JP6854286B2 - コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用 - Google Patents

コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用 Download PDF

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JP6854286B2
JP6854286B2 JP2018520100A JP2018520100A JP6854286B2 JP 6854286 B2 JP6854286 B2 JP 6854286B2 JP 2018520100 A JP2018520100 A JP 2018520100A JP 2018520100 A JP2018520100 A JP 2018520100A JP 6854286 B2 JP6854286 B2 JP 6854286B2
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copper
vector
atp7a
nucleic acid
composition
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JP2018533941A (ja
JP2018533941A5 (enExample
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スティーブン ジー． カーラー，
スティーブン ジー． カーラー，
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US Department of Health and Human Services
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    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K33/00—Medicinal preparations containing inorganic active ingredients
    • A61K33/24—Heavy metals; Compounds thereof
    • A61K33/34—Copper; Compounds thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
    • C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
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  • Medicinal Chemistry (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
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  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Inorganic Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2018520100A 2015-10-21 2016-10-21 コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用 Active JP6854286B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562244594P 2015-10-21 2015-10-21
US62/244,594 2015-10-21
PCT/US2016/058124 WO2017070472A1 (en) 2015-10-21 2016-10-21 Codon-optimized reduced-size atp7a cdna and uses for treatment of copper transport disorders

Related Child Applications (1)

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JP2020218556A Division JP2021052796A (ja) 2015-10-21 2020-12-28 コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用

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JP2018533941A JP2018533941A (ja) 2018-11-22
JP2018533941A5 JP2018533941A5 (enExample) 2019-11-28
JP6854286B2 true JP6854286B2 (ja) 2021-04-07

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JP2018520100A Active JP6854286B2 (ja) 2015-10-21 2016-10-21 コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用
JP2020218556A Pending JP2021052796A (ja) 2015-10-21 2020-12-28 コドン最適化された小型ＡＴＰ７Ａ ｃＤＮＡおよび銅輸送障害を処置するための使用

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US (3) US10988778B2 (enExample)
EP (1) EP3365438B1 (enExample)
JP (2) JP6854286B2 (enExample)
CN (1) CN108431216B (enExample)
AU (1) AU2016341983B8 (enExample)
CA (1) CA3001574A1 (enExample)
DK (1) DK3365438T3 (enExample)
ES (1) ES2879400T3 (enExample)
HU (1) HUE054802T2 (enExample)
PL (1) PL3365438T3 (enExample)
PT (1) PT3365438T (enExample)
WO (1) WO2017070472A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020014594A1 (en) * 2018-07-12 2020-01-16 The Taxas A&M University System Compositions for the treatment of copper deficiency and methods of use
AU2022240726A1 (en) * 2021-03-18 2023-09-21 Cyprium Therapeutics, Inc. Copper histidinate compositions and uses thereof

Family Cites Families (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1993017321A1 (en) 1992-02-25 1993-09-02 Unisearch Limited Electrothermal atomic absorption and preconcentration device
DE606516T1 (de) 1993-01-13 1995-03-16 Idemitsu Kosan Co Gegen menschlichen Ceruloplasmin monoklonaler Antikörper.
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6812339B1 (en) * 2000-09-08 2004-11-02 Applera Corporation Polymorphisms in known genes associated with human disease, methods of detection and uses thereof
KR100459644B1 (ko) 2001-03-31 2004-12-03 제노백(주) 혈액 여과지에서의 효소면역측정법을 이용한셀룰로플라스민 농도 측정 방법 및 이 방법을 이용한윌슨병 스크리닝 검사용 키트와 진단 시약
EP1625210B1 (en) 2003-05-21 2010-12-01 Genzyme Corporation Methods for producing preparations of recombinant aav virions substantially free of empty capsids
US20070161120A1 (en) 2006-01-10 2007-07-12 Viorica Lopez-Avila Ceruloplasmin Assay
MX2008012485A (es) * 2006-03-30 2008-10-10 Novartis Ag Composiciones y metodos de uso para anticuerpos de c-met.
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
WO2009097129A1 (en) 2008-01-29 2009-08-06 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
WO2010042102A1 (en) 2008-10-06 2010-04-15 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Identification of subjects likely to benefit from copper treatment
WO2010071832A1 (en) * 2008-12-19 2010-06-24 Nationwide Children's Hospital Delivery of polynucleotides across the blood brain barrier using recombinant aav9
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
JP6373763B2 (ja) * 2012-02-17 2018-08-22 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法
AU2013243949A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
AU2013243954A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides

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Publication number Publication date
CN108431216A (zh) 2018-08-21
DK3365438T3 (da) 2021-07-12
EP3365438B1 (en) 2021-04-28
WO2017070472A1 (en) 2017-04-27
CA3001574A1 (en) 2017-04-27
ES2879400T3 (es) 2021-11-22
AU2016341983B2 (en) 2022-05-26
US20250129382A1 (en) 2025-04-24
JP2021052796A (ja) 2021-04-08
HUE054802T2 (hu) 2021-09-28
JP2018533941A (ja) 2018-11-22
US10988778B2 (en) 2021-04-27
US20180312871A1 (en) 2018-11-01
AU2016341983A8 (en) 2022-10-06
US20210214751A1 (en) 2021-07-15
EP3365438A1 (en) 2018-08-29
AU2016341983A1 (en) 2018-04-26
PT3365438T (pt) 2021-07-12
US12173306B2 (en) 2024-12-24
CN108431216B (zh) 2022-08-05
PL3365438T3 (pl) 2021-10-18
AU2016341983B8 (en) 2022-10-06

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