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 PDFInfo
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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 |
| EP3201339A4 (en) | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| EP3359685A1 (en) | 2015-10-09 | 2018-08-15 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102604132B1 (ko) | 2015-12-14 | 2023-11-17 | 콜드스프링하버러보러토리 | 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머 |
| KR102712656B1 (ko) | 2017-01-23 | 2024-10-04 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
| US11479802B2 (en) | 2017-04-11 | 2022-10-25 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxy steroid (17-beta) dehydrogenase (HSD17B) family |
| SI3673080T1 (sl) | 2017-08-25 | 2024-03-29 | Stoke Therapeutics, Inc. | Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni |
| 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 |
| 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 |
| BR112020022512A2 (pt) | 2018-05-04 | 2021-05-04 | Stoke Therapeutics, Inc. | métodos e composições para tratamento de doença de armazenamento de éster de colesteril |
| EP3802829A4 (en) * | 2018-06-08 | 2022-10-19 | University of Massachusetts | ANTISENSE OLIGONUCLEOTIDES TO RESTORE DYSFERLIN PROTEIN EXPRESSION IN CELLS FROM PATIENTS WITH DYSFERLINOPATHY |
| US12422440B2 (en) | 2018-10-05 | 2025-09-23 | Seattle Children's Hospital | Newborn screening for primary immunodeficiencies, cystinosis, and Wilson disease |
| AU2020210924A1 (en) * | 2019-01-23 | 2021-09-16 | The Florey Institute Of Neuroscience And Mental Health | Antisense oligonucleotides targeting SCN2A retained introns |
| CN114746550A (zh) * | 2019-08-19 | 2022-07-12 | 斯托克制药公司 | 用于调节剪接和蛋白质表达的组合物和方法 |
| US11940448B2 (en) | 2020-03-31 | 2024-03-26 | Seattle Children's Hospital | Proteomic screening for lysosomal storage diseases |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
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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 |
| 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 |
| US9909128B2 (en) * | 2012-11-15 | 2018-03-06 | The Regents Of The University Of California | Splice modulating oligonucleotides that inhibit cancer |
| CA2930859C (en) * | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
| EP3201339A4 (en) * | 2014-10-03 | 2018-09-19 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
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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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| PNAS，1996年，Vol.93，pp.12840-12844 |
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| JP2022062143A (ja) | 2022-04-19 |
| CA3005249A1 (en) | 2017-06-22 |
| EP3390666A4 (en) | 2019-08-07 |
| JP2019500346A (ja) | 2019-01-10 |
| EP3390666A1 (en) | 2018-10-24 |
| WO2017106292A1 (en) | 2017-06-22 |
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