[go: up one dir, main page]
More Web Proxy on the site http://driver.im/

MA44683A - Délivrance de vecteur de virus adéno-associé pour traiter la dystrophie musculaire - Google Patents

Délivrance de vecteur de virus adéno-associé pour traiter la dystrophie musculaire

Info

Publication number
MA44683A
MA44683A MA044683A MA44683A MA44683A MA 44683 A MA44683 A MA 44683A MA 044683 A MA044683 A MA 044683A MA 44683 A MA44683 A MA 44683A MA 44683 A MA44683 A MA 44683A
Authority
MA
Morocco
Prior art keywords
adeno
associated virus
virus vector
vector delivery
muscle dystrophy
Prior art date
Application number
MA044683A
Other languages
English (en)
Inventor
Kristin N Heller
Jerry R Mendell
Louise Rodino-Klapac
Original Assignee
Res Inst Nationwide Childrens Hospital
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 Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of MA44683A publication Critical patent/MA44683A/fr

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
    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A61K35/761—Adenovirus
    • 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
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • 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
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C07K14/4708—Duchenne 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N15/65—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression using markers
    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Neurology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Dermatology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA044683A 2016-04-15 2017-04-14 Délivrance de vecteur de virus adéno-associé pour traiter la dystrophie musculaire MA44683A (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662323163P 2016-04-15 2016-04-15
US201762473253P 2017-03-17 2017-03-17

Publications (1)

Publication Number Publication Date
MA44683A true MA44683A (fr) 2019-02-20

Family

ID=60041940

Family Applications (2)

Application Number Title Priority Date Filing Date
MA045477A MA45477A (fr) 2016-04-15 Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
MA044683A MA44683A (fr) 2016-04-15 2017-04-14 Délivrance de vecteur de virus adéno-associé pour traiter la dystrophie musculaire

Family Applications Before (1)

Application Number Title Priority Date Filing Date
MA045477A MA45477A (fr) 2016-04-15 Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire

Country Status (17)

Country Link
US (5) US11298429B2 (fr)
EP (4) EP3442601B1 (fr)
JP (5) JP7189771B2 (fr)
KR (2) KR20240042136A (fr)
CN (2) CN109069672B (fr)
AU (3) AU2017250791B2 (fr)
BR (1) BR112018071182A2 (fr)
CA (2) CA3020996A1 (fr)
CO (1) CO2018012084A2 (fr)
ES (2) ES2985822T3 (fr)
IL (2) IL302200A (fr)
MA (2) MA44683A (fr)
MX (2) MX2018012604A (fr)
MY (1) MY195438A (fr)
SG (1) SG11201808941PA (fr)
WO (3) WO2017181015A1 (fr)
ZA (1) ZA201806746B (fr)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP3596112A2 (fr) 2017-03-17 2020-01-22 Newcastle University Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
AU2018233732B2 (en) * 2017-03-17 2024-10-24 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy
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
BR112020007790A2 (pt) 2017-10-20 2020-10-20 Research Institute At Nationwide Children's Hospital métodos e materiais para terapia genética com nt-3
WO2019178412A1 (fr) * 2018-03-16 2019-09-19 Research Institute At Nationwide Children's Hospital Augmentation de l'administration de gènes spécifiques d'un tissu par modification de capside
WO2019220435A1 (fr) * 2018-05-15 2019-11-21 Ramot At Tel-Aviv University Ltd. Mir126-5p pour le traitement de maladies des motoneurones
CA3104471A1 (fr) * 2018-06-18 2019-12-26 Research Institute At Nationwide Children's Hospital Produits de virus adeno-associes de recombinaison et methodes de traitement de dystroglycanopathies et de dystrophies musculaires deficientes en laminine
CN112823210A (zh) * 2018-06-18 2021-05-18 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送
EP3598978B1 (fr) 2018-07-26 2024-05-29 EXOFIX S.r.l. Exosomes dérivées de cellules fibroadipogéniques progénitrices pour la régénération des muscles dystrophiques
EP3893940A4 (fr) * 2018-12-12 2022-09-28 Solidus Biosciences, Inc. Polythérapie pour le traitement de la dystrophie musculaire
US20220204574A1 (en) * 2019-03-25 2022-06-30 Genethon Production of large-sized quasidystrophins using overlapping aav vectors
WO2021142435A1 (fr) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Polythérapie pour le traitement de la dystrophie musculaire
US20230183740A1 (en) * 2020-01-10 2023-06-15 Solid Biosciences Inc. Viral vector for combination therapy
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.
EP4164668A1 (fr) * 2020-06-15 2023-04-19 Research Institute at Nationwide Children's Hospital Administration de vecteur de virus adéno-associé contre les dystrophies musculaires
CN111733185A (zh) * 2020-06-30 2020-10-02 华南农业大学 通过Mdfi基因编辑来实现动物肉品质改良的方法和应用
US20240350674A1 (en) 2021-08-11 2024-10-24 Solid Biosciences Inc. Treatment of muscular dystrophy
WO2023044441A1 (fr) * 2021-09-16 2023-03-23 University Of Florida Research Foundation, Incorporated Particules de vaa avec répétitions terminales inversées modifiées pour expression génique accrue dans le muscle
EP4215614A1 (fr) 2022-01-24 2023-07-26 Dynacure Thérapie de combinaison pour maladies liées à la dystrophine
WO2024044725A2 (fr) 2022-08-24 2024-02-29 Regenxbio Inc. Virus adéno-associés recombinants et leurs utilisations

Family Cites Families (52)

* 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
DK0728214T3 (da) 1993-11-09 2004-11-29 Targeted Genetics Corp Stabile cellelinjer, der er i stand til at udtrykke det adenoassocierede virusreplikationsgen
CA2176117C (fr) 1993-11-09 2006-01-03 Terence R. Flotte Production de titres eleves de vecteurs d'aav recombinants
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 (fr) 1994-12-06 1996-06-13 Targeted Genetics Corporation Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
US6143548A (en) 1995-08-30 2000-11-07 Genzyme Corporation Chromatographic purification of adeno-associated virus (AAV)
AU715543B2 (en) 1995-09-08 2000-02-03 Genzyme Corporation Improved 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
CA2264483C (fr) 1996-09-06 2011-03-22 James M. Wilson Methode de therapie genique basee sur des virus adeno-associes de recombinaison
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
PT1009808E (pt) 1997-09-05 2013-01-29 Genzyme Corp Métodos para a produção de preparações de elevado título de vectores de aav recombinante isentas de auxiliar
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
ES2330615T3 (es) 2000-04-28 2009-12-14 Asklepios Biopharmaceutical, Inc. Secuencias de adn que codifican para minigenes de distrofina y metodos de uso de las mismas.
CA2406743A1 (fr) 2000-04-28 2001-11-08 The Trustees Of The University Of Pennsylvania Vecteurs aav recombinants dotes de capsides aav5 et vecteurs aav5 pseudotypes dans des capsides heterologues
EP1303617B1 (fr) 2000-07-14 2007-09-12 The Regents Of The University Of Michigan Activateurs specifiques du muscle et mutants
AU2001296600A1 (en) 2000-10-06 2002-04-15 Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
WO2003052051A2 (fr) 2001-12-17 2003-06-26 The Trustees Of The University Of Pennsylvania Sequences du serotype 8 du virus associe a l'adenovirus (aav), vecteurs les contenant et utilisations correspondantes
EP2295604B1 (fr) * 2004-02-09 2015-04-08 Thomas Jefferson University Diagnostic et traitement de cancers à l'aide de microARN présent dans ou au voisinage de caractéristiques chromosomiques associées aux cancers
US20080044393A1 (en) 2004-07-16 2008-02-21 White Robert L Retinal dystrophin transgene and methods of use thereof
EP1986697B1 (fr) 2006-02-17 2016-06-29 GE Healthcare Dharmacon, Inc. Compositions et procédés permettant l'inhibition de silençage de gènes par l'interférence arn
EP2125006B1 (fr) 2007-01-18 2013-10-16 University of Missouri-Columbia Mini/microgenes synthetiques de la dystrophine pour redonner de la no-synthase neuronale au sarcolemme
DK2182969T3 (en) 2007-07-31 2017-02-27 Univ Texas Micro-RNA family that modulates fibrosis and its applications
US8236557B2 (en) * 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
WO2010071454A1 (fr) * 2008-12-17 2010-06-24 Auckland Uniservices Limited Vecteurs viraux adéno-associés et leurs utilisations
EP3290055B1 (fr) 2011-07-25 2024-08-28 Nationwide Children's Hospital, Inc. Virus recombinants et procédés pour l'inhibition de l'expression de dux4
WO2013075008A1 (fr) * 2011-11-16 2013-05-23 University Of Florida Research Foundation Inc. Systèmes de vecteur double aav pour une thérapie génique
US9434928B2 (en) 2011-11-23 2016-09-06 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
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
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
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
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
SG11201605906UA (en) 2014-01-21 2016-08-30 Univ Bruxelles Muscle-specific nucleic acid regulatory elements and methods and use thereof
WO2015141521A1 (fr) 2014-03-21 2015-09-24 株式会社日立国際電気 Appareil de traitement de substrat, procédé de fabrication de dispositif semi-conducteur et support d'enregistrement
WO2015157306A1 (fr) 2014-04-09 2015-10-15 University Of Houston Miarns thérapeutiques pour le traitement de maladies cardiaques et de maladies musculo-squelettiques
US10119140B2 (en) 2014-04-18 2018-11-06 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
EP2960336A1 (fr) 2014-06-27 2015-12-30 Genethon Traitement systémique efficace de pathologies dystrophiques musculaires
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
WO2016057975A2 (fr) * 2014-10-10 2016-04-14 Research Institute At Nationwide Children's Hospital Injections guidées pour transfert de gène par vaa au muscle
NZ734019A (en) 2015-01-16 2023-01-27 Univ 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
CA2998636A1 (fr) 2015-09-17 2017-03-23 Research Institute At Nationwide Children's Hospital Procedes et materiaux pour therapie genique par galgt2
WO2017165859A1 (fr) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Protéines de capside virale modifiées
US11358993B2 (en) 2016-04-15 2022-06-14 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of B-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
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
CA2971303A1 (fr) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Genes de mini-dystrophine optimises et cassettes d'expression et leur utilisation
CN112823210A (zh) 2018-06-18 2021-05-18 全国儿童医院研究所 用于治疗肌营养不良的肌肉特异性微小肌营养不良蛋白的腺相关病毒载体递送

Also Published As

Publication number Publication date
AU2017250790A1 (en) 2018-11-15
KR20240042136A (ko) 2024-04-01
CN118895311A (zh) 2024-11-05
BR112018071182A2 (pt) 2019-04-09
ZA201806746B (en) 2023-12-20
KR20180133486A (ko) 2018-12-14
JP2022020839A (ja) 2022-02-01
JP2019513779A (ja) 2019-05-30
US20190055581A1 (en) 2019-02-21
JP2019513393A (ja) 2019-05-30
EP3442601B1 (fr) 2024-07-03
JP7431789B2 (ja) 2024-02-15
EP3442602A4 (fr) 2019-11-27
JP2024023439A (ja) 2024-02-21
SG11201808941PA (en) 2018-11-29
CA3020996A1 (fr) 2017-10-19
CO2018012084A2 (es) 2019-02-08
AU2017250790B2 (en) 2024-06-20
CA3020999A1 (fr) 2017-10-19
MA45477A (fr) 2019-02-20
EP4410979A3 (fr) 2024-11-06
IL262194A (en) 2018-11-29
US11723986B2 (en) 2023-08-15
WO2017181015A1 (fr) 2017-10-19
ES2977730T3 (es) 2024-08-29
EP3442602A1 (fr) 2019-02-20
EP3442561A4 (fr) 2019-12-18
EP3442601A4 (fr) 2019-11-20
EP3442601A1 (fr) 2019-02-20
IL302200A (en) 2023-06-01
JP2019513389A (ja) 2019-05-30
JP7078546B2 (ja) 2022-05-31
MX2018012604A (es) 2019-07-04
MY195438A (en) 2023-01-21
US20190117795A1 (en) 2019-04-25
WO2017181011A1 (fr) 2017-10-19
AU2023201093A1 (en) 2023-05-18
WO2017181014A1 (fr) 2017-10-19
EP3442561B1 (fr) 2024-02-14
JP7153562B2 (ja) 2022-10-14
AU2017250791B2 (en) 2022-11-24
US20230173101A1 (en) 2023-06-08
KR102649707B1 (ko) 2024-03-22
MX2024005644A (es) 2024-05-24
IL262194B1 (en) 2023-05-01
CN109069672A (zh) 2018-12-21
US20230001015A1 (en) 2023-01-05
US11406717B2 (en) 2022-08-09
US20190060489A1 (en) 2019-02-28
IL262194B2 (en) 2023-09-01
CN109069672B (zh) 2024-06-21
EP3442561A1 (fr) 2019-02-20
AU2017250791A1 (en) 2018-10-25
ES2985822T3 (es) 2024-11-07
JP7189771B2 (ja) 2022-12-14
US11298429B2 (en) 2022-04-12
EP3442602B1 (fr) 2025-02-19
EP4410979A2 (fr) 2024-08-07

Similar Documents

Publication Publication Date Title
MA44683A (fr) Délivrance de vecteur de virus adéno-associé pour traiter la dystrophie musculaire
IL279154B (en) Adenovirus-related vector
IL269391A (en) Administration of muscle-specific microdystrophin using an adeno-associated virus vector for the treatment of muscular dystrophy
KR102502091B9 (ko) β-사르코글리칸 및 마이크로RNA-29의 아데노-관련 바이러스 벡터 전달 및 근이영양증의 처리
EP3294323A4 (fr) Virus adéno-associé pour une administration thérapeutique au système nerveux central
HK1247823A1 (zh) 骨質疏鬆的治療
EP3233878A4 (fr) Phosphoramidates pour le traitement du virus de l'hépatite b
MA39748A (fr) Cenicriviroc pour le traitement de la fibrose
EP3380062A4 (fr) Compositions pour le traitement des cheveux
EP3391454A4 (fr) Liquides ioniques fonctionnalisés au silane
MA47800A (fr) Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
EP3383407A4 (fr) Conjugués de cytarabine pour le traitement du cancer
MA43570A (fr) Thérapie génique pour traiter l'hypercholestérolémie familiale
EP3535588A4 (fr) Cibles thérapeutiques pour dystrophie musculaire facio-scapulo-humérale
MA46999A (fr) Oligonucléotides modifiés pour traiter la polykystose rénale
EP3307742A4 (fr) Méthodes pour traiter ou prévenir une protéopathie
EP3386593A4 (fr) Composition immunomodulatrice pour le traitement
MA50836A (fr) Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
EP3363449A4 (fr) Agent de renforcement musculaire
MA41899A (fr) Anticorps anti-flt-1 pour traiter la dystrophie musculaire de duchenne
MA51688A (fr) Protéines prohémostatiques pour le traitement de saignement
MA44244A (fr) Formulation pour traiter la migraine
GB201512656D0 (en) Helly's whales
TH1501004218B (th) ปั๊มเเบบหมุนเหวี่ยง