JP7610923B2 - Ｃｎｓ障害を治療するための方法及びベクター - Google Patents

Ｃｎｓ障害を治療するための方法及びベクター Download PDF

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JP7610923B2
JP7610923B2 JP2019512292A JP2019512292A JP7610923B2 JP 7610923 B2 JP7610923 B2 JP 7610923B2 JP 2019512292 A JP2019512292 A JP 2019512292A JP 2019512292 A JP2019512292 A JP 2019512292A JP 7610923 B2 JP7610923 B2 JP 7610923B2
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aav
nucleic acid
apoe
vector
aav4
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JP2019529385A5 (enExample
JP2019529385A (ja
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キャサリン エー． ハイ，
ビバリー エル． デイビッドソン，
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Children's Hospital of Philadelphia
Spark Therapeutics Inc
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Spark Therapeutics Inc
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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
    • A61K48/0075—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
    • 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
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/06—Sulfuric ester hydrolases (3.1.6)
    • C12Y301/06004—N-Acetylgalactosamine-6-sulfatase (3.1.6.4)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01031—Beta-glucuronidase (3.2.1.31)
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • 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
    • A01K2267/0312—Animal model for Alzheimer's disease
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/14—Dipeptidyl-peptidases and tripeptidyl-peptidases (3.4.14)
    • C12Y304/14009—Tripeptidyl-peptidase I (3.4.14.9)

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
JP2019512292A 2016-09-02 2017-09-01 Ｃｎｓ障害を治療するための方法及びベクター Active JP7610923B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022002163A JP2022050574A (ja) 2016-09-02 2022-01-11 Ｃｎｓ障害を治療するための方法及びベクター

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662383274P 2016-09-02 2016-09-02
US62/383,274 2016-09-02
PCT/US2017/049959 WO2018045347A1 (en) 2016-09-02 2017-09-01 Methods and vectors for treating cns disorders

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Publications (3)

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JP2019529385A JP2019529385A (ja) 2019-10-17
JP2019529385A5 JP2019529385A5 (enExample) 2020-09-17
JP7610923B2 true JP7610923B2 (ja) 2025-01-09

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JP2022002163A Pending JP2022050574A (ja) 2016-09-02 2022-01-11 Ｃｎｓ障害を治療するための方法及びベクター

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US (1) US20190192693A1 (enExample)
EP (1) EP3506930A4 (enExample)
JP (2) JP7610923B2 (enExample)
CN (1) CN110121356A (enExample)
AU (2) AU2017318717B2 (enExample)
BR (1) BR112019004353A2 (enExample)
CA (1) CA3035628A1 (enExample)
MX (1) MX2019002518A (enExample)
WO (1) WO2018045347A1 (enExample)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
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GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
US20200164008A1 (en) * 2017-07-17 2020-05-28 Spark Therapeutics, Inc. Apheresis methods and uses
IL313246B2 (en) 2018-06-28 2025-10-01 Univ North Carolina Chapel Hill Optimal cln5 expression genes and cassettes and their use
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
EP3860659A1 (en) 2018-10-01 2021-08-11 The Trustees of the University of Pennsylvania Compositions useful for treating gm1 gangliosidosis
KR20260086827A (ko) * 2018-11-14 2026-06-10 리젠엑스바이오 인크. 신경원성 세로이드 리포푸신증에 대한 유전자 요법
CA3121211A1 (en) * 2018-11-28 2020-06-04 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
MX2021010134A (es) 2019-02-22 2021-09-23 Univ Pennsylvania Virus adenoasociado recombinante para el tratamiento de la neurodegeneracion de inicio en adultos asociada a grn.
US20220348638A1 (en) * 2019-09-24 2022-11-03 The J. David Gladstone Institutes, a testamentary trust estabilished under the Will of J. David Glad Method of targeting neuronal apoe to treat a neurocognitive disorder
JP2023500793A (ja) * 2019-10-16 2023-01-11 コーネル・ユニバーシティー アルツハイマー病に対する遺伝子治療方法
KR20220107243A (ko) 2019-11-25 2022-08-02 코넬 유니버시티 Apoe 유전자 요법
KR20230079139A (ko) * 2020-10-01 2023-06-05 상가모 테라퓨틱스, 인코포레이티드 항-aav 항체의 검출 방법
CN112852881B (zh) * 2021-02-09 2023-08-15 南京贝思奥生物科技有限公司 利用细胞穿膜肽增强腺相关病毒在中枢神经系统转导效率的方法
JP2025514794A (ja) * 2022-04-22 2025-05-09 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィア ヒト上衣特異的プロモーターおよびその使用方法
TW202426479A (zh) * 2022-10-10 2024-07-01 美商星火治療公司 Apoe基因療法

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WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2015077473A1 (en) 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits

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US20150183850A1 (en) * 2012-05-18 2015-07-02 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits
RU2664471C2 (ru) * 2013-07-26 2018-08-17 Юниверсити Оф Айова Рисерч Фаундейшн Способы и композиции для лечения болезней мозга
CA2977355A1 (en) * 2015-02-20 2016-08-25 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases

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WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
WO2015077473A1 (en) 2013-11-20 2015-05-28 University Of Iowa Research Foundation Methods and compositions for treating amyloid deposits

Non-Patent Citations (1)

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Title
Ａｎｎ． Ｎｅｕｒｏｌ． （２０１４） ｖｏｌ．７６， ｓｕｕｐｌ．１８， ｐ．Ｓ１７６（ＰＬ１－６）

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MX2019002518A (es) 2019-07-18
JP2022050574A (ja) 2022-03-30
AU2024227138A1 (en) 2024-10-31
AU2017318717B2 (en) 2024-10-24
WO2018045347A1 (en) 2018-03-08
US20190192693A1 (en) 2019-06-27
CN110121356A (zh) 2019-08-13
BR112019004353A2 (pt) 2019-05-28
CA3035628A1 (en) 2018-03-08
EP3506930A4 (en) 2020-09-23
EP3506930A1 (en) 2019-07-10
JP2019529385A (ja) 2019-10-17
AU2017318717A1 (en) 2019-03-21

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