JP7397488B2 - Sod1二重発現ベクターおよびその使用 - Google Patents

Sod1二重発現ベクターおよびその使用 Download PDF

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JP7397488B2
JP7397488B2 JP2020516885A JP2020516885A JP7397488B2 JP 7397488 B2 JP7397488 B2 JP 7397488B2 JP 2020516885 A JP2020516885 A JP 2020516885A JP 2020516885 A JP2020516885 A JP 2020516885A JP 7397488 B2 JP7397488 B2 JP 7397488B2
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nucleic acid
sod1
promoter
isolated nucleic
region
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JP2020535804A5 (enExample
JP2020535804A (ja
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ミュラー,クリスティアン
ブラウン,ロバート,エイチ.,ジュニア
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ユニバーシティ オブ マサチューセッツ
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
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    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/44Oxidoreductases (1)
    • A61K38/446Superoxide dismutase (1.15)
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K48/005Medicinal 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
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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
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    • C12Y115/01Oxidoreductases acting on superoxide as acceptor (1.15) with NAD or NADP as acceptor (1.15.1)
    • C12Y115/01001Superoxide dismutase (1.15.1.1)
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/141MicroRNAs, miRNAs
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    • C12N2750/14011Parvoviridae
    • C12N2750/14041Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/14011Parvoviridae
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    • 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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JP2020516885A 2017-09-22 2018-09-21 Sod1二重発現ベクターおよびその使用 Active JP7397488B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023198877A JP7717398B2 (ja) 2017-09-22 2023-11-24 Sod1二重発現ベクターおよびその使用
JP2025119064A JP2025169472A (ja) 2017-09-22 2025-07-15 Sod1二重発現ベクターおよびその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762561932P 2017-09-22 2017-09-22
US62/561,932 2017-09-22
PCT/US2018/052173 WO2019060686A1 (en) 2017-09-22 2018-09-21 NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF

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JP2023198877A Division JP7717398B2 (ja) 2017-09-22 2023-11-24 Sod1二重発現ベクターおよびその使用

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JP2020535804A JP2020535804A (ja) 2020-12-10
JP2020535804A5 JP2020535804A5 (enExample) 2021-11-04
JP7397488B2 true JP7397488B2 (ja) 2023-12-13

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JP2020516885A Active JP7397488B2 (ja) 2017-09-22 2018-09-21 Sod1二重発現ベクターおよびその使用
JP2023198877A Active JP7717398B2 (ja) 2017-09-22 2023-11-24 Sod1二重発現ベクターおよびその使用
JP2025119064A Pending JP2025169472A (ja) 2017-09-22 2025-07-15 Sod1二重発現ベクターおよびその使用

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US (3) US11739330B2 (enExample)
EP (1) EP3684937A4 (enExample)
JP (3) JP7397488B2 (enExample)
CN (2) CN111448321A (enExample)
AU (2) AU2018338188B2 (enExample)
CA (1) CA3075643A1 (enExample)
WO (1) WO2019060686A1 (enExample)

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EP3622073A4 (en) 2017-05-09 2021-01-06 University of Massachusetts METHOD FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
US11739330B2 (en) 2017-09-22 2023-08-29 University Of Massachusetts SOD1 dual expression vectors and uses thereof
AR123524A1 (es) * 2020-09-16 2022-12-14 Biogen Ma Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela) con aav-mir-sod1
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
US20250101424A1 (en) * 2023-09-25 2025-03-27 California Institute Of Technology Mirna circuits for controlled gene expression

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