AU2018233732B2 - Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy - Google Patents

Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy Download PDF

Info

Publication number
AU2018233732B2
AU2018233732B2 AU2018233732A AU2018233732A AU2018233732B2 AU 2018233732 B2 AU2018233732 B2 AU 2018233732B2 AU 2018233732 A AU2018233732 A AU 2018233732A AU 2018233732 A AU2018233732 A AU 2018233732A AU 2018233732 B2 AU2018233732 B2 AU 2018233732B2
Authority
AU
Australia
Prior art keywords
dystrophin
aav vector
recombinant aav
micro
muscle
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
AU2018233732A
Other languages
English (en)
Other versions
AU2018233732A1 (en
Inventor
Jerry R. MENDELL
Louise RODINO-KLAPAC
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nationwide Childrens Hospital Inc
Original Assignee
Nationwide Childrens Hospital Inc
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 Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of AU2018233732A1 publication Critical patent/AU2018233732A1/en
Application granted granted Critical
Publication of AU2018233732B2 publication Critical patent/AU2018233732B2/en
Priority to AU2024266707A priority Critical patent/AU2024266707B2/en
Priority to AU2025200437A priority patent/AU2025200437A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A61K9/0029—Parenteral nutrition; Parenteral nutrition compositions as drug carriers
    • 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
    • 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
    • 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
    • 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
    • C12N15/11—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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • 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
    • 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
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Nutrition Science (AREA)
  • Dermatology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2018233732A 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy Active AU2018233732B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2024266707A AU2024266707B2 (en) 2017-03-17 2024-11-19 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
AU2025200437A AU2025200437A1 (en) 2017-03-17 2025-01-22 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy

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

Related Child Applications (2)

Application Number Title Priority Date Filing Date
AU2024266707A Division AU2024266707B2 (en) 2017-03-17 2024-11-19 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
AU2025200437A Division AU2025200437A1 (en) 2017-03-17 2025-01-22 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy

Publications (2)

Publication Number Publication Date
AU2018233732A1 AU2018233732A1 (en) 2019-10-03
AU2018233732B2 true AU2018233732B2 (en) 2024-10-24

Family

ID=63523321

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2018233732A Active AU2018233732B2 (en) 2017-03-17 2018-03-16 Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
AU2024266707A Active AU2024266707B2 (en) 2017-03-17 2024-11-19 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
AU2025200437A Pending AU2025200437A1 (en) 2017-03-17 2025-01-22 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2024266707A Active AU2024266707B2 (en) 2017-03-17 2024-11-19 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
AU2025200437A Pending AU2025200437A1 (en) 2017-03-17 2025-01-22 Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy

Country Status (33)

Country Link
US (2) US20200199621A1 (enExample)
EP (2) EP4245852A3 (enExample)
JP (3) JP7676111B2 (enExample)
KR (3) KR102887103B1 (enExample)
CN (2) CN110997923B (enExample)
AR (1) AR111292A1 (enExample)
AU (3) AU2018233732B2 (enExample)
BR (1) BR112019019248A2 (enExample)
CA (1) CA3056638A1 (enExample)
CO (1) CO2019011250A2 (enExample)
CY (1) CY1126080T1 (enExample)
DK (1) DK3596222T5 (enExample)
EA (1) EA201992201A1 (enExample)
ES (1) ES2948233T3 (enExample)
FI (1) FI3596222T3 (enExample)
HR (1) HRP20230522T1 (enExample)
HU (1) HUE062476T2 (enExample)
IL (3) IL311713B2 (enExample)
LT (1) LT3596222T (enExample)
MA (1) MA52112B1 (enExample)
MD (1) MD3596222T2 (enExample)
MX (3) MX2019011046A (enExample)
MY (1) MY203547A (enExample)
PL (1) PL3596222T3 (enExample)
PT (1) PT3596222T (enExample)
RS (1) RS64299B1 (enExample)
SA (1) SA519410146B1 (enExample)
SG (1) SG11201908575SA (enExample)
SI (1) SI3596222T1 (enExample)
SM (1) SMT202300201T1 (enExample)
TW (2) TW202525835A (enExample)
WO (1) WO2018170408A1 (enExample)
ZA (1) ZA201906251B (enExample)

Families Citing this family (27)

* 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
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
AU2018351528B2 (en) 2017-10-20 2025-02-27 Research Institute At Nationwide Children's Hospital Methods and materials for NT-3 gene therapy
JP7361701B2 (ja) 2018-01-31 2023-10-16 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 肢帯型筋ジストロフィー２ｃ型のための遺伝子治療
JP7646362B2 (ja) 2018-06-18 2025-03-17 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デリバリー
WO2020006458A1 (en) 2018-06-29 2020-01-02 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
EP3897689B1 (en) * 2018-12-21 2025-02-05 Northwestern University Use of annexins in preventing and treating muscle membrane injury
US20220136005A1 (en) * 2019-02-02 2022-05-05 University Of Pittsburgh - Of The Commonwealth System Of Higher Education One-step gene therapy for duchenne muscular dystrophy via gene replacement and anti-inflammation
EP3930765A1 (en) 2019-02-26 2022-01-05 Research Institute at Nationwide Children's Hospital Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy
EP3976806A1 (en) * 2019-05-30 2022-04-06 Solidus Biosciences, Inc. Recombinant herpesvirales vector
SI4017871T1 (sl) 2019-08-21 2024-06-28 Research Institute At Nationwide Children's Hospital Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije
CN114302968A (zh) * 2019-08-29 2022-04-08 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
US20230270886A1 (en) * 2019-11-28 2023-08-31 Regenxbio Inc. Microdystrophin gene therapy constructs and uses thereof
EP4117783A4 (en) * 2019-12-16 2024-08-21 Research Institute at Nationwide Children's Hospital COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
IL297753A (en) 2020-04-29 2022-12-01 Bristol Myers Squibb Co Minimized dystrophins with spectrin fusion domains and uses thereof
WO2022029543A1 (en) * 2020-08-06 2022-02-10 Intas Pharmaceuticals Ltd. Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
EP4244342A1 (en) * 2020-11-12 2023-09-20 Precision BioSciences, Inc. Engineered meganucleases having specificity for recognition sequences in the dystrophin gene
EP4086276A1 (en) 2021-05-03 2022-11-09 Université d'Aix-Marseille Composition for treating dysferlinopathy
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
AU2022325165A1 (en) * 2021-08-05 2024-01-18 Insmed Incorporated Adeno-associated virus particles and methods of use thereof
WO2023019168A1 (en) * 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
JP2023059858A (ja) 2021-10-15 2023-04-27 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 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
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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015197232A1 (en) * 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies

Family Cites Families (31)

* 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
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
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
CA2207927A1 (en) 1994-12-06 1996-06-13 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
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved aav vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
CA2264483C (en) 1996-09-06 2011-03-22 James M. Wilson Method for recombinant adeno-associated virus-directed gene therapy
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP1944362B1 (en) 1997-09-05 2015-10-21 Genzyme Corporation Methods for generating high titer helper-free preparations of recombinant AAV vectors
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
JP2004514407A (ja) 2000-04-28 2004-05-20 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 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
EP1453547B1 (en) 2001-12-17 2016-09-21 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor
US20080044393A1 (en) * 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
HUE028662T2 (en) * 2007-10-26 2016-12-28 Academisch Ziekenhuis Leiden Preparations and methods for controlling muscle disorders
JP2012524540A (ja) * 2009-04-24 2012-10-18 プロセンサ テクノロジーズ ビー．ブイ． Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド
EP4488370A2 (en) 2011-07-25 2025-01-08 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expresssion of dux4
US20150045416A1 (en) * 2012-01-05 2015-02-12 Hadasit Medical Research Services & Development Ltd. Methods and Compositions for Gene Delivery
JP6373763B2 (ja) * 2012-02-17 2018-08-22 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
WO2014172669A1 (en) * 2013-04-20 2014-10-23 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus delivery of exon 2-targeted u7snrna polynucleotide constructs
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
SG11201605906UA (en) * 2014-01-21 2016-08-30 Univ Bruxelles 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
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
GB201507842D0 (en) * 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015197232A1 (en) * 2014-06-27 2015-12-30 Genethon Efficient systemic treatment of dystrophic muscle pathologies

Also Published As

Publication number Publication date
IL311713B1 (en) 2025-06-01
EP4245852A3 (en) 2023-11-22
JP7676111B2 (ja) 2025-05-14
PT3596222T (pt) 2023-06-20
SG11201908575SA (en) 2019-10-30
IL269391A (en) 2019-11-28
TWI878204B (zh) 2025-04-01
RS64299B1 (sr) 2023-07-31
KR20250163423A (ko) 2025-11-20
AR111292A1 (es) 2019-06-26
WO2018170408A1 (en) 2018-09-20
KR102887103B1 (ko) 2025-11-18
MA52112B1 (fr) 2023-08-31
DK3596222T5 (da) 2024-09-02
IL311713B2 (en) 2025-10-01
IL269391B2 (en) 2024-09-01
TW202525835A (zh) 2025-07-01
EP4245852A2 (en) 2023-09-20
EP3596222A4 (en) 2021-01-06
KR20240112964A (ko) 2024-07-19
TW201840850A (zh) 2018-11-16
IL269391B1 (en) 2024-05-01
CY1126080T1 (el) 2023-11-15
AU2018233732A1 (en) 2019-10-03
CA3056638A1 (en) 2018-09-20
SA519410146B1 (ar) 2024-11-10
AU2024266707A1 (en) 2025-01-30
CN110997923A (zh) 2020-04-10
JP2025061676A (ja) 2025-04-11
MD3596222T2 (ro) 2023-08-31
AU2024266707B2 (en) 2025-11-27
MX2019011046A (es) 2019-10-17
BR112019019248A2 (pt) 2020-04-28
IL320791A (en) 2025-07-01
CN118497275A (zh) 2024-08-16
LT3596222T (lt) 2023-07-25
CO2019011250A2 (es) 2020-02-28
US20220364117A1 (en) 2022-11-17
PL3596222T3 (pl) 2023-10-09
IL311713A (en) 2024-05-01
MY203547A (en) 2024-07-02
HRP20230522T1 (hr) 2023-08-04
MX2025007402A (es) 2025-07-01
JP2020513811A (ja) 2020-05-21
EP3596222A1 (en) 2020-01-22
AU2025200437A1 (en) 2025-02-13
JP2023053254A (ja) 2023-04-12
EP3596222B1 (en) 2023-04-12
KR102683682B1 (ko) 2024-07-09
ES2948233T3 (es) 2023-09-06
KR20190130591A (ko) 2019-11-22
MX2025007401A (es) 2025-08-01
SI3596222T1 (sl) 2024-05-31
MA52112A (fr) 2020-01-22
SMT202300201T1 (it) 2023-09-06
CN110997923B (zh) 2024-01-02
DK3596222T3 (da) 2023-06-19
FI3596222T3 (fi) 2023-06-08
ZA201906251B (en) 2024-01-31
EA201992201A1 (ru) 2020-03-19
US20200199621A1 (en) 2020-06-25
HUE062476T2 (hu) 2023-11-28

Similar Documents

Publication Publication Date Title
AU2024266707B2 (en) Adeno-Associated Virus Vector Delivery Of Muscle Specific Micro-Dystrophin To Treat Muscular Dystrophy
AU2023201093B2 (en) Adeno-Associated Virus Vector Delivery Of Micro-Dystrophin To Treat Muscular Dystrophy
KR20250092292A (ko) 근육 이영양증을 치료하기 위한 근육 특이적 마이크로-디스트로핀의 아데노-연관 바이러스 벡터 전달
US20210393801A1 (en) Adeno-Associated Virus Vector Delivery for Muscular Dystrophies
WO2019012336A2 (en) ADENO-ASSOCIATED VIRAL VECTOR DELIVERY OF A MICRO-DYSTROPHIN FRAGMENT FOR TREATING MUSCLE DYSTROPHY
KR20240021765A (ko) 근이영양증 치료를 위한 재조합 aav 벡터의 제조
US20240366789A1 (en) Aav-ids vectors for treatment of mucopolysaccharidosis ii
CN117642509A (zh) 产生用于治疗肌营养不良的重组aav载体

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)