JP2019500346A5 - - Google Patents

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Publication number
JP2019500346A5
JP2019500346A5 JP2018529237A JP2018529237A JP2019500346A5 JP 2019500346 A5 JP2019500346 A5 JP 2019500346A5 JP 2018529237 A JP2018529237 A JP 2018529237A JP 2018529237 A JP2018529237 A JP 2018529237A JP 2019500346 A5 JP2019500346 A5 JP 2019500346A5
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JP
Japan
Prior art keywords
mrna
peroxisome proliferator
activated receptor
seq
pharmaceutical composition
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JP2018529237A
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English (en)
Japanese (ja)
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JP7049247B2 (ja
JP2019500346A (ja
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Priority claimed from PCT/US2016/066576 external-priority patent/WO2017106292A1/en
Publication of JP2019500346A publication Critical patent/JP2019500346A/ja
Publication of JP2019500346A5 publication Critical patent/JP2019500346A5/ja
Priority to JP2022013876A priority Critical patent/JP2022062143A/ja
Application granted granted Critical
Publication of JP7049247B2 publication Critical patent/JP7049247B2/ja
Expired - Fee Related legal-status Critical Current
Anticipated expiration legal-status Critical

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JP2018529237A 2015-12-14 2016-12-14 腎臓病の処置のための組成物と方法 Expired - Fee Related JP7049247B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022013876A JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562267242P 2015-12-14 2015-12-14
US62/267,242 2015-12-14
PCT/US2016/066576 WO2017106292A1 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of kidney diseases

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2022013876A Division JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

Publications (3)

Publication Number Publication Date
JP2019500346A JP2019500346A (ja) 2019-01-10
JP2019500346A5 true JP2019500346A5 (https=) 2020-01-30
JP7049247B2 JP7049247B2 (ja) 2022-04-06

Family

ID=59057520

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2018529237A Expired - Fee Related JP7049247B2 (ja) 2015-12-14 2016-12-14 腎臓病の処置のための組成物と方法
JP2022013876A Pending JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

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JP2022013876A Pending JP2022062143A (ja) 2015-12-14 2022-02-01 腎臓病の処置のための組成物と方法

Country Status (4)

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EP (1) EP3390666A4 (https=)
JP (2) JP7049247B2 (https=)
CA (1) CA3005249A1 (https=)
WO (1) WO2017106292A1 (https=)

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WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
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
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
RU2019126483A (ru) 2017-01-23 2021-02-24 Ридженерон Фармасьютикалз, Инк. Варианты 17-бета-гидроксистероиддегидрогеназы 13 (hsd17b13) и их применение
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
KR102318434B1 (ko) 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
AU2018348195B2 (en) 2017-10-11 2025-05-15 Regeneron Pharmaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
AU2019239971B2 (en) 2018-03-21 2025-09-11 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
EP3861018A4 (en) 2018-10-05 2022-10-26 Seattle Children's Hospital d/b/a Seattle Children's Research Institute NEWBORN SCREENING FOR PRIMARY IMMUNODEFICIENCY CYSTINosis AND WILSON'S DISEASE
US20220090087A1 (en) * 2019-01-23 2022-03-24 The Florey Institute Of Neuroscience And Mental Health Antisense oligonucleotides targeting scn2a retained introns
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
EP4017979A4 (en) * 2019-08-19 2024-03-27 Stoke Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION
JP2023519969A (ja) 2020-03-31 2023-05-15 シアトル・チルドレンズ・ホスピタル・ドゥーイング／ビジネス／アズ・シアトル・チルドレンズ・リサーチ・インスティテュート リソソーム蓄積症に関するプロテオミクススクリーニング
US12523662B2 (en) 2020-04-02 2026-01-13 Seattle Children's Hospital Antibodies that specifically bind peptides associated with the primary immunodeficiencies: Wiskott-Aldrich syndrome and x-linked agammaglobulinemia
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030224514A1 (en) * 2002-05-31 2003-12-04 Isis Pharmaceuticals Inc. Antisense modulation of PPAR-delta expression
US20060134670A1 (en) * 2004-11-19 2006-06-22 Fabrice Piu Enabling tools to identify ligands for hormone nuclear receptors
US20110269735A1 (en) * 2010-04-19 2011-11-03 Celera Corporation Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof
WO2012138487A2 (en) * 2011-04-07 2012-10-11 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
US9909128B2 (en) * 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
EP3041958B1 (en) * 2013-09-04 2019-12-04 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
AU2015327836B2 (en) * 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output

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