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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    • C12N15/113—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

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Application Number Priority Date Filing Date Title
GB1517937.7A GB2546719B (en) 2015-10-09 2015-10-09 Modulation of gene expression and screening for deregulated protein expression
GB1517937.7 2015-10-09
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)
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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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EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
MX2020001425A (es) 2017-08-04 2020-08-06 Skyhawk Therapeutics Inc Metodos y composiciones para modular el empalme.
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
EP3781214A4 (en) * 2018-04-17 2022-04-13 Applied StemCell, Inc. Compositions and methods for treating spinal muscular atrophy
WO2019232251A1 (en) * 2018-05-30 2019-12-05 The Regents Of The University Of California Gene editing of monogenic disorders in human hematopoietic stem cells -- correction of x-linked hyper-igm syndrome (xhim)
CN114007613A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
JP7603595B2 (ja) 2019-02-06 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
US11129829B2 (en) * 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CN114746550A (zh) * 2019-08-19 2022-07-12 斯托克制药公司 用于调节剪接和蛋白质表达的组合物和方法
IL295542A (en) * 2020-02-12 2022-10-01 Childrens Hospital 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
WO2022162020A2 (en) * 2021-01-26 2022-08-04 Syddansk Universitet 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
EP4469572A1 (en) * 2022-01-26 2024-12-04 Syddansk Universitet Allele specific splice switching oligonucleotides targeting 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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