JP2022540446A - Ｒｎａ標的ノックダウン及び置換の組成物及び使用方法 - Google Patents

Ｒｎａ標的ノックダウン及び置換の組成物及び使用方法 Download PDF

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JP2022540446A
JP2022540446A JP2022500898A JP2022500898A JP2022540446A JP 2022540446 A JP2022540446 A JP 2022540446A JP 2022500898 A JP2022500898 A JP 2022500898A JP 2022500898 A JP2022500898 A JP 2022500898A JP 2022540446 A JP2022540446 A JP 2022540446A
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rna
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デイビッド エー． ネルズ，
ランジャン バトラ，
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ロックアネイビオ， インコーポレイテッド
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    • C12N15/113—Non-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
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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JP2022500898A 2019-07-10 2020-07-10 Ｒｎａ標的ノックダウン及び置換の組成物及び使用方法 Pending JP2022540446A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962872604P 2019-07-10 2019-07-10
US62/872,604 2019-07-10
US202062968819P 2020-01-31 2020-01-31
US62/968,819 2020-01-31
PCT/US2020/041634 WO2021007529A1 (en) 2019-07-10 2020-07-10 Rna-targeting knockdown and replacement compositions and methods for use

Publications (2)

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JP2022540446A true JP2022540446A (ja) 2022-09-15
JP2022540446A5 JP2022540446A5 (https=) 2023-07-18

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US (1) US20210009987A1 (https=)
EP (1) EP3997227A1 (https=)
JP (1) JP2022540446A (https=)
CN (1) CN114450031A (https=)
AU (1) AU2020310201A1 (https=)
CA (1) CA3145309A1 (https=)
WO (1) WO2021007529A1 (https=)

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WO2011160052A2 (en) 2010-06-18 2011-12-22 The University Of North Carolina At Chapel Hill Methods and compositions for sequence specific rna endonucleases
EP3901262A1 (en) * 2020-04-20 2021-10-27 Universität Regensburg Compositions for use in treating autosomal dominant best1-related retinopathies
EP4216964A4 (en) * 2020-09-24 2025-02-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing apoe expression
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
WO2022221278A1 (en) * 2021-04-12 2022-10-20 Locanabio, Inc. Compositions and methods comprising hybrid promoters
WO2022241059A2 (en) * 2021-05-11 2022-11-17 Mammoth Biosciences, Inc. Effector proteins and methods of use
WO2023015153A2 (en) * 2021-08-02 2023-02-09 President And Fellows Of Harvard College ANTISENSE OLIGONUCLEOTIDE TARGETING APOE ε4 AND USES THEREOF
EP4419688A2 (en) * 2021-10-21 2024-08-28 Prime Medicine, Inc. Genome editing compositions and methods for treatment of usher syndrome type 3
US12173368B2 (en) * 2022-03-11 2024-12-24 New England Biolabs, Inc. Compositions and analysis of dephosphorylated oligoribonucleotides
EP4493702A4 (en) * 2022-03-18 2026-04-01 Univ Florida METHODS AND COMPOSITIONS FOR TREATING TNNT2-RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
WO2023215761A1 (en) * 2022-05-03 2023-11-09 Tacit Therapeutics, Inc. Localization of trans-splicing nucleic acid molecules to and within the cellular nucleus
WO2024196955A2 (en) * 2023-03-20 2024-09-26 Duke University Apoe-targeted microglia-specific gene therapy for neurological diseases
WO2025043118A1 (en) * 2023-08-23 2025-02-27 Ohio State Innovation Foundation Making tiny rnas inside the cell
WO2025043278A1 (en) * 2023-08-25 2025-03-06 PYC Therapeutics Limited Gene-mediated conditions
WO2025159427A1 (ko) * 2024-01-26 2025-07-31 서울대학교산학협력단 신규한 apoe 안티센스 올리고뉴클레오타이드 및 이의 용도
WO2025217591A1 (en) * 2024-04-12 2025-10-16 Strand Therapeutics Inc. Human-derived synthetic regulators and uses thereof
WO2026010243A1 (ko) * 2024-07-03 2026-01-08 주식회사 아바타테라퓨틱스 Crispr/cas 시스템을 사용하는 best1 유전자 편집 방법

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WO2016176690A2 (en) * 2015-04-30 2016-11-03 The Trustees Of Columbia University In The City Of New York Gene therapy for autosomal dominant diseases
JP2018507713A (ja) * 2015-03-13 2018-03-22 ザ ジャクソン ラボラトリー 三構成要素ＣＲＩＳＰＲ／Ｃａｓ複合体系およびその使用

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ES2714007T3 (es) 2007-04-09 2019-05-24 Univ Florida Composiciones de vectores rAAV que tienen proteínas de la cápside modificadas en tirosina y métodos para su uso
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
CN101895633A (zh) 2010-07-14 2010-11-24 中兴通讯股份有限公司 一种移动终端及其解锁方法
WO2012068627A1 (en) 2010-11-24 2012-05-31 The University Of Western Australia Peptides for the specific binding of rna targets
US9951349B2 (en) * 2011-09-27 2018-04-24 Yale University Compositions and methods for transient expression of recombinant RNA
DK2784157T3 (da) 2011-10-21 2019-10-21 Univ Kyushu Nat Univ Corp Designfremgangsmåde til et rna-bindende protein under anvendelse af ppr-motiv og anvendelse deraf
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WO2021007529A1 (en) 2021-01-14
EP3997227A1 (en) 2022-05-18
US20210009987A1 (en) 2021-01-14
AU2020310201A1 (en) 2022-01-27
CA3145309A1 (en) 2021-01-14
CN114450031A (zh) 2022-05-06

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