JP2018531600A5 - - Google Patents

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JP2018531600A5
JP2018531600A5 JP2018518426A JP2018518426A JP2018531600A5 JP 2018531600 A5 JP2018531600 A5 JP 2018531600A5 JP 2018518426 A JP2018518426 A JP 2018518426A JP 2018518426 A JP2018518426 A JP 2018518426A JP 2018531600 A5 JP2018531600 A5 JP 2018531600A5
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pharmaceutical composition
composition according
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JP2018518426A
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JP6923517B2 (ja
JP2018531600A (ja
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Priority claimed from GB1517937.7A external-priority patent/GB2546719B/en
Priority claimed from GB1614744.9A external-priority patent/GB2559100A/en
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Priority claimed from PCT/GB2016/053136 external-priority patent/WO2017060731A1/en
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Priority to JP2021123345A priority Critical patent/JP2021180669A/ja
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Priority to JP2023171631A priority patent/JP2024009853A/ja
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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)

Priority Applications (2)

Application Number Priority Date Filing Date Title
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
GB1517937.7A 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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JP2018531600A JP2018531600A (ja) 2018-11-01
JP2018531600A5 true JP2018531600A5 (enExample) 2019-11-14
JP6923517B2 JP6923517B2 (ja) 2021-08-18

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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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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

Country Status (10)

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US (4) US10196639B2 (enExample)
EP (1) EP3359685B1 (enExample)
JP (3) JP6923517B2 (enExample)
KR (2) KR102422625B1 (enExample)
CN (1) CN108603230A (enExample)
AU (3) AU2016334804B2 (enExample)
CA (1) CA3000971A1 (enExample)
IL (2) IL258528B2 (enExample)
SG (1) SG11201802870RA (enExample)
WO (1) WO2017060731A1 (enExample)

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JP7603594B2 (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
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
US11129829B2 (en) 2019-06-17 2021-09-28 Skyhawk Therapeutics, Inc. Methods for modulating splicing
CA3147970A1 (en) * 2019-08-19 2021-02-25 Isabel AZNAREZ Compositions and methods for modulating splicing and protein expression
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
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças
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
WO2023144263A1 (en) * 2022-01-26 2023-08-03 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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