TW202045530A - 腺相關病毒對ｃｌｎ３聚核苷酸的遞送 - Google Patents

腺相關病毒對ｃｌｎ３聚核苷酸的遞送 Download PDF

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TW202045530A
TW202045530A TW109103419A TW109103419A TW202045530A TW 202045530 A TW202045530 A TW 202045530A TW 109103419 A TW109103419 A TW 109103419A TW 109103419 A TW109103419 A TW 109103419A TW 202045530 A TW202045530 A TW 202045530A
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cln3
nucleic acid
scaav9
seq
sequence
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TW109103419A
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Chinese (zh)
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凱瑟琳 梅爾
布萊恩 Ｋ 卡斯帕
凱文 福斯特
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美國全美兒童醫院之研究學會
美國俄亥俄州創新基金會
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Publication of TW202045530A publication Critical patent/TW202045530A/zh

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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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    • 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
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
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    • C07—ORGANIC CHEMISTRY
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    • 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
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    • 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
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    • 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
    • C12N2750/14171—Demonstrated in vivo effect

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
TW109103419A 2019-02-04 2020-02-04 腺相關病毒對ｃｌｎ３聚核苷酸的遞送 TW202045530A (zh)

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US201962800911P 2019-02-04 2019-02-04
US62/800,911 2019-02-04

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TW202045530A true TW202045530A (zh) 2020-12-16

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US (1) US12404523B2 (enExample)
EP (1) EP3921430A1 (enExample)
JP (2) JP7691367B2 (enExample)
KR (1) KR20210124300A (enExample)
CN (1) CN113646436A (enExample)
AR (1) AR118005A1 (enExample)
AU (1) AU2020217708A1 (enExample)
BR (1) BR112021015285A2 (enExample)
CA (1) CA3129077A1 (enExample)
CL (1) CL2021002049A1 (enExample)
EA (1) EA202192160A1 (enExample)
IL (1) IL285328A (enExample)
MX (1) MX2021009401A (enExample)
SG (1) SG11202107986YA (enExample)
TW (1) TW202045530A (enExample)
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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2879719B1 (en) 2012-08-01 2018-07-04 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
WO2022170038A1 (en) * 2021-02-05 2022-08-11 Amicus Therapeutics, Inc. Adeno-associated virus delivery of cln3 polynucleotide
JP2025524569A (ja) * 2022-07-06 2025-07-30 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 Ｃｌｎ１ポリヌクレオチドのアデノ随伴ウイルス送達

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US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DE69433922T2 (de) 1993-11-09 2005-07-28 Medical College Of Ohio, Toledo Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
DK0733103T3 (da) 1993-11-09 2004-07-12 Univ Johns Hopkins Fremstilling af höje titre af rekombinante AAV vektorer
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
CA2207927A1 (en) 1994-12-06 1996-06-13 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
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
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US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
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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
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
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
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2013078316A1 (en) 2011-11-23 2013-05-30 Nationwide Children's Hospital, Inc. Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
EP2879719B1 (en) * 2012-08-01 2018-07-04 Nationwide Children's Hospital Intrathecal delivery of recombinant adeno-associated virus 9
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JP6202701B2 (ja) 2014-03-21 2017-09-27 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及びプログラム
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
CA2967468A1 (en) * 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
SG10202105090WA (en) * 2016-11-17 2021-06-29 Nationwide Childrens Hospital Inc Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2

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EP3921430A1 (en) 2021-12-15
EA202192160A1 (ru) 2021-11-17
US12404523B2 (en) 2025-09-02
CL2021002049A1 (es) 2022-04-01
US20220127641A1 (en) 2022-04-28
MX2021009401A (es) 2021-11-12
SG11202107986YA (en) 2021-08-30
KR20210124300A (ko) 2021-10-14
JP2025138648A (ja) 2025-09-25
WO2020163300A9 (en) 2021-04-15
AR118005A1 (es) 2021-09-08
WO2020163300A1 (en) 2020-08-13
IL285328A (en) 2021-09-30
JP2022519596A (ja) 2022-03-24
CA3129077A1 (en) 2020-08-13
JP7691367B2 (ja) 2025-06-11
CN113646436A (zh) 2021-11-12
AU2020217708A1 (en) 2021-08-19
BR112021015285A2 (pt) 2021-10-05

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