KR20220047538A - 트랜스진 발현을 위한 방법 및 조성물 - Google Patents

트랜스진 발현을 위한 방법 및 조성물 Download PDF

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KR20220047538A
KR20220047538A KR1020217037211A KR20217037211A KR20220047538A KR 20220047538 A KR20220047538 A KR 20220047538A KR 1020217037211 A KR1020217037211 A KR 1020217037211A KR 20217037211 A KR20217037211 A KR 20217037211A KR 20220047538 A KR20220047538 A KR 20220047538A
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raav
transgene
enhancer
polynucleotide
cell
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Korean (ko)
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존 에프. 엥겔하르트
쯔잉 옌
잉화 탕
에릭 웬
선 린
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유니버시티 오브 아이오와 리써치 파운데이션
스피로반트 사이언시스, 인크.
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • A61K31/00Medicinal preparations containing organic active ingredients
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    • A61K31/7028Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
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    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • A61K35/761Adenovirus
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    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/05Dipeptides
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K38/00Medicinal preparations containing peptides
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    • A61K38/06Tripeptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/04Peptides having up to 20 amino acids in a fully defined sequence; Derivatives thereof
    • A61K38/07Tetrapeptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
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  • Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
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  • General Engineering & Computer Science (AREA)
  • Physics & Mathematics (AREA)
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  • General Chemical & Material Sciences (AREA)
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  • Biophysics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biochemistry (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Pulmonology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
KR1020217037211A 2019-04-15 2020-04-15 트랜스진 발현을 위한 방법 및 조성물 Ceased KR20220047538A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962833979P 2019-04-15 2019-04-15
US62/833,979 2019-04-15
US201962926317P 2019-10-25 2019-10-25
US62/926,317 2019-10-25
US202062967219P 2020-01-29 2020-01-29
US62/967,219 2020-01-29
PCT/US2020/028269 WO2020214672A1 (en) 2019-04-15 2020-04-15 Methods and compositions for transgene expression

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KR20220047538A true KR20220047538A (ko) 2022-04-18

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KR1020217037211A Ceased KR20220047538A (ko) 2019-04-15 2020-04-15 트랜스진 발현을 위한 방법 및 조성물

Country Status (13)

Country Link
US (1) US20220195461A1 (https=)
EP (1) EP3955970A1 (https=)
JP (2) JP2022529470A (https=)
KR (1) KR20220047538A (https=)
CN (1) CN114340683A (https=)
AU (1) AU2020257182A1 (https=)
BR (1) BR112021020706A2 (https=)
CA (1) CA3137078A1 (https=)
IL (1) IL287262A (https=)
MX (1) MX2021012682A (https=)
PH (1) PH12021552609A1 (https=)
SG (1) SG11202111353QA (https=)
WO (1) WO2020214672A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017139381A1 (en) 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3198936A1 (en) 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
US20220241436A1 (en) * 2019-04-15 2022-08-04 University Of Iowa Research Foundation Compositions and methods for treatment of cystic fibrosis

Family Cites Families (20)

* Cited by examiner, † Cited by third party
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US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
US5656785A (en) 1995-08-07 1997-08-12 The Charles Stark Draper Laboratory, Inc. Micromechanical contact load force sensor for sensing magnitude and distribution of loads and tool employing micromechanical contact load force sensor
AU5445998A (en) 1996-11-19 1998-06-10 Surgx Corporation A transient voltage protection device and method of making same
AU741605B2 (en) 1996-12-18 2001-12-06 Targeted Genetics Corporation AAV split-packaging genes and cell lines comprising such genes for use in the production of recombinant AAV vectors
JP2007524379A (ja) * 2003-03-31 2007-08-30 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン 上皮ナトリウムチャンネル関連障害のPharmico−遺伝子治療
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
CA2788682C (en) * 2010-02-05 2019-03-05 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
KR20210122917A (ko) * 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
AU2014251099B2 (en) * 2013-04-08 2019-01-17 The University Of Kansas Chimeric adeno-associated virus/ bocavirus parvovirus vector
KR102537394B1 (ko) * 2014-03-17 2023-05-30 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
WO2017139381A1 (en) * 2016-02-08 2017-08-17 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
CA3198936A1 (en) * 2016-03-07 2017-09-14 University Of Iowa Research Foundation Aav-mediated expression using a synthetic promoter and enhancer
CA3040179A1 (en) * 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CN106928336B (zh) * 2017-01-20 2019-09-24 首都医科大学附属北京儿童医院 囊性纤维化患者的cftr基因突变形式及其应用
AU2018227483A1 (en) * 2017-02-28 2019-09-12 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
CN110770346B (zh) * 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
US20220241436A1 (en) * 2019-04-15 2022-08-04 University Of Iowa Research Foundation Compositions and methods for treatment of cystic fibrosis

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Publication number Publication date
MX2021012682A (es) 2022-03-25
CN114340683A (zh) 2022-04-12
IL287262A (en) 2021-12-01
AU2020257182A1 (en) 2021-12-09
PH12021552609A1 (en) 2022-06-27
BR112021020706A2 (pt) 2022-03-15
JP2023126658A (ja) 2023-09-07
JP2022529470A (ja) 2022-06-22
SG11202111353QA (en) 2021-11-29
WO2020214672A1 (en) 2020-10-22
CA3137078A1 (en) 2020-10-22
US20220195461A1 (en) 2022-06-23
EP3955970A1 (en) 2022-02-23

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