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

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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
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muscle
aav
muscular dystrophy
recombinant aav
aav vector
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JP2020510447A (ja
JP2020510447A5 (OSRAM
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ハンス ロックミュラー，
オリバー ミューラー，
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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
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    • 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
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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
    • 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

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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
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  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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)

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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

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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

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JP2020510447A JP2020510447A (ja) 2020-04-09
JP2020510447A5 JP2020510447A5 (OSRAM) 2021-05-13
JP7162021B2 true JP7162021B2 (ja) 2022-10-27

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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

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US (3) US11338045B2 (OSRAM)
EP (1) EP3596112A2 (OSRAM)
JP (2) JP7162021B2 (OSRAM)
MA (1) MA47800A (OSRAM)
WO (1) WO2019012336A2 (OSRAM)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1257955A1 (zh) 2015-11-16 2019-11-01 The Research Institute At Nationwide Children's Hospital 用於治疗肌联蛋白类肌病和其他肌联蛋白病变的材料和方法
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
KR102824021B1 (ko) 2018-01-31 2025-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
US12491265B2 (en) 2018-06-18 2025-12-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
CN120400170A (zh) 2019-08-21 2025-08-01 全国儿童医院研究所 Α-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
KR20220105643A (ko) * 2019-10-25 2022-07-27 오덴테스 테라퓨틱스, 인크. 글리코겐 축적 장애를 치료하기 위한 조성물 및 방법
JP2023509118A (ja) * 2019-12-24 2023-03-07 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 調節性核酸配列
BR112022021635A2 (pt) 2020-04-29 2022-12-06 Bristol Myers Squibb Co Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas
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
WO2023135316A1 (en) * 2022-01-17 2023-07-20 Dinaqor Ag Gene therapy composition and treatment for 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
JPH09509564A (ja) 1993-11-09 1997-09-30 ターゲテッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成
WO1995013392A1 (en) 1993-11-09 1995-05-18 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
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
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
JPH11514853A (ja) 1995-09-08 1999-12-21 ジエンザイム コーポレイション 遺伝子治療のための改良されたａａｖベクター
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
WO1998009657A2 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Method for recombinant adeno-associated virus-directed gene therapy
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
CA2682108C (en) 1997-09-05 2013-12-24 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
CA2321837C (en) 1998-02-03 2011-08-16 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
WO2001083692A2 (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
CA2407309C (en) 2000-04-28 2011-08-02 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
WO2002006495A2 (en) 2000-07-14 2002-01-24 The Regents Of The University Of Michigan Mutant muscle-specific enhancers
AU2001296600A1 (en) 2000-10-06 2002-04-15 Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US20060194265A1 (en) 2001-10-23 2006-08-31 Morris David W Novel therapeutic targets in cancer
JP4810062B2 (ja) 2001-12-17 2011-11-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア アデノ随伴ウイルス（ａａｖ）血清型８の配列
DK1519714T3 (da) 2002-06-28 2011-01-31 Protiva Biotherapeutics Inc Fremgangsmåde og apparat til fremstilling af liposomer
US20050064595A1 (en) 2003-07-16 2005-03-24 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
AU2004272646B2 (en) 2003-09-15 2011-11-24 Arbutus Biopharma Corporation Polyethyleneglycol-modified lipid compounds and uses thereof
EP2295604B1 (en) 2004-02-09 2015-04-08 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
CA2569645C (en) 2004-06-07 2014-10-28 Protiva Biotherapeutics, Inc. Cationic lipids and methods of use
JP4796062B2 (ja) 2004-06-07 2011-10-19 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 脂質封入干渉ｒｎａ
US20080044393A1 (en) 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
WO2007012191A1 (en) 2005-07-27 2007-02-01 Protiva Biotherapeutics, Inc. Systems and methods for manufacturing liposomes
EP1986697B1 (en) 2006-02-17 2016-06-29 GE Healthcare Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
WO2008066965A2 (en) 2006-06-23 2008-06-05 The Regents Of The University Of California Articles comprising large-surface-area bio-compatible materials and methods for making and using them
CN101711164B (zh) 2007-01-18 2014-06-04 密苏里-哥伦比亚大学 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因
WO2009018493A1 (en) 2007-07-31 2009-02-05 The Board Of Regents Of The University Of Texas System A micro-rna family that modulates fibrosis and uses thereof
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
CA2709875C (en) 2008-01-02 2019-07-16 Tekmira Pharmaceuticals Corporation Improved 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.
CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel 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
WO2010054406A1 (en) 2008-11-10 2010-05-14 Alnylam Pharmaceuticals, Inc. Novel lipids and compositions for the delivery of therapeutics
WO2010071454A1 (en) 2008-12-17 2010-06-24 Auckland Uniservices Limited Adeno-associated viral vectors and uses thereof
CA2759899A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
JP5766188B2 (ja) 2009-07-01 2015-08-19 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 固形腫瘍に治療剤を送達するための脂質製剤
US8569256B2 (en) 2009-07-01 2013-10-29 Protiva Biotherapeutics, Inc. Cationic lipids and methods for the delivery of therapeutic agents
WO2011004395A1 (en) 2009-07-06 2011-01-13 Intas Biopharmaceuticals Limited Microemulsion formulation for biologicals
WO2011066651A1 (en) 2009-12-01 2011-06-09 Protiva Biotherapeutics, Inc. Snalp formulations containing antioxidants
EP2509636B1 (en) 2009-12-07 2017-07-19 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
NZ600725A (en) 2009-12-18 2015-08-28 Univ British Colombia Methods and compositions for delivery of nucleic acids
CA2800497C (en) 2010-05-10 2019-03-12 The Regents Of The University Of California Methods for preparing ratiometric combinatorial nanoparticules
JP2013527856A (ja) 2010-05-12 2013-07-04 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 陽イオン性脂質およびその使用方法
SG186085A1 (en) 2010-06-03 2013-01-30 Alnylam Pharmaceuticals Inc Biodegradable lipids for the delivery of active agents
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. 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
US9999673B2 (en) 2011-01-11 2018-06-19 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
US9763891B2 (en) 2011-07-22 2017-09-19 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
CA3106285A1 (en) 2011-07-25 2013-01-31 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013049328A1 (en) 2011-09-27 2013-04-04 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
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
CA2856737C (en) 2011-12-07 2023-09-26 Alnylam Pharmaceuticals, Inc. Branched alkyl and cycloalkyl terminated biodegradable lipids for the delivery of active agents
HK1200089A1 (en) 2011-12-07 2015-07-31 Alnylam Pharmaceuticals, Inc. Biodegradable lipids for the delivery of active agents
WO2013086373A1 (en) 2011-12-07 2013-06-13 Alnylam Pharmaceuticals, Inc. Lipids for the delivery of active agents
SI2791160T1 (sl) 2011-12-16 2022-07-29 Modernatx, Inc. Sestave modificirane MRNA
US20150045416A1 (en) 2012-01-05 2015-02-12 Hadasit Medical Research Services & Development Ltd. Methods and Compositions for Gene Delivery
CN104520428B (zh) 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
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
CN109652385A (zh) 2013-04-20 2019-04-19 全国儿童医院研究所 外显子2靶向U7snRNA多核苷酸构建体的重组腺相关病毒递送
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
JP6202701B2 (ja) 2014-03-21 2017-09-27 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プログラム
US9439924B2 (en) 2014-04-09 2016-09-13 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
IL298516B2 (en) 2014-06-25 2025-03-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle compositions for nucleic acid delivery
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
CA2972811A1 (en) 2015-01-16 2016-07-21 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
SI3313829T1 (sl) 2015-06-29 2024-09-30 Acuitas Therapeutics Inc. Lipidi in formulacije lipidnih nanodelcev za dostavo nukleinskih kislin
US10980897B2 (en) 2015-09-17 2021-04-20 Research Institute At Nationwide Children's Hospital Methods and materials for GALGT2 gene therapy
RS63986B1 (sr) 2015-10-28 2023-03-31 Acuitas Therapeutics Inc Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina
IL259178B (en) 2015-11-12 2022-08-01 Res Inst Nationwide Childrens Hospital A vector encoding ano5 for the treatment of muscular dystrophy
EP3397613A1 (en) 2015-12-30 2018-11-07 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
CA3021057A1 (en) 2016-04-15 2017-10-19 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
CA2971303A1 (en) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use
IL311713B2 (en) 2017-03-17 2025-10-01 Res Inst Nationwide Childrens Hospital Muscle-specific delivery of microdystrophin using an adeno-associated virus vector for the treatment of muscular dystrophy
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
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
JP7213238B2 (ja) 2017-10-18 2023-01-26 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 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
WO2019089828A1 (en) 2017-10-31 2019-05-09 Acuitas Therapeutics, Inc. Lamellar lipid nanoparticles
WO2019118806A1 (en) 2017-12-14 2019-06-20 Solid Biosciences Inc. Non-viral production and delivery of genes
KR102824021B1 (ko) 2018-01-31 2025-06-24 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 2c형 지대근이영양증에 대한 유전자 치료
US12318456B2 (en) 2018-03-10 2025-06-03 Koc Universitesi Therapeutic nanoparticles containing argonaute for microRNA delivery and compositions and methods using same
US20210139550A1 (en) 2018-04-03 2021-05-13 Curators Of The University Of Missouri Hinges 1 and/or 4 modified dystrophins for dystrophinopathy therapy
US12152242B2 (en) 2018-04-23 2024-11-26 The Curators Of The University Of Missouri CRISPR therapy
US12491265B2 (en) 2018-06-18 2025-12-09 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
MX2020014119A (es) 2018-06-29 2021-06-18 Res Inst Nationwide Childrens Hospital Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a.
WO2020081938A1 (en) 2018-10-18 2020-04-23 Acuitas Therapeutics, Inc. Lipids for lipid nanoparticle delivery of active agents
AU2019395388A1 (en) 2018-12-12 2021-07-29 Solid Biosciences Inc. Combination therapy for treating muscular dystrophy
MX2021010356A (es) 2019-02-26 2022-03-04 Res Inst Nationwide Childrens Hospital SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR.
CN120400170A (zh) 2019-08-21 2025-08-01 全国儿童医院研究所 Α-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
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

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