JP7049247B2 - 腎臓病の処置のための組成物と方法 - Google Patents

腎臓病の処置のための組成物と方法 Download PDF

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JP7049247B2
JP7049247B2 JP2018529237A JP2018529237A JP7049247B2 JP 7049247 B2 JP7049247 B2 JP 7049247B2 JP 2018529237 A JP2018529237 A JP 2018529237A JP 2018529237 A JP2018529237 A JP 2018529237A JP 7049247 B2 JP7049247 B2 JP 7049247B2
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aso
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nucleobases
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アズナレズ，イザベル
エム． ナッシュ，ヒュー
クライナー，エイドリアン
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コールド スプリング ハーバー ラボラトリー
ストーク セラピューティクス，インク．
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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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)

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US201562267242P 2015-12-14 2015-12-14
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PCT/US2016/066576 WO2017106292A1 (en) 2015-12-14 2016-12-14 Compositions and methods for treatment of kidney diseases

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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
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

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US20140128449A1 (en) 2011-04-07 2014-05-08 The Board Of Regents Of The University Of Texas System Oligonucleotide modulation of splicing

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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
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

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Non-Patent Citations (1)

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CA3005249A1 (en) 2017-06-22
JP2019500346A (ja) 2019-01-10
WO2017106292A1 (en) 2017-06-22
EP3390666A1 (en) 2018-10-24
EP3390666A4 (en) 2019-08-07
JP2022062143A (ja) 2022-04-19

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