JP7054675B2 - 網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 - Google Patents
網膜色素変性症１８と網膜色素変性症１３の処置のための組成物と方法 Download PDFInfo
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| 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 |
| EP3426349A4 (en) | 2016-03-09 | 2020-01-01 | Ionis Pharmaceuticals, Inc. | Methods and compositions for inhibiting pmp22 expression |
| 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 |
| CA3092817A1 (en) * | 2018-03-02 | 2019-09-06 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Inhibition of polyomavirus replication |
| 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 |
| JP7561129B2 (ja) * | 2018-12-21 | 2024-10-03 | アイオーニス ファーマシューティカルズ， インコーポレーテッド | Ｐｍｐ２２の発現を低減するための化合物及び方法 |
| AU2021270720A1 (en) | 2020-05-11 | 2022-12-08 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
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| US4866042A (en) | 1987-11-18 | 1989-09-12 | Neuwelt Edward A | Method for the delivery of genetic material across the blood brain barrier |
| US6294520B1 (en) | 1989-03-27 | 2001-09-25 | Albert T. Naito | Material for passage through the blood-brain barrier |
| US5151510A (en) | 1990-04-20 | 1992-09-29 | Applied Biosystems, Inc. | Method of synethesizing sulfurized oligonucleotide analogs |
| FI951404A7 (fi) | 1992-09-25 | 1995-03-24 | Rhone Poulenc Rorer Sa | Adenovirusvektoreita vieraiden geenien siirtämiseksi keskushermostojärjestelmän , erityisesti aivojen, soluihin |
| US5656612A (en) | 1994-05-31 | 1997-08-12 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of raf gene expression |
| FR2727867B1 (fr) | 1994-12-13 | 1997-01-31 | Rhone Poulenc Rorer Sa | Transfert de genes dans les motoneurones medullaires au moyen de vecteurs adenoviraux |
| US6936589B2 (en) | 2001-09-28 | 2005-08-30 | Albert T. Naito | Parenteral delivery systems |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| EP3705125B1 (en) | 2007-12-04 | 2023-07-05 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| SI3449926T1 (sl) | 2009-06-17 | 2020-04-30 | Biogen Ma Inc. | Sestave in metode za modulacijo združevanja SMN2 pri subjektu |
| EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
| CN104004826B (zh) * | 2013-01-07 | 2016-03-02 | 赵晨 | 突变的基因prpf4在制备遗传性视网膜疾病诊断试剂中的应用 |
| CA2930859C (en) | 2013-09-04 | 2022-05-03 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
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| Human Molecular Genetics，2011年，Vol.20, No.11，p.2116-2130 |
| IOVS，Vol.56，2015年10月，p.6362-6375 |
| 実験医学，2008年，Vol.26, No.10，p.1590-1595 |
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| JP2019500347A (ja) | 2019-01-10 |
| WO2017106364A3 (en) | 2017-07-13 |
| EP3390642A2 (en) | 2018-10-24 |
| CA3005254A1 (en) | 2017-06-22 |
| JP2022088621A (ja) | 2022-06-14 |
| EP3390642B1 (en) | 2021-11-24 |
| ES2903394T3 (es) | 2022-04-01 |
| EP3390642A4 (en) | 2019-06-26 |
| WO2017106364A2 (en) | 2017-06-22 |
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