CN110709095B - 小基因治疗 - Google Patents

小基因治疗 Download PDF

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Publication number
CN110709095B
CN110709095B CN201880037056.0A CN201880037056A CN110709095B CN 110709095 B CN110709095 B CN 110709095B CN 201880037056 A CN201880037056 A CN 201880037056A CN 110709095 B CN110709095 B CN 110709095B
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CN110709095A (zh
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高光平
H·康纳
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University of Massachusetts Amherst
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    • 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
    • A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
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    • 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
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    • 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
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    • 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
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • 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
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    • A61K9/0048—Eye, e.g. artificial tears
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N15/09—Recombinant DNA-technology
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • 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
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers

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  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
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  • Mycology (AREA)
  • Cell Biology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201880037056.0A 2017-04-05 2018-04-05 小基因治疗 Active CN110709095B (zh)

Priority Applications (1)

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CN202410492766.XA CN118360335A (zh) 2017-04-05 2018-04-05 小基因治疗

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US201762481727P 2017-04-05 2017-04-05
US62/481,727 2017-04-05
PCT/US2018/026230 WO2018187552A1 (en) 2017-04-05 2018-04-05 Minigene therapy

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EP (1) EP3606544B1 (enExample)
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AU (2) AU2018248304C1 (enExample)
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WO2018187552A1 (en) 2017-04-05 2018-10-11 University Of Massachusetts Minigene therapy
EP3956452A4 (en) * 2019-04-19 2023-03-15 University of Massachusetts GENE THERAPIES FOR USHER SYNDROME (USH2A)
WO2020214797A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for usher syndrome (ush1b)
CN114206353B (zh) * 2019-05-20 2024-11-08 马萨诸塞大学 小基因疗法
JP2022549138A (ja) 2019-09-18 2022-11-24 インターガラクティック セラピューティクス インコーポレイテッド 合成ｄｎａベクターおよびその使用法
WO2022023284A1 (en) 2020-07-27 2022-02-03 Anjarium Biosciences Ag Compositions of dna molecules, methods of making therefor, and methods of use thereof
CN112062827B (zh) * 2020-09-16 2022-10-21 中国人民解放军军事科学院军事医学研究院 Cep55蛋白在调控纤毛去组装和制备纤毛相关疾病模型中的应用
WO2022120080A1 (en) * 2020-12-03 2022-06-09 University Of Massachusetts Development of novel gene therapeutics for fibrodysplasia ossificans progressiva
US11926842B2 (en) * 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors

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WO2016033338A1 (en) * 2014-08-27 2016-03-03 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Methods and compositions for treating leber congenital amaurosis

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CN110709095A (zh) 2020-01-17
AU2023200880B2 (en) 2024-04-11
US12454702B2 (en) 2025-10-28
AU2018248304B2 (en) 2022-11-17
EP3606544A1 (en) 2020-02-12
US11739346B2 (en) 2023-08-29
CN118360335A (zh) 2024-07-19
AU2023200880A1 (en) 2023-03-16
US20200056204A1 (en) 2020-02-20
WO2018187552A1 (en) 2018-10-11
AU2018248304C1 (en) 2023-02-16
JP7535332B2 (ja) 2024-08-16
JP2023088902A (ja) 2023-06-27
JP7307480B2 (ja) 2023-07-12
EP3606544A4 (en) 2021-04-07
JP2020512822A (ja) 2020-04-30
WO2018187552A9 (en) 2019-10-17
US20240076692A1 (en) 2024-03-07
EP3606544B1 (en) 2025-06-04
AU2018248304A1 (en) 2019-10-24
CA3059112A1 (en) 2018-10-11

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