CA3005131A1 - Antisense oligomers for treatment of tuberous sclerosis complex - Google Patents
Antisense oligomers for treatment of tuberous sclerosis complex Download PDFInfo
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- CA3005131A1 CA3005131A1 CA3005131A CA3005131A CA3005131A1 CA 3005131 A1 CA3005131 A1 CA 3005131A1 CA 3005131 A CA3005131 A CA 3005131A CA 3005131 A CA3005131 A CA 3005131A CA 3005131 A1 CA3005131 A1 CA 3005131A1
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- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
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US201562267212P | 2015-12-14 | 2015-12-14 | |
US62/267,212 | 2015-12-14 | ||
PCT/US2016/066705 WO2017106375A1 (en) | 2015-12-14 | 2016-12-14 | Antisense oligomers for treatment of tuberous sclerosis complex |
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CA3005131A1 true CA3005131A1 (en) | 2017-06-22 |
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CA3005131A Pending CA3005131A1 (en) | 2015-12-14 | 2016-12-14 | Antisense oligomers for treatment of tuberous sclerosis complex |
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EP (1) | EP3390635A4 (enrdf_load_stackoverflow) |
JP (2) | JP7051683B2 (enrdf_load_stackoverflow) |
CA (1) | CA3005131A1 (enrdf_load_stackoverflow) |
WO (1) | WO2017106375A1 (enrdf_load_stackoverflow) |
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GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
CA2963288A1 (en) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
WO2017060731A1 (en) | 2015-10-09 | 2017-04-13 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
CA3005256A1 (en) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
CN110446785B (zh) | 2017-01-23 | 2024-04-26 | 瑞泽恩制药公司 | Hsd17b13变体及其应用 |
MX2019012169A (es) | 2017-04-11 | 2019-12-11 | Regeneron Pharma | Ensayos para evaluar la actividad de moduladores de miembros de la familia de hidroxiesteroide (17-beta) deshidrogenasa (hsd17b). |
ES2965696T3 (es) | 2017-08-25 | 2024-04-16 | Stoke Therapeutics Inc | Oligómeros antisentido para el tratamiento de afecciones y enfermedades |
SG11202003254SA (en) | 2017-10-11 | 2020-05-28 | Regeneron Pharma | Inhibition of hsd17b13 in the treatment of liver disease in patients expressing the pnpla3 i148m variation |
CN112020556A (zh) | 2018-03-21 | 2020-12-01 | 瑞泽恩制药公司 | 第13型17β羟基类固醇脱氢酶(HSD17B13)iRNA组成物及其使用方法 |
JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス,インク. | コレステリルエステル蓄積症の処置のための方法及び組成物 |
WO2021034985A1 (en) * | 2019-08-19 | 2021-02-25 | Stoke Therapeutics, Inc. | Compositions and methods for modulating splicing and protein expression |
IL298063A (en) | 2020-05-11 | 2023-01-01 | Stoke Therapeutics Inc | Opa1 antisense oligomers for treatment of conditions and diseases |
EP4560019A1 (en) * | 2023-11-22 | 2025-05-28 | Sylentis S.A.U. | Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions |
EP4560020A1 (en) * | 2023-11-22 | 2025-05-28 | Sylentis S.A.U. | Sirna and compositions for prophylactic and therapeutic treatment of ocular retinal conditions |
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CA2666185A1 (en) * | 2006-10-11 | 2008-04-17 | Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. | Influenza targets |
US20120252877A1 (en) * | 2009-08-14 | 2012-10-04 | Theramind Research, Llc | Methods and compositions for treatment of tuberous sclerosis complex |
SG179038A1 (en) * | 2009-09-08 | 2012-04-27 | Lab Corp America Holdings | Compositions and methods for diagnosing autism spectrum disorders |
WO2015035091A1 (en) * | 2013-09-04 | 2015-03-12 | Cold Spring Harbor Laboratory | Reducing nonsense-mediated mrna decay |
GB201410693D0 (en) * | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
CA2963288A1 (en) * | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
WO2017106382A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of central nervous system diseases |
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JP2022088533A (ja) | 2022-06-14 |
JP2019500350A (ja) | 2019-01-10 |
WO2017106375A1 (en) | 2017-06-22 |
EP3390635A4 (en) | 2019-05-01 |
JP7051683B2 (ja) | 2022-04-11 |
EP3390635A1 (en) | 2018-10-24 |
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