JP2019500345A5 - - Google Patents
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- JP2019500345A5 JP2019500345A5 JP2018529229A JP2018529229A JP2019500345A5 JP 2019500345 A5 JP2019500345 A5 JP 2019500345A5 JP 2018529229 A JP2018529229 A JP 2018529229A JP 2018529229 A JP2018529229 A JP 2018529229A JP 2019500345 A5 JP2019500345 A5 JP 2019500345A5
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267238P | 2015-12-14 | 2015-12-14 | |
| US62/267,238 | 2015-12-14 | ||
| US201662319015P | 2016-04-06 | 2016-04-06 | |
| US62/319,015 | 2016-04-06 | ||
| PCT/US2016/066564 WO2017106283A1 (en) | 2015-12-14 | 2016-12-14 | Compositions and methods for treatment of liver diseases |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019500345A JP2019500345A (ja) | 2019-01-10 |
| JP2019500345A5 true JP2019500345A5 (zh) | 2020-01-30 |
Family
ID=59057460
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018529229A Pending JP2019500345A (ja) | 2015-12-14 | 2016-12-14 | 肝臓病の処置のための組成物および方法 |
Country Status (4)
| Country | Link |
|---|---|
| EP (1) | EP3389672A4 (zh) |
| JP (1) | JP2019500345A (zh) |
| CA (1) | CA3005090A1 (zh) |
| WO (1) | WO2017106283A1 (zh) |
Families Citing this family (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| JP6867945B2 (ja) | 2014-10-03 | 2021-05-12 | コールド スプリング ハーバー ラボラトリー | 核内遺伝子出力の標的とされた増強 |
| WO2017060731A1 (en) | 2015-10-09 | 2017-04-13 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| EP3933041B1 (en) | 2015-12-14 | 2024-01-31 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant retardation |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| KR102619197B1 (ko) | 2017-01-23 | 2024-01-03 | 리제너론 파마슈티칼스 인코포레이티드 | Hsd17b13 변종 및 이것의 용도 |
| AU2018250727A1 (en) | 2017-04-11 | 2019-10-31 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (HSD17B) family |
| PL3673080T3 (pl) | 2017-08-25 | 2024-03-11 | Stoke Therapeutics, Inc. | Oligomery antysensowne do leczenia stanów i chorób |
| EP3461837A1 (en) * | 2017-09-28 | 2019-04-03 | Secarna Pharmaceuticals GmbH & Co. KG | Inhibitor inhibiting the expression of pprx1 |
| EP3695012B1 (en) | 2017-10-11 | 2023-03-08 | Regeneron Pharmaceuticals, Inc. | Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation |
| KR20200120675A (ko) * | 2018-02-14 | 2020-10-21 | 딥 지노믹스 인코포레이티드 | 윌슨병을 위한 올리고뉴클레오티드 요법 |
| SG11202007652UA (en) * | 2018-02-21 | 2020-09-29 | Bristol Myers Squibb Co | Camk2d antisense oligonucleotides and uses thereof |
| TW202023573A (zh) | 2018-09-19 | 2020-07-01 | 美商Ionis製藥公司 | Pnpla3表現之調節劑 |
| US10913951B2 (en) * | 2018-10-31 | 2021-02-09 | University of Pittsburgh—of the Commonwealth System of Higher Education | Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure |
| CN111849968A (zh) * | 2019-04-30 | 2020-10-30 | 中美瑞康核酸技术(南通)研究院有限公司 | 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用 |
| EP4017979A4 (en) * | 2019-08-19 | 2024-03-27 | Stoke Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING SPLICING AND PROTEIN EXPRESSION |
| AU2020349991A1 (en) * | 2019-09-20 | 2022-04-07 | Ractigen Therapeutics | Nucleic acid molecule for treating immune thrombocytopenia and application thereof |
| CN114728017A (zh) * | 2019-10-14 | 2022-07-08 | 阿斯利康(瑞典)有限公司 | Pnpla3表达的调节剂 |
| KR20230022409A (ko) | 2020-05-11 | 2023-02-15 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 opa1 안티센스 올리고머 |
| WO2021231698A1 (en) * | 2020-05-15 | 2021-11-18 | Korro Bio, Inc. | Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl) |
| CN116209761A (zh) * | 2020-07-23 | 2023-06-02 | 豪夫迈·罗氏有限公司 | 靶向rna结合蛋白位点的寡核苷酸 |
| WO2022109030A1 (en) * | 2020-11-17 | 2022-05-27 | Skyhawk Therapeutics, Inc. | Rna-targeting splicing modifiers for treatment of pnpla3-associated conditions and diseases |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO1994008026A1 (en) | 1992-09-25 | 1994-04-14 | Rhone-Poulenc Rorer S.A. | Adenovirus vectors for the transfer of foreign genes into cells of the central nervous system, particularly in brain |
| 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 |
| US7291461B2 (en) * | 2002-06-21 | 2007-11-06 | Ptc Therapeutics, Inc. | Methods for identifying small molecules that modulate premature translation termination and nonsense mRNA decay |
| JP5202296B2 (ja) * | 2005-04-01 | 2013-06-05 | ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド | 肝臓傷害のバイオマーカー |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| CA2910760C (en) | 2007-12-04 | 2019-07-09 | Muthiah Manoharan | Targeting lipids |
| CN106983768B (zh) | 2009-06-17 | 2023-04-07 | 冷泉港实验室 | 用于在对象中调节smn2剪接的组合物和方法 |
| WO2012075040A2 (en) * | 2010-11-30 | 2012-06-07 | Shire Human Genetic Therapies, Inc. | mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES |
| WO2012138487A2 (en) * | 2011-04-07 | 2012-10-11 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| EP3690048A1 (en) | 2013-09-04 | 2020-08-05 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
-
2016
- 2016-12-14 JP JP2018529229A patent/JP2019500345A/ja active Pending
- 2016-12-14 CA CA3005090A patent/CA3005090A1/en active Pending
- 2016-12-14 EP EP16876543.6A patent/EP3389672A4/en not_active Withdrawn
- 2016-12-14 WO PCT/US2016/066564 patent/WO2017106283A1/en active Application Filing
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