JP7162021B2 - 筋ジストロフィーを治療するためのマイクロジストロフィン断片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マイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 Download PDF

Info

Publication number
JP7162021B2
JP7162021B2 JP2019571814A JP2019571814A JP7162021B2 JP 7162021 B2 JP7162021 B2 JP 7162021B2 JP 2019571814 A JP2019571814 A JP 2019571814A JP 2019571814 A JP2019571814 A JP 2019571814A JP 7162021 B2 JP7162021 B2 JP 7162021B2
Authority
JP
Japan
Prior art keywords
muscle
aav
muscular dystrophy
recombinant aav
aav vector
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
JP2019571814A
Other languages
English (en)
Japanese (ja)
Other versions
JP2020510447A5 (enExample
JP2020510447A (ja
Inventor
ハンス ロックミュラー，
オリバー ミューラー，
Original Assignee
ニューカッスル ユニバーシティ
ユニバーシティ オブ ハイデルベルク
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ニューカッスル ユニバーシティ, ユニバーシティ オブ ハイデルベルク filed Critical ニューカッスル ユニバーシティ
Publication of JP2020510447A publication Critical patent/JP2020510447A/ja
Publication of JP2020510447A5 publication Critical patent/JP2020510447A5/ja
Priority to JP2022166267A priority Critical patent/JP7493566B2/ja
Application granted granted Critical
Publication of JP7162021B2 publication Critical patent/JP7162021B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • 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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2019571814A 2017-03-17 2018-03-16 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 Active JP7162021B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022166267A JP7493566B2 (ja) 2017-03-17 2022-10-17 筋ジストロフィーを治療するためのマイクロジストロフィン断片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
US201762473255P 2017-03-17 2017-03-17
US62/473,255 2017-03-17
PCT/IB2018/001201 WO2019012336A2 (en) 2017-03-17 2018-03-16 ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022166267A Division JP7493566B2 (ja) 2017-03-17 2022-10-17 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達

Publications (3)

Publication Number Publication Date
JP2020510447A JP2020510447A (ja) 2020-04-09
JP2020510447A5 JP2020510447A5 (enExample) 2021-05-13
JP7162021B2 true JP7162021B2 (ja) 2022-10-27

Family

ID=64564915

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2019571814A Active JP7162021B2 (ja) 2017-03-17 2018-03-16 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
JP2022166267A Active JP7493566B2 (ja) 2017-03-17 2022-10-17 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2022166267A Active JP7493566B2 (ja) 2017-03-17 2022-10-17 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達

Country Status (5)

Country Link
US (3) US11338045B2 (enExample)
EP (1) EP3596112A2 (enExample)
JP (2) JP7162021B2 (enExample)
MA (1) MA47800A (enExample)
WO (1) WO2019012336A2 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102877920B1 (ko) 2015-11-16 2025-10-30 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 티틴-기반 근증 및 다른 티틴성병증의 치료를 위한 물질 및 방법
JP7162021B2 (ja) * 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
SG11202006722RA (en) 2018-01-31 2020-08-28 Res Inst Nationwide Childrens Hospital Gene therapy for limb-girdle muscular dystrophy type 2c
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
CA3131390A1 (en) 2019-02-26 2020-09-03 Research Institute Of Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
HRP20240505T1 (hr) 2019-08-21 2024-07-05 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije
KR20220105643A (ko) * 2019-10-25 2022-07-27 오덴테스 테라퓨틱스, 인크. 글리코겐 축적 장애를 치료하기 위한 조성물 및 방법
CN115151646A (zh) * 2019-12-24 2022-10-04 阿斯克肋匹奥生物制药公司 调节核酸序列
MX2022013477A (es) 2020-04-29 2022-11-30 Bristol Myers Squibb Co Distrofinas miniaturizadas con dominios de fusion de espectrina y usos de las mismas.
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
CA3248878A1 (en) * 2022-01-17 2023-07-20 Dinaqor Ag GENE THERAPY COMPOSITION AND TREATMENT OF DYSTROPHIN-RELATED CARDIOMYOPATHY
US20250288641A1 (en) * 2022-04-27 2025-09-18 The Curators Of The University Of Missouri Micro-dystrophin for heart protection
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
AR133921A1 (es) 2023-09-25 2025-11-12 Sarepta Therapeutics Inc Distrofinas humanas adaptadas con mayor afinidad de unión a la actina para el tratamiento de las distrofias musculares
NL2037199B1 (en) 2024-03-07 2025-09-15 C2Ca Tech B V Method for obtaining a cement constituent from a concrete element

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015197869A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic pathologies

Family Cites Families (116)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
EP0733103B1 (en) 1993-11-09 2004-03-03 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
ATE272123T1 (de) 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
EP0796339A1 (en) 1994-12-06 1997-09-24 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
JPH11511326A (ja) 1995-08-30 1999-10-05 ジエンザイム コーポレイション アデノウィルスおよびａａｖの精製
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5795872A (en) 1995-09-19 1998-08-18 Pharmadigm, Inc. DNA construct for immunization
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
ATE465267T1 (de) 1996-09-06 2010-05-15 Univ Pennsylvania Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen
US5965542A (en) 1997-03-18 1999-10-12 Inex Pharmaceuticals Corp. Use of temperature to control the size of cationic liposome/plasmid DNA complexes
EP1004603B1 (en) 1997-08-15 2003-01-15 Chisso Corporation Polydisperse propylene polymer and process for producing the same
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999011764A2 (en) 1997-09-05 1999-03-11 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of recombinant aav vectors
US6410328B1 (en) 1998-02-03 2002-06-25 Protiva Biotherapeutics Inc. Sensitizing cells to compounds using lipid-mediated gene and compound delivery
WO1999039741A2 (en) 1998-02-03 1999-08-12 Inex Pharmaceuticals Corporation Systemic delivery of serum stable plasmid lipid particles for cancer therapy
JPH11318467A (ja) * 1998-05-08 1999-11-24 Japan Science & Technology Corp 短縮型ジストロフィン
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6211140B1 (en) 1999-07-26 2001-04-03 The Procter & Gamble Company Cationic charge boosting systems
CA2406743A1 (en) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
CN100422320C (zh) 2000-04-28 2008-10-01 肖啸 编码抗肌萎缩蛋白小基因的dna序列及其使用方法
ATE373095T1 (de) 2000-07-14 2007-09-15 Univ Michigan Mutierte muskelspezifische enhancer
DE60134786D1 (de) 2000-10-06 2008-08-21 Univ Michigan Mini-dystrophin nukleinsäure- und peptidsequenzen
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US20060194265A1 (en) 2001-10-23 2006-08-31 Morris David W Novel therapeutic targets in cancer
ES2602352T3 (es) 2001-12-17 2017-02-20 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
US6927663B2 (en) 2003-07-23 2005-08-09 Cardiac Pacemakers, Inc. Flyback transformer wire attach method to printed circuit board
EP1664316B1 (en) 2003-09-15 2012-08-29 Protiva Biotherapeutics Inc. Polyethyleneglycol-modified lipid compounds and uses thereof
CA2554818A1 (en) 2004-02-09 2005-08-25 Thomas Jefferson University Diagnosis and treatment of cancers with microrna located in or near cancer-associated chromosomal features
US20070053845A1 (en) 2004-03-02 2007-03-08 Shiladitya Sengupta Nanocell drug delivery system
JP4764426B2 (ja) 2004-06-07 2011-09-07 プロチバ バイオセラピューティクス インコーポレイティッド カチオン性脂質および使用方法
AU2005252273B2 (en) 2004-06-07 2011-04-28 Arbutus Biopharma Corporation Lipid encapsulated interfering RNA
US20080044393A1 (en) 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
JP5639338B2 (ja) 2005-07-27 2014-12-10 プロチバ バイオセラピューティクス インコーポレイティッド リポソームの製造システムおよび製造方法
WO2007095387A2 (en) 2006-02-17 2007-08-23 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
US9149564B2 (en) 2006-06-23 2015-10-06 The Regents Of The University Of California Articles comprising large-surface-area bio-compatible materials and methods for making and using them
JP5575486B2 (ja) 2007-01-18 2014-08-20 ユニヴァーシティ オブ ミズーリー−コロンビア 筋鞘にｎＮＯＳを回復させる合成ミニ／ミクロジストロフィン遺伝子
ES2621161T3 (es) 2007-07-31 2017-07-03 The Board Of Regents Of The University Of Texas System Familia de micro-ARN que modula la fibrosis y usos de la misma
PT2203173E (pt) 2007-10-26 2016-03-15 Academisch Ziekenhuis Leiden Resumo
US20110117125A1 (en) 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
FR2927845B1 (fr) 2008-02-21 2010-03-19 Michelin & Cie Pneumatique pour roulage a plat pourvu d'une armature supplementaire de flanc.
US8058069B2 (en) 2008-04-15 2011-11-15 Protiva Biotherapeutics, Inc. Lipid formulations for nucleic acid delivery
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
CN105152939A (zh) 2008-11-10 2015-12-16 阿尔尼拉姆医药品有限公司 用于递送治疗剂的脂质和组合物
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
NZ595955A (en) 2009-04-24 2012-10-26 Prosensa Technologies Bv Oligonucleotide comprising an inosine for treating dmd
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
WO2011004395A1 (en) 2009-07-06 2011-01-13 Intas Biopharmaceuticals Limited Microemulsion formulation for biologicals
EP2506879A4 (en) 2009-12-01 2014-03-19 Protiva Biotherapeutics Inc PREPARATIONS OF SNALP CONTAINING ANTIOXIDANTS
US9687550B2 (en) 2009-12-07 2017-06-27 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
CA2784568A1 (en) 2009-12-18 2011-06-23 Martin A. Maier Lipid particles for delivery of nucleic acids
RU2012151134A (ru) 2010-05-10 2014-06-20 Дзе Реджентс Оф Дзе Юниверсити Оф Калифорния Логометрическая комбинаторная доставка лекарственных средств
US20130123338A1 (en) 2010-05-12 2013-05-16 Protiva Biotherapeutics, Inc. Novel cationic lipids and methods of use thereof
NZ605079A (en) 2010-06-03 2015-08-28 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
US8466122B2 (en) 2010-09-17 2013-06-18 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
AU2012207606B2 (en) 2011-01-11 2017-02-23 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
WO2013016126A1 (en) 2011-07-22 2013-01-31 The General Hospital Corporation Therapeutic nanoparticles and methods of use thereof
WO2013016058A1 (en) 2011-07-22 2013-01-31 Merck Sharp & Dohme Corp. Novel bis-nitrogen containing cationic lipids for oligonucleotide delivery
ES2990023T3 (es) 2011-07-25 2024-11-28 Nationwide Childrens Hospital Inc Productos de virus recombinantes y métodos para inhibición de la expresión de DUX4
EP3456317B1 (en) 2011-09-27 2025-09-24 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
US8762704B2 (en) 2011-09-29 2014-06-24 Apple Inc. Customized content for electronic devices
WO2013075008A1 (en) 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Aav dual vector systems for gene therapy
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
CA2856742A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
JP6305343B2 (ja) 2011-12-07 2018-04-04 アルニラム・ファーマシューティカルズ・インコーポレーテッド 活性薬剤の送達のための分岐アルキルおよびシクロアルキルを末端とする生分解性脂質
KR20140102759A (ko) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
JP2015503924A (ja) 2012-01-05 2015-02-05 ハダシット メディカル リサーチ サービシーズ アンド ディベロップメント リミテッド 遺伝子導入のための方法及び組成物
BR112014020325A2 (pt) 2012-02-17 2017-08-08 Childrens Hospital Philadelphia composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
US9415109B2 (en) 2012-07-06 2016-08-16 Alnylam Pharmaceuticals, Inc. Stable non-aggregating nucleic acid lipid particle formulations
US9624282B2 (en) 2012-11-26 2017-04-18 The Curators Of The University Of Missouri Microdystrophin peptides and methods for treating muscular dystrophy using the same
MX388221B (es) 2013-04-20 2025-03-19 Res Institute At Nationwide Children´S Hospital ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2.
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
GB201400598D0 (en) 2014-01-14 2014-03-05 Univ Glasgow Materials and methods for modulation of tendon healing
CA2936726C (en) 2014-01-21 2024-04-30 Vrije Universiteit Brussel Muscle-specific nucleic acid regulatory elements and methods and use thereof
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
WO2015157306A1 (en) 2014-04-09 2015-10-15 University Of Houston Therapeutic mirnas for treating heart and skeletal muscle diseases
WO2015161255A1 (en) 2014-04-18 2015-10-22 Board Of Regents Of The Nevada System Of Higher Education, On Behalf Of The University Of Nevada, Reno Methods for enhancing or decreasing the levels of mir124 and mir29 in subjects with muscular dystrophy
HRP20221536T1 (hr) 2014-06-25 2023-02-17 Acuitas Therapeutics Inc. Novi lipidi i formulacije lipidnih nanočestica za isporuku nukleinskih kiselina
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
AU2016206518B2 (en) * 2015-01-16 2020-03-05 University Of Washington Novel micro-dystrophins and related methods of use
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
CA2990202A1 (en) 2015-06-29 2017-01-05 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
IL258005B2 (en) 2015-09-17 2025-01-01 Res Inst Nationwide Childrens Hospital Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2
PT3368507T (pt) 2015-10-28 2023-02-07 Acuitas Therapeutics Inc Novos lípidos e formulações de nanopartículas lipídicas para distribuição de ácidos nucleicos
IL259178B (en) 2015-11-12 2022-08-01 Res Inst Nationwide Childrens Hospital A vector encoding ano5 for the treatment of muscular dystrophy
US20190022247A1 (en) 2015-12-30 2019-01-24 Acuitas Therapeutics, Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
WO2017165859A1 (en) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Modified viral capsid proteins
MA45477A (fr) 2016-04-15 2019-02-20 Res Inst Nationwide Childrens Hospital Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
HUE066442T2 (hu) 2016-04-15 2024-08-28 Res Institute At Nationwide ß-szarkoglikán és mikroRNS-29 adenoasszociáltvírus-vektorral történõ bejuttatása és izomdisztrófia kezelése
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
WO2018170408A1 (en) 2017-03-17 2018-09-20 Research Institute At Nationwide Children's Hospital, Inc. Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
JP7162021B2 (ja) * 2017-03-17 2022-10-27 ニューカッスル ユニバーシティ 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
CN107369008A (zh) 2017-07-17 2017-11-21 北京京东金融科技控股有限公司 用于提高票据交易安全性的管理方法、装置及系统
US11534501B2 (en) 2017-10-18 2022-12-27 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
TW201936201A (zh) 2017-12-14 2019-09-16 美商堅固生物科技公司 基因之非病毒生產及遞送
SG11202006722RA (en) 2018-01-31 2020-08-28 Res Inst Nationwide Childrens Hospital Gene therapy for limb-girdle muscular dystrophy type 2c
WO2019177550A1 (en) 2018-03-10 2019-09-19 Koc Universitesi Therapeutic nanoparticles containing argonaute for microrna delivery and compositions and methods using same
JP2021520196A (ja) 2018-04-03 2021-08-19 ザ・キュレイターズ・オブ・ザ・ユニバーシティー・オブ・ミズーリＴｈｅ Ｃｕｒａｔｏｒｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍｉｓｓｏｕｒｉ ジストロフィン異常症（ｄｙｓｔｒｏｐｈｉｎｏｐａｔｈｙ）治療のためのヒンジ１および／または４の改変ジストロフィン
US12152242B2 (en) 2018-04-23 2024-11-26 The Curators Of The University Of Missouri CRISPR therapy
MY208145A (en) 2018-06-18 2025-04-18 Res Inst Nationwide Childrens Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
AU2019361129A1 (en) 2018-10-18 2021-05-20 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
US20220031865A1 (en) 2018-12-12 2022-02-03 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
CA3131390A1 (en) 2019-02-26 2020-09-03 Research Institute Of Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
HRP20240505T1 (hr) 2019-08-21 2024-07-05 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana vektorom povezanim s adenovirusom i liječenje mišićne distrofije
US12391745B2 (en) 2020-06-15 2025-08-19 Sarepta Therapeutics, Inc. Adeno-associated virus antibodies and fragments thereof

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015197869A1 (en) 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic pathologies

Also Published As

Publication number Publication date
US11338045B2 (en) 2022-05-24
US20230049491A1 (en) 2023-02-16
WO2019012336A2 (en) 2019-01-17
JP7493566B2 (ja) 2024-05-31
JP2020510447A (ja) 2020-04-09
WO2019012336A3 (en) 2019-03-07
US12257317B2 (en) 2025-03-25
US20250360225A1 (en) 2025-11-27
JP2022185154A (ja) 2022-12-13
EP3596112A2 (en) 2020-01-22
US20200078473A1 (en) 2020-03-12
MA47800A (fr) 2020-01-22

Similar Documents

Publication Publication Date Title
JP7493566B2 (ja) 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達
US20220364117A1 (en) Adeno-Associated Virus Vector Delivery of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
US12491265B2 (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
US20230302157A1 (en) Adeno-Associated Virus Vector Delivery of Muscle Specific Micro-Dystrophin to Treat Muscular Dystrophy
JP2025118741A (ja) 筋ジストロフィーを治療するための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
KR20240095165A (ko) 근이영양증의 치료
JP7781057B2 (ja) Ｉｒｆ２ｂｐｌ遺伝子の変異に関連する障害の治療のための材料および方法
JP2024545507A (ja) Ａａｖベクターを用いたトロポニンｃ（ｔｎｎｃ１）遺伝子治療
JP2023059858A (ja) 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
JP7534961B2 (ja) ジストログリカノパチーおよびラミニン欠損筋ジストロフィーを治療するための組換えアデノ随伴ウイルス生成物および方法
HK40099903A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40046539B (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40046539A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40021330A (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
HK40021330B (en) Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20210316

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210330

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20211228

A977 Report on retrieval

Free format text: JAPANESE INTERMEDIATE CODE: A971007

Effective date: 20211228

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220325

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220527

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220628

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220818

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20220913

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20221017

R150 Certificate of patent or registration of utility model

Ref document number: 7162021

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250