JP2019500346A5 - - Google Patents

Download PDF

Info

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
Authority
JP
Japan
Prior art keywords
mrna
peroxisome proliferator
activated receptor
seq
pharmaceutical composition
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Granted
Application number
JP2018529237A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019500346A (ja
JP7049247B2 (ja
Filing date
Publication date
Application filed filed Critical
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

Links

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

Family Applications After (1)

Application Number Title Priority Date Filing Date
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)

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

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP3359685B1 (en) 2015-10-09 2026-01-28 University Of Southampton Modulation of gene expression for deregulated protein expression
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
EP3933041B1 (en) 2015-12-14 2024-01-31 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant retardation
IL314915B1 (en) 2017-01-23 2026-02-01 Regeneron Pharma Variants of 17-beta-hydroxysteroid dehydrogenase and their uses
KR20190139869A (ko) 2017-04-11 2019-12-18 리제너론 파마슈티칼스 인코포레이티드 하이드록시스테로이드 (17-베타) 탈수소효소(hsd17b) 패밀리의 구성원의 조절인자의 활성도를 스크리닝하기 위한 검정
SG11202001590RA (en) 2017-08-25 2020-03-30 Stoke Therapeutics Inc Antisense oligomers for treatment of conditions and diseases
US10961583B2 (en) 2017-10-11 2021-03-30 Regeneron Phramaceuticals, Inc. Inhibition of HSD17B13 in the treatment of liver disease in patients expressing the PNPLA3 I148M variation
JP7507093B2 (ja) 2018-03-21 2024-06-27 リジェネロン・ファーマシューティカルズ・インコーポレイテッド １７β－水酸化ステロイド脱水素酵素１３型（ＨＳＤ１７Ｂ１３）ｉＲＮＡ組成物およびその使用方法
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
CN121253826A (zh) 2018-10-05 2026-01-02 西雅图儿童医院以西雅图儿童研究机构名义经营 针对原发性免疫缺陷、胱氨酸病和威尔逊病的新生儿筛查
AU2020210924A1 (en) * 2019-01-23 2021-09-16 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
EP4017979A4 (en) * 2019-08-19 2024-03-27 Stoke Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING PROTEIN SPLICING AND EXPRESSION
EP4126966A4 (en) 2020-03-31 2024-08-07 Seattle Children's Hospital d/b/a Seattle Children's Research Institute PROTEOMIC SCREENING FOR LYSOSOMAL STORAGE DISEASES
CN115298215A (zh) 2020-04-02 2022-11-04 西雅图儿童医院以西雅图儿童研究机构名义经营 特异性地结合与原发性免疫缺陷：维斯科特-奥尔德里奇综合征和x连锁无丙种球蛋白血症相关的肽的抗体
BR112022022889A2 (pt) 2020-05-11 2023-04-04 Stoke Therapeutics Inc Oligômeros antissentido de opa1 para tratamento de condições e doenças

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
WO2006055786A2 (en) * 2004-11-19 2006-05-26 Acadia Pharmaceuticals Inc. Methods to identify ligands of 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
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
PL3041958T4 (pl) * 2013-09-04 2020-11-02 Cold Spring Harbor Laboratory Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi
CN107109411B (zh) * 2014-10-03 2022-07-01 冷泉港实验室 核基因输出的定向增加

Similar Documents

Publication Publication Date Title
JP2019500346A5 (https=)
JP2019501892A5 (https=)
JP2019500347A5 (https=)
JP2019500349A5 (https=)
JP2018538288A5 (https=)
JP2018538287A5 (https=)
JP2024056778A5 (https=)
JP2019500350A5 (https=)
US20240011028A9 (en) Compositions and Methods Using SNRNA Components
JP2019500345A5 (https=)
AU2017379073B2 (en) Compositions and methods for treating alpha-1 antitrypsin deficiency
EP2027267B1 (en) Means and method for inducing exon-skipping
CN107406852B (zh) 用于莱伯氏先天性黑蒙的寡核苷酸疗法
AU2010335039B2 (en) Molecule for treating an inflammatory disorder
KR20080031164A (ko) ＳＲ 단백질의 결합의 방해에 의해 그리고 이차 ＲＮＡ구조의 방해에 의한 ｐｒｅ－ｍＲＮＡ에서 엑손 인식의조절
CN113195721A (zh) 治疗α-1抗胰蛋白酶缺乏症的组合物和方法
WO2017050836A1 (en) Antisense oligonucleotides and uses thereof
US20200407721A1 (en) Compounds of chemically modified oligonucleotides and methods of use thereof
JP2021523227A5 (https=)
JP7760130B2 (ja) Ａｐｏｃ３を標的としたアンチセンス核酸
US20250327093A1 (en) Viral vector and cancer cell proliferation inhibitor comprising the same
JPWO2019213525A5 (https=)
EP3690046A2 (en) Composition for treatment of hemophilia, comprising crispr/cas system having coagulation factor viii gene inversion correction potential
CA2999192C (en) Antisense oligonucleotides hybridizing with a key element of the polyadenylation region of a dux4 pre-mrna and uses thereof
JPWO2022169947A5 (https=)