JP6923517B2 - 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング - Google Patents

遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング Download PDF

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JP6923517B2
JP6923517B2 JP2018518426A JP2018518426A JP6923517B2 JP 6923517 B2 JP6923517 B2 JP 6923517B2 JP 2018518426 A JP2018518426 A JP 2018518426A JP 2018518426 A JP2018518426 A JP 2018518426A JP 6923517 B2 JP6923517 B2 JP 6923517B2
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polynucleic acid
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pharmaceutical composition
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JP2018531600A5 (enExample
JP2018531600A (ja
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ヴォアチョフスキー，イゴール
クラロヴィコヴァ，ヤナ
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ユニバーシティ・オブ・サザンプトン
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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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JP2018518426A 2015-10-09 2016-10-07 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング Active JP6923517B2 (ja)

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JP2021123345A JP2021180669A (ja) 2015-10-09 2021-07-28 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング
JP2023171631A JP2024009853A (ja) 2015-10-09 2023-10-02 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB1517937.7 2015-10-09
GB1517937.7A GB2546719B (en) 2015-10-09 2015-10-09 Modulation of gene expression and screening for deregulated protein expression
GB1614744.9A GB2559100A (en) 2016-08-31 2016-08-31 Modulation of gene expression and screening for deregulated protein expression
GB1614744.9 2016-08-31
PCT/GB2016/053136 WO2017060731A1 (en) 2015-10-09 2016-10-07 Modulation of gene expression and screening for deregulated protein expression

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JP2021123345A Pending JP2021180669A (ja) 2015-10-09 2021-07-28 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング
JP2023171631A Pending JP2024009853A (ja) 2015-10-09 2023-10-02 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング

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JP2023171631A Pending JP2024009853A (ja) 2015-10-09 2023-10-02 遺伝子発現の調節及び脱制御されたタンパク質発現のスクリーニング

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US (4) US10196639B2 (enExample)
EP (1) EP3359685A1 (enExample)
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KR (2) KR20220105174A (enExample)
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US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases

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EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
KR20220105174A (ko) 2015-10-09 2022-07-26 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017106377A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
IL272413B (en) 2017-08-04 2022-09-01 Skyhawk Therapeutics Inc Methods and compositions for fusion modulation
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
EP3781214A4 (en) * 2018-04-17 2022-04-13 Applied StemCell, Inc. Compositions and methods for treating spinal muscular atrophy
US12492411B2 (en) 2018-05-30 2025-12-09 The Regents Of The University Of California Gene editing of monogenic disorders in human hematopoietic stem cells—correction of X-linked agammaglobulinemia (XLA)
EP3920926A4 (en) 2019-02-05 2022-10-19 Skyhawk Therapeutics, Inc. Methods and compositions for modulating splicing
JP7603592B2 (ja) 2019-02-05 2024-12-20 スカイホーク・セラピューティクス・インコーポレーテッド スプライシングを調節するための方法および組成物
EP3920928A4 (en) 2019-02-06 2022-09-28 Skyhawk Therapeutics, Inc. METHODS AND COMPOSITIONS FOR MODULATION OF SPLICING
CN113661162A (zh) 2019-02-06 2021-11-16 斯基霍克疗法公司 用于调节剪接的方法和组合物
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
JP2022544702A (ja) * 2019-08-19 2022-10-20 ストーク セラピューティクス，インク． スプライシングおよびタンパク質発現を調節するための組成物および方法
US20230340498A1 (en) * 2020-02-12 2023-10-26 The Children's Hospital Of Philadelphia Compositions and methods for inducible alternative splicing regulation of gene expression
CN111349649B (zh) * 2020-03-16 2020-11-17 三峡大学 一种用于双孢蘑菇的基因编辑的方法及应用
CA3204779A1 (en) * 2021-01-26 2022-08-04 Brage Storstein Andresen Splice switching oligonucleotides targeting pseudoexons
CN112908470B (zh) * 2021-02-08 2023-10-03 深圳市人民医院 一种基于rna结合蛋白基因的肝细胞癌预后评分系统及其应用
CA3247528A1 (en) * 2022-01-26 2023-08-03 Syddansk Universitet SPLIT-SWITCHING ALLELE-SPECIFIC OLIGONUCLEOTIDES TARGETTING PSEUDOEXONS
CN119424374A (zh) * 2024-11-14 2025-02-14 中国药科大学 一种具有细菌靶向性的岩藻多糖改良复合物及其制备方法和应用

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