JP7684296B2 - Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法 - Google Patents

Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法 Download PDF

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JP7684296B2
JP7684296B2 JP2022529542A JP2022529542A JP7684296B2 JP 7684296 B2 JP7684296 B2 JP 7684296B2 JP 2022529542 A JP2022529542 A JP 2022529542A JP 2022529542 A JP2022529542 A JP 2022529542A JP 7684296 B2 JP7684296 B2 JP 7684296B2
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raav
composition
ighmbp2
seq
virus
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JP2023502474A (ja
JPWO2021102435A5 (https=
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キャスリン クリスティーン マイヤー，
シビ リカイト，
ケビン ファウスト，
ブライアン ケー． カスパー，
モニカ ニッツァルド，
ステファニア パオラ コルティ，
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Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
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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
    • A61K48/005—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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/04—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; involved in cellular and subcellular movement (3.6.4)
    • C12Y306/04012—DNA helicase (3.6.4.12)
    • 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

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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2022529542A 2019-11-22 2020-11-23 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法 Active JP7684296B2 (ja)

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JP2025081888A JP2025124694A (ja) 2019-11-22 2025-05-15 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法

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US201962939270P 2019-11-22 2019-11-22
US62/939,270 2019-11-22
PCT/US2020/061863 WO2021102435A1 (en) 2019-11-22 2020-11-23 Materials and methods for treatment of disorders associated with the ighmbp2 gene

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JP2023502474A JP2023502474A (ja) 2023-01-24
JPWO2021102435A5 JPWO2021102435A5 (https=) 2023-11-30
JP2023502474A5 JP2023502474A5 (https=) 2023-11-30
JP7684296B2 true JP7684296B2 (ja) 2025-05-27

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JP2025081888A Pending JP2025124694A (ja) 2019-11-22 2025-05-15 Ｉｇｈｍｂｐ２遺伝子に関連する障害の治療のための材料および方法

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US (1) US20230211018A1 (https=)
EP (1) EP4061831A1 (https=)
JP (2) JP7684296B2 (https=)
AU (1) AU2020385387A1 (https=)
IL (1) IL293210A (https=)
WO (1) WO2021102435A1 (https=)

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AU2023360998A1 (en) 2022-10-11 2025-05-22 Research Institute At Nationwide Children's Hospital Adeno-associated virus delivery to treat spinal muscular atrophy with respiratory distress type 1 (smard1) and charcot-marie-tooth type 2s (cmt2s)

Citations (3)

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WO2010134939A2 (en) 2008-12-19 2010-11-25 Zirus, Inc. Mammalian genes involved in infection
WO2018094251A1 (en) 2016-11-17 2018-05-24 Kaspar Brian K Intrathecal delivery of recombinant adeno-associated virus encoding methyl-cpg binding protein 2
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II

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PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
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
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US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
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AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
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EP4061831A1 (en) 2022-09-28
JP2025124694A (ja) 2025-08-26
JP2023502474A (ja) 2023-01-24
AU2020385387A1 (en) 2022-06-02
IL293210A (en) 2022-07-01
US20230211018A1 (en) 2023-07-06
WO2021102435A8 (en) 2022-07-07

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