MA47800A - Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire - Google Patents

Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire

Info

Publication number
MA47800A
MA47800A MA047800A MA47800A MA47800A MA 47800 A MA47800 A MA 47800A MA 047800 A MA047800 A MA 047800A MA 47800 A MA47800 A MA 47800A MA 47800 A MA47800 A MA 47800A
Authority
MA
Morocco
Prior art keywords
dystrophine
adeno
fragment
micro
viral vector
Prior art date
Application number
MA047800A
Other languages
English (en)
Inventor
Hanns Lochmuller
Oliver Muller
Original Assignee
Univ Newcastle
Univ Of Heidelberg
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 Univ Newcastle, Univ Of Heidelberg filed Critical Univ Newcastle
Publication of MA47800A publication Critical patent/MA47800A/fr

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
MA047800A 2017-03-17 2018-03-16 Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire MA47800A (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201762473255P 2017-03-17 2017-03-17

Publications (1)

Publication Number Publication Date
MA47800A true MA47800A (fr) 2020-01-22

Family

ID=64564915

Family Applications (1)

Application Number Title Priority Date Filing Date
MA047800A MA47800A (fr) 2017-03-17 2018-03-16 Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire

Country Status (5)

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

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US11617783B2 (en) 2015-11-16 2023-04-04 Research Institute At Nationwide Children's Hospital Repairing a mutant human titin gene using CRISPR technology
WO2019012336A2 (fr) * 2017-03-17 2019-01-17 Newcastle University Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire
AU2019216257B2 (en) 2018-01-31 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for limb-girdle muscular dystrophy type 2C
EP4591888A3 (fr) 2018-06-18 2026-01-21 Research Institute at Nationwide Children's Hospital Administration de vecteur de virus adéno-associé de micro-dystrophine spécifique aux muscles pour traiter la dystrophie musculaire
US12391928B2 (en) 2018-06-29 2025-08-19 Research Institute At Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
CN113766935A (zh) 2019-02-26 2021-12-07 全国儿童医院研究所 β-肌聚糖的腺相关病毒载体递送和肌营养不良症的治疗
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
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EP4108263A3 (fr) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Produits de virus adéno-associés recombinants et méthodes de traitement de la dystrophie musculaire des ceintures 2a
EP4219726A1 (fr) 2021-10-15 2023-08-02 Research Institute at Nationwide Children's Hospital Vecteur de virus adéno-associé auto-complémentaire et son utilisation dans le traitement de la dystrophie musculaire
CN119233831A (zh) * 2022-01-17 2024-12-31 迪纳柯公司 用于与肌营养不良蛋白有关的心肌病的基因疗法组合物和治疗
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CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用
TW202530248A (zh) 2023-09-25 2025-08-01 美商薩羅塔治療公司 用於治療肌肉萎縮症之具有增強的肌動蛋白結合親和力之適應性人類肌肉萎縮蛋白
NL2037199B1 (en) 2024-03-07 2025-09-15 C2Ca Tech B V Method for obtaining a cement constituent from a concrete element

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WO2019012336A2 (fr) 2019-01-17
JP7162021B2 (ja) 2022-10-27
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