (ja) * 2021-08-11 2024-08-29 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 筋ジストロフィーを処置するための組成物および方法
US12285497B2 (en) 2021-10-15 2025-04-29 Research Institute At Nationwide Children's Hospital Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
CN114316070B (zh) * 2021-12-29 2022-11-15 上海勉亦生物科技有限公司 用于治疗肌营养不良症的转基因表达盒
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
CN118460618A (zh) * 2022-04-19 2024-08-09 康霖生物科技（杭州）有限公司 一种用于遗传性凝血因子缺乏病治疗的核酸构建体
WO2023248251A1 (en) * 2022-06-24 2023-12-28 Indian Institute Of Technology Kanpur An optimized aav vector for gene therapy of muscular dystrophy
CN115819546B (zh) * 2022-08-19 2025-12-05 成都金唯科生物科技有限公司 表达微缩抗肌营养不良蛋白基因的腺相关病毒载体及其应用
WO2026076414A1 (en) * 2024-10-03 2026-04-09 General Medicines Llc Compositions and methods for modulating expression of therapeutic transgenes
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