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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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
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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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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
KR102422625B1 (ko) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CN118325899A (zh) 2017-01-23 2024-07-12 瑞泽恩制药公司 Hsd17b13变体及其应用
RU2019135845A (ru) 2017-04-11 2021-05-11 Ридженерон Фармасьютикалз, Инк. Анализы для скрининга активности модуляторов членов семейства (17-бета)гидроксистероиддегидрогеназ (hsd17b)
CN111278991B (zh) 2017-08-25 2022-04-01 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
EP4234719B1 (en) 2017-10-11 2025-11-26 Regeneron Pharmaceuticals, Inc. Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation
BR112020018758A2 (pt) 2018-03-21 2021-01-26 Regeneron Pharmaceuticals, Inc. agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios
US12060558B2 (en) 2018-05-04 2024-08-13 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
US12422440B2 (en) 2018-10-05 2025-09-23 Seattle Children's Hospital Newborn screening for primary immunodeficiencies, cystinosis, and Wilson disease
WO2020154462A1 (en) * 2019-01-23 2020-07-30 Rogcon U.R., Inc. Antisense oligonucleotides targeting scn2a retained introns
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein 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
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

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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
CA2588646A1 (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
US9909128B2 (en) * 2012-11-15 2018-03-06 The Regents Of The University Of California Splice modulating oligonucleotides that inhibit cancer
PL3041958T3 (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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JP2022062143A (ja) 2022-04-19
EP3390666A1 (en) 2018-10-24
CA3005249A1 (en) 2017-06-22
EP3390666A4 (en) 2019-08-07
JP2019500346A (ja) 2019-01-10

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