JP2015523082A5 - - Google Patents

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Publication number
JP2015523082A5
JP2015523082A5 JP2015521573A JP2015521573A JP2015523082A5 JP 2015523082 A5 JP2015523082 A5 JP 2015523082A5 JP 2015521573 A JP2015521573 A JP 2015521573A JP 2015521573 A JP2015521573 A JP 2015521573A JP 2015523082 A5 JP2015523082 A5 JP 2015523082A5
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JP
Japan
Prior art keywords
oligonucleotide
sequence
target rna
change
seq
Prior art date
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Application number
JP2015521573A
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English (en)
Japanese (ja)
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JP2015523082A (ja
JP6373833B2 (ja
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Priority claimed from PCT/NL2013/050534 external-priority patent/WO2014011053A1/en
Publication of JP2015523082A publication Critical patent/JP2015523082A/ja
Publication of JP2015523082A5 publication Critical patent/JP2015523082A5/ja
Application granted granted Critical
Publication of JP6373833B2 publication Critical patent/JP6373833B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2015521573A 2012-07-12 2013-07-12 生細胞に存在する標的rna分子の配列を変化させるためのオリゴヌクレオチド Expired - Fee Related JP6373833B2 (ja)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201261670681P 2012-07-12 2012-07-12
US61/670,681 2012-07-12
US201261718801P 2012-10-26 2012-10-26
US61/718,801 2012-10-26
EP13166465.8 2013-05-03
EP13166465 2013-05-03
EP13172515.2 2013-06-18
EP13172515 2013-06-18
EP13173818.9 2013-06-26
EP13173818 2013-06-26
PCT/NL2013/050534 WO2014011053A1 (en) 2012-07-12 2013-07-12 Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell

Publications (3)

Publication Number Publication Date
JP2015523082A JP2015523082A (ja) 2015-08-13
JP2015523082A5 true JP2015523082A5 (enExample) 2016-07-14
JP6373833B2 JP6373833B2 (ja) 2018-08-15

Family

ID=49916369

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015521573A Expired - Fee Related JP6373833B2 (ja) 2012-07-12 2013-07-12 生細胞に存在する標的rna分子の配列を変化させるためのオリゴヌクレオチド

Country Status (23)

Country Link
US (2) US9605255B2 (enExample)
EP (2) EP2852668B1 (enExample)
JP (1) JP6373833B2 (enExample)
KR (1) KR20150037968A (enExample)
CN (1) CN104640986B (enExample)
AU (1) AU2013287353B2 (enExample)
BR (1) BR112015000710A8 (enExample)
CA (1) CA2878934A1 (enExample)
CY (1) CY1117858T1 (enExample)
DK (2) DK3103872T3 (enExample)
ES (2) ES2584856T3 (enExample)
HR (1) HRP20160854T1 (enExample)
HU (1) HUE029977T2 (enExample)
IL (1) IL236654B (enExample)
MX (1) MX356625B (enExample)
NZ (1) NZ703824A (enExample)
PL (1) PL2852668T3 (enExample)
PT (1) PT2852668T (enExample)
RS (1) RS54944B1 (enExample)
RU (1) RU2663110C2 (enExample)
SI (1) SI2852668T1 (enExample)
SM (1) SMT201600234B (enExample)
WO (1) WO2014011053A1 (enExample)

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US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
US8802868B2 (en) 2010-03-25 2014-08-12 Vertex Pharmaceuticals Incorporated Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide
RU2569678C2 (ru) 2010-04-22 2015-11-27 Вертекс Фармасьютикалз Инкорпорейтед Способ получения циклоалкилкарбоксамидо-индольных соединений
KR20150037968A (ko) 2012-07-12 2015-04-08 프로큐알 테라퓨틱스 엔.브이. 살아있는 세포 내에 존재하는 목적 rna 분자의 서열을 변화시키기 위한 올리고뉴클레오타이드
PL3925607T5 (pl) 2014-04-15 2026-06-01 Vertex Pharmaceuticals Incorporated Kompozycje farmaceutyczne do leczenia chorób, w których pośredniczy mukowiscydozowy przezbłonowy regulator przewodnictwa
US10278991B2 (en) 2014-05-14 2019-05-07 Targimmune Therapeutics Ag Polyethyleneimine polyethyleneglycol vectors
CA2963945C (en) 2014-10-07 2023-01-10 Vertex Pharmaceuticals Incorporated Co-crystals of modulators of cystic fibrosis transmembrane conductance regulator
GB201418892D0 (en) * 2014-10-23 2014-12-10 Proqr Therapeutics B V DNA editing
CN107532162A (zh) * 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
AU2015367378A1 (en) * 2014-12-17 2017-06-15 Proqr Therapeutics Ii B.V. Targeted RNA editing
US20210401869A1 (en) * 2015-02-20 2021-12-30 Rosalind Franklin University Of Medicine And Science Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
GB201507926D0 (en) * 2015-05-08 2015-06-24 Proqr Therapeutics N V Improved treatments using oligonucleotides
AU2016335032B2 (en) 2015-10-05 2022-04-14 Proqr Therapeutics Ii B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
CA3001723A1 (en) 2015-10-15 2017-04-20 City Of Hope Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
CA3022319A1 (en) * 2016-05-06 2017-11-09 Tod M. Woolf Improved methods for genome editing with and without programmable nucleases
EP3475424A1 (en) 2016-06-22 2019-05-01 ProQR Therapeutics II B.V. Single-stranded rna-editing oligonucleotides
WO2018041973A1 (en) * 2016-09-01 2018-03-08 Proqr Therapeutics Ii B.V. Chemically modified single-stranded rna-editing oligonucleotides
US11274300B2 (en) 2017-01-19 2022-03-15 Proqr Therapeutics Ii B.V. Oligonucleotide complexes for use in RNA editing
CN110612353A (zh) 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CN107267516B (zh) * 2017-07-28 2020-09-29 佛山科学技术学院 双sgRNA介导的基因精确修饰方法及应用
EP3697904A1 (en) * 2017-10-17 2020-08-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment for cystic fibrosis
CN110573184B (zh) * 2018-02-09 2025-05-13 俄亥俄州立创新基金会 Rna纳米结构,其制备方法和用途
ES2959701T3 (es) 2018-03-27 2024-02-27 Univ Rochester Moléculas de ácido nucleico para pseudouridilación
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
CA3127241A1 (en) * 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
AU2020245332B2 (en) * 2019-03-28 2026-04-30 Splisense Ltd. Compositions and methods for treating cystic fibrosis
US11827880B2 (en) 2019-12-02 2023-11-28 Shape Therapeutics Inc. Therapeutic editing
CN113397996A (zh) * 2021-07-23 2021-09-17 河南省人民医院 一种抗菌漱口水及其制备方法
CN114917227B (zh) * 2022-05-19 2023-10-31 中国人民解放军海军军医大学 依伐卡托在制备抗蜱传脑炎病毒、西尼罗病毒、黄热病毒和基孔肯雅热病毒感染药中的应用
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KR20150037968A (ko) 2012-07-12 2015-04-08 프로큐알 테라퓨틱스 엔.브이. 살아있는 세포 내에 존재하는 목적 rna 분자의 서열을 변화시키기 위한 올리고뉴클레오타이드
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