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 (enExample) 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 (enExample)
JP (2) JP7049247B2 (enExample)
CA (1) CA3005249A1 (enExample)
WO (1) WO2017106292A1 (enExample)

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US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
CN107109411B (zh) 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加
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
CN116059236A (zh) 2015-12-14 2023-05-05 冷泉港实验室 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CA3049980A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
CA3059348A1 (en) 2017-04-11 2018-10-18 Regeneron Pharmaceuticals, Inc. Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family
KR20260009385A (ko) 2017-08-25 2026-01-19 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
MY207352A (en) 2018-03-21 2025-02-21 Regeneron Pharma 17-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
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
KR20210134003A (ko) 2019-02-27 2021-11-08 스톡 테라퓨틱스, 인크. 병태 및 질환의 치료를 위한 안티센스 올리고머
KR20220104677A (ko) * 2019-08-19 2022-07-26 스톡 테라퓨틱스, 인크. 스플라이싱 및 단백질 발현을 조절하기 위한 조성물 및 방법
WO2021202807A1 (en) 2020-03-31 2021-10-07 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Proteomic screening for lysosomal storage diseases
WO2021203031A2 (en) 2020-04-02 2021-10-07 Seattle Children's Hospital D/B/A Seattle Children's Research Institute Antibodies that specifically bind peptides associated with the primary immunodeficiencies: wiskott-aldrich syndrome and x-linked agammaglobulinemia
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases

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
JP2008520244A (ja) * 2004-11-19 2008-06-19 アカディア ファーマシューティカルズ インコーポレイテッド ホルモン核内受容体のリガンドの同定方法
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
US20140128449A1 (en) * 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing
WO2014078749A1 (en) * 2012-11-15 2014-05-22 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
WO2015035091A1 (en) * 2013-09-04 2015-03-12 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
CN107109411B (zh) * 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加

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