MA45477A - Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire - Google Patents

Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire

Info

Publication number
MA45477A
MA45477A MA045477A MA45477A MA45477A MA 45477 A MA45477 A MA 45477A MA 045477 A MA045477 A MA 045477A MA 45477 A MA45477 A MA 45477A MA 45477 A MA45477 A MA 45477A
Authority
MA
Morocco
Prior art keywords
vector
microarn
dystrophine
micro
muscle dystrophy
Prior art date
Application number
MA045477A
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.)
Publication date
Publication of MA45477A publication Critical patent/MA45477A/fr

Links

Classifications

    • 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
    • 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
    • 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
    • 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 nucleic acids [NA]
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Toxicology (AREA)
  • Dermatology (AREA)
  • Mycology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
MA045477A 2016-04-15 Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire MA45477A (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
MA45477A true MA45477A (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
MA44683A MA44683B1 (fr) 2016-04-15 2017-04-14 Délivrance de vecteur de virus adéno-associé pour traiter la dystrophie musculaire

Family Applications After (1)

Application Number Title Priority Date Filing Date
MA44683A MA44683B1 (fr) 2016-04-15 2017-04-14 Délivrance de vecteur de virus adéno-associé pour traiter la dystrophie musculaire

Country Status (28)

Country Link
US (6) US11298429B2 (fr)
EP (5) EP4541894A3 (fr)
JP (6) JP7078546B2 (fr)
KR (3) KR102649707B1 (fr)
CN (2) CN118895311A (fr)
AU (4) AU2017250791B2 (fr)
BR (1) BR112018071182A2 (fr)
CA (2) CA3020996A1 (fr)
CO (1) CO2018012084A2 (fr)
DK (1) DK3442602T3 (fr)
ES (3) ES2985822T3 (fr)
FI (1) FI3442602T3 (fr)
HR (1) HRP20250541T1 (fr)
HU (1) HUE071243T2 (fr)
IL (3) IL319636A (fr)
LT (1) LT3442602T (fr)
MA (2) MA44683B1 (fr)
MD (1) MD3442602T2 (fr)
MX (2) MX2018012604A (fr)
MY (2) MY209041A (fr)
NZ (2) NZ787316A (fr)
PL (1) PL3442602T3 (fr)
PT (1) PT3442602T (fr)
RS (1) RS66847B1 (fr)
SG (1) SG11201808941PA (fr)
SI (1) SI3442602T1 (fr)
WO (3) WO2017181011A1 (fr)
ZA (2) ZA201806746B (fr)

Families Citing this family (30)

* 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
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
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
CN110997923B (zh) * 2017-03-17 2024-01-02 全国儿童医院研究所 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
EP3697915A4 (fr) * 2017-10-18 2021-12-08 Research Institute at Nationwide Children's Hospital Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
KR20240164969A (ko) * 2017-10-20 2024-11-21 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 Nt-3 유전자 치료를 위한 방법 및 물질
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
JP7304878B2 (ja) * 2018-03-16 2023-07-07 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 キャプシド改変による組織特異的遺伝子送達の増加
US20210244828A1 (en) * 2018-05-15 2021-08-12 Ramot At Tel-Aviv University Ltd. Miri26-5p for treating motor neuron diseases
JP7534961B2 (ja) * 2018-06-18 2024-08-15 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 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
CN120393053A (zh) * 2018-06-18 2025-08-01 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
WO2020006458A1 (fr) 2018-06-29 2020-01-02 Research Institute At Nationwide Children's Hospital Produits de virus adéno-associés recombinants et méthodes de traitement de la dystrophie des ceintures 2a
EP3598978B1 (fr) 2018-07-26 2024-05-29 EXOFIX S.r.l. Exosomes dérivées de cellules fibroadipogéniques progénitrices pour la régénération des muscles dystrophiques
WO2020123645A1 (fr) * 2018-12-12 2020-06-18 Solid Biosciences Inc. Polythérapie pour le traitement de la dystrophie musculaire
US12377170B2 (en) 2019-02-26 2025-08-05 Research Institute At Nationwide Children's Hospital Adeno-associated virus vector delivery of β-sarcoglycan and the treatment of muscular dystrophy
JP7635139B2 (ja) * 2019-03-25 2025-02-25 ジェネトン オーバーラップａａｖベクターを使用する大きいサイズのクアシジストロフィンの産生
EP4017871B1 (fr) 2019-08-21 2024-01-17 Research Institute at Nationwide Children's Hospital Administration de vecteur de virus adéno-associé d'alpha-sarcoglycane et traitement de dystrophie musculaire
WO2021142447A1 (fr) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Vecteur viral pour polythérapie
WO2021142435A1 (fr) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Polythérapie pour le traitement de la dystrophie musculaire
BR112022021635A2 (pt) 2020-04-29 2022-12-06 Bristol Myers Squibb Co Distrofinas miniaturizadas tendo domínios de fusão de espectrina e usos das mesmas
IL299094A (en) * 2020-06-15 2023-02-01 Res Inst Nationwide Childrens Hospital Administration of an adeno-associated virus vector for muscular dystrophies
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
EP3936616A1 (fr) * 2020-07-10 2022-01-12 Charité - Universitätsmedizin Berlin Thérapie ciblée par micro-arn pour réparation cardiaque
KR20240095165A (ko) 2021-08-11 2024-06-25 솔리드 바이오사이언시즈 인크. 근이영양증의 치료
EP4401794A4 (fr) * 2021-09-16 2025-09-17 Univ Florida Particules de vaa avec répétitions terminales inversées modifiées pour expression génique accrue dans le muscle
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
EP4215614A1 (fr) 2022-01-24 2023-07-26 Dynacure Thérapie de combinaison pour maladies liées à la dystrophine
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
JP2025527658A (ja) 2022-08-24 2025-08-22 リジェネックスバイオ インコーポレイテッド 組換えアデノ随伴ウイルス及びその使用
WO2025188755A1 (fr) * 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Compositions de virus adéno-associés pour le traitement de la dystrophie musculaire de duchenne

Family Cites Families (54)

* 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
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
EP0728214B1 (fr) 1993-11-09 2004-07-28 Medical College Of Ohio Lignees cellulaires stables aptes a exprimer le gene de replication du virus adeno-associe
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
AU707866B2 (en) 1994-12-06 1999-07-22 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
CA2625279A1 (fr) 1995-08-30 1997-03-06 Genzyme Corporation Purification d'adenovirus et de virus adeno-associe (aav) par voie chromatographique
ES2317646T3 (es) 1995-09-08 2009-04-16 Genzyme Corporation Vectores aav mejorados para terapia genica.
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
CN1233291A (zh) 1996-09-06 1999-10-27 宾西法尼亚大学托管会 重组腺伴随病毒定向基因治疗的方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ES2399232T3 (es) 1997-09-05 2013-03-26 Genzyme Corporation Procedimientos para la generación de preparaciones de vectores de AAV recombinantes de títulos altos exentas de auxiliares
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
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
AU2001261063A1 (en) 2000-04-28 2001-11-12 Xiao Xiao Dna sequences encoding dystrophin minigenes and methods of use thereof
ATE373095T1 (de) 2000-07-14 2007-09-15 Univ Michigan Mutierte muskelspezifische enhancer
EP1366160B1 (fr) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Sequences peptidiques et d'acides nucleiques de mini-dystrophine
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
PT2359869T (pt) 2001-12-17 2019-04-16 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
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
US20100184209A1 (en) * 2006-02-17 2010-07-22 Dharmacon, Inc. Compositions and methods for inhibiting gene silencing by rna interference
US7892824B2 (en) * 2007-01-18 2011-02-22 University Of Missouri-Columbia Synthetic mini/micro-dystrophin genes to restore nNOS to the sarcolemma
NZ583024A (en) 2007-07-31 2012-04-27 Regents The Univeristy Of Texas System Board Of An agonist of miR-29a, miR-29b or miR-29c that prevents tissue fibrosis and uses thereof
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
WO2013078316A1 (fr) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Administration de virus adéno-associé recombinant de polynucléotides alpha-sarcoglycanes
WO2013102904A1 (fr) * 2012-01-05 2013-07-11 Hadasit Medical Research Services & Development Ltd. Procédés et compositions pour l'apport d'un gène
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
CA2909807C (fr) * 2013-04-20 2023-08-08 Research Institute At Nationwide Children's Hospital Administration de virus adeno-associe recombinant de constructions polynucleotidiques u7snarn ciblant l'exon 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
PL3097197T3 (pl) * 2014-01-21 2021-06-28 Vrije Universiteit Brussel Mięśniowo-specyficzne elementy regulatorowe kwasów nukleinowych oraz sposoby i ich zastosowanie
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
WO2015161255A1 (fr) * 2014-04-18 2015-10-22 Board Of Regents Of The Nevada System Of Higher Education, On Behalf Of The University Of Nevada, Reno Procédés d'augmentation ou de diminution des taux de mir124 et mir29 chez des sujets atteints de dystrophie musculaire
EP2960336A1 (fr) 2014-06-27 2015-12-30 Genethon Traitement systémique efficace de pathologies dystrophiques musculaires
JP6986444B2 (ja) * 2014-08-09 2021-12-22 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 Ｄｍｄ遺伝子のエクソン５内の内部リボソーム進入部位を活性化するための方法及び材料
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
US10842886B2 (en) * 2014-10-10 2020-11-24 Research Institute At Nationwide Children's Hospital Guided injections for AAV gene transfer to muscle
CN107250364A (zh) 2015-01-16 2017-10-13 华盛顿大学 新的微小肌养蛋白及相关使用方法
GB201507842D0 (en) 2015-05-07 2015-06-17 New Royal Holloway & Bedford Production of large-sized microdystrophins in an AAV-based vector configuration
JP7338970B2 (ja) 2015-09-17 2023-09-05 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 Ｇａｌｇｔ２遺伝子治療のための方法および物質
WO2017165859A1 (fr) 2016-03-24 2017-09-28 Research Institute At Nationwide Children's Hospital Protéines de capside virale modifiées
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
FI3442600T3 (fi) 2016-04-15 2024-05-14 Res Inst Nationwide Childrens Hospital Adeno-assosioidulla virusvektorilla b-sarkoglykaanin ja mikro-rna-29:n toimittaminen ja lihasdystrofian hoito
CA2971303A1 (fr) 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Genes de mini-dystrophine optimises et cassettes d'expression et leur utilisation
CN120393053A (zh) 2018-06-18 2025-08-01 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
WO2017181014A1 (fr) 2017-10-19
KR20240042136A (ko) 2024-04-01
JP7153562B2 (ja) 2022-10-14
DK3442602T3 (da) 2025-05-19
EP3442561B1 (fr) 2024-02-14
IL302200B2 (en) 2025-08-01
US11406717B2 (en) 2022-08-09
US20190055581A1 (en) 2019-02-21
CN109069672B (zh) 2024-06-21
IL319636A (en) 2025-05-01
MA44683A (fr) 2019-02-20
BR112018071182A2 (pt) 2019-04-09
MX2018012604A (es) 2019-07-04
EP4410979A2 (fr) 2024-08-07
ES2985822T3 (es) 2024-11-07
EP3442601A4 (fr) 2019-11-20
WO2017181015A1 (fr) 2017-10-19
US20250161490A1 (en) 2025-05-22
JP2025118741A (ja) 2025-08-13
HUE071243T2 (hu) 2025-08-28
EP3442602A4 (fr) 2019-11-27
EP3442602B1 (fr) 2025-02-19
AU2017250790A1 (en) 2018-11-15
IL262194A (en) 2018-11-29
US11723986B2 (en) 2023-08-15
EP3442601A1 (fr) 2019-02-20
JP2022020839A (ja) 2022-02-01
EP4541894A2 (fr) 2025-04-23
JP2019513393A (ja) 2019-05-30
US20230001015A1 (en) 2023-01-05
PT3442602T (pt) 2025-05-07
JP7431789B2 (ja) 2024-02-15
EP3442601B1 (fr) 2024-07-03
CO2018012084A2 (es) 2019-02-08
MA44683B1 (fr) 2025-07-31
WO2017181011A1 (fr) 2017-10-19
EP3442602A1 (fr) 2019-02-20
ES2977730T3 (es) 2024-08-29
CA3020996A1 (fr) 2017-10-19
SG11201808941PA (en) 2018-11-29
US20230173101A1 (en) 2023-06-08
CA3020999A1 (fr) 2017-10-19
FI3442602T3 (fi) 2025-05-16
KR20250061772A (ko) 2025-05-08
JP2019513389A (ja) 2019-05-30
HRP20250541T1 (hr) 2025-07-18
AU2025208472A1 (en) 2025-08-14
NZ747049A (en) 2025-10-31
AU2023201093A1 (en) 2023-05-18
RS66847B1 (sr) 2025-06-30
EP3442561A4 (fr) 2019-12-18
MY209041A (en) 2025-06-17
US20190060489A1 (en) 2019-02-28
IL302200A (en) 2023-06-01
IL302200B1 (en) 2025-04-01
LT3442602T (lt) 2025-06-10
IL262194B2 (en) 2023-09-01
EP4410979A3 (fr) 2024-11-06
AU2023201093B2 (en) 2025-05-15
JP2019513779A (ja) 2019-05-30
ZA201806746B (en) 2023-12-20
NZ787316A (en) 2025-10-31
MY195438A (en) 2023-01-21
CN118895311A (zh) 2024-11-05
ES3026989T3 (en) 2025-06-12
JP2024023439A (ja) 2024-02-21
JP7189771B2 (ja) 2022-12-14
PL3442602T3 (pl) 2025-07-07
US20190117795A1 (en) 2019-04-25
IL262194B1 (en) 2023-05-01
MD3442602T2 (ro) 2025-07-31
AU2017250791A1 (en) 2018-10-25
KR102649707B1 (ko) 2024-03-22
EP4541894A3 (fr) 2025-06-11
EP3442561A1 (fr) 2019-02-20
US11298429B2 (en) 2022-04-12
CN109069672A (zh) 2018-12-21
MX2024005644A (es) 2024-05-24
JP7078546B2 (ja) 2022-05-31
KR20180133486A (ko) 2018-12-14
AU2017250791B2 (en) 2022-11-24
ZA201906608B (en) 2025-03-26
SI3442602T1 (sl) 2025-07-31
KR102801562B1 (ko) 2025-04-30
AU2017250790B2 (en) 2024-06-20

Similar Documents

Publication Publication Date Title
MA45477A (fr) Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire
EP3596222A4 (fr) Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique du muscle pour traiter la dystrophie musculaire
EP3442600A4 (fr) Administration de b-sarcoglycane et de microarn-29 à l'aide d'un vecteur de virus adéno-associé, et traitement de la dystrophie musculaire
IL256652A (en) Therapeutic compositions, combinations, and methods of use
MA50836A (fr) Administration par vecteur à virus adéno-associé de micro-dystrophine spécifique de muscles pour traiter la dystrophie musculaire
ZA201708039B (en) Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
EP3360554A4 (fr) Agent anti-virus de l'hépatite b
HUE057795T2 (hu) Adenoasszociált vírus terápiás szerek központi idegrendszerbe történõ bejuttatására
MA40867A (fr) Méthodes pour le traitement d'infections virales à filoviridae
PT3519569T (pt) Vetores de vírus adenoassociados para o tratamento de mucopolissacaridoses
MA43570A (fr) Thérapie génique pour traiter l'hypercholestérolémie familiale
EP3389673A4 (fr) Procédés et compositions pour l'administration de polynucléotides
EP3317290A4 (fr) Compositions et méthodes de traitement d'une infection virale
MA41120A (fr) Compositions comprenant le 15-hepe et méthodes de traitement ou de prévention de la fibrose à l'aide de celles-ci
IL251404A0 (en) Substituted aromatic compounds and pharmaceutical compositions for the prevention and treatment of osteoporosis
EP3373922A4 (fr) Compositions et méthodes pour le traitement de l'homocystinurie
EP3294313A4 (fr) Méthodes associées à la prévention et et au traitement d'une pharmacorésistance
PL3377500T3 (pl) Związki oksadiazaspiro do leczenia nadużywania i uzależnienia od narkotyków/leków
EP3393462A4 (fr) Compositions et méthodes de traitement, d'amélioration et de prévention de l'hypothermie induite par une anesthésie
HK40118571A (zh) 腺相关病毒载体传递微肌营养不良蛋白以治疗肌营养不良症