JP2018538288A - アラジール症候群の処置のためのアンチセンスオリゴマー - Google Patents
アラジール症候群の処置のためのアンチセンスオリゴマー Download PDFInfo
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- JP2018538288A JP2018538288A JP2018529221A JP2018529221A JP2018538288A JP 2018538288 A JP2018538288 A JP 2018538288A JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018529221 A JP2018529221 A JP 2018529221A JP 2018538288 A JP2018538288 A JP 2018538288A
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| PCT/US2016/066414 WO2017106210A1 (en) | 2015-12-14 | 2016-12-13 | Antisense oligomers for treatment of alagille syndrome |
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| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| AU2015327836B2 (en) | 2014-10-03 | 2021-07-01 | Cold Spring Harbor Laboratory | Targeted augmentation of nuclear gene output |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | 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 |
| JP7094963B2 (ja) | 2017-01-23 | 2022-07-04 | リジェネロン・ファーマシューティカルズ・インコーポレイテッド | Ｈｓｄ１７ｂ１３バリアントおよびその使用 |
| CN110536964A (zh) | 2017-03-10 | 2019-12-03 | 国立研究开发法人国立成育医疗研究中心 | 反义寡核苷酸和糖原贮积病Ia型预防或治疗用组合物 |
| CA3059348A1 (en) | 2017-04-11 | 2018-10-18 | Regeneron Pharmaceuticals, Inc. | Assays for screening activity of modulators of members of the hydroxysteroid (17-beta) dehydrogenase (hsd17b) family |
| EP3668984A4 (en) | 2017-08-18 | 2021-09-08 | Ionis Pharmaceuticals, Inc. | MODULATION OF THE NOTCH SIGNALING PATH FOR THE TREATMENT OF RESPIRATORY DISORDERS |
| CA3073515A1 (en) | 2017-08-25 | 2019-02-28 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
| 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 |
| WO2019172286A1 (ja) | 2018-03-09 | 2019-09-12 | 第一三共株式会社 | 糖原病Ｉａ型治療薬 |
| MX2020009812A (es) | 2018-03-21 | 2021-01-08 | Regeneron Pharma | COMPOSICIONES DE ARNI DE 17ß-HIDROXIESTEROIDE DESHIDROGENASA TIPO 13 (HSD17B13) Y MÉTODOS DE USO DE LAS MISMAS. |
| WO2019210279A1 (en) * | 2018-04-27 | 2019-10-31 | The Regents Of The University Of California | De novo formation of the biliary system by hepatocyte transdifferentiation |
| 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 |
| CN119193702A (zh) * | 2018-09-06 | 2024-12-27 | 加利福尼亚大学董事会 | 经由改造的adar募集的rna和dna碱基编辑 |
| TW202208627A (zh) | 2020-05-11 | 2022-03-01 | 美商斯托克治療公司 | 用於病症及疾病之治療的opa1反義寡聚物 |
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| US20040102401A1 (en) * | 2002-11-22 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of jagged 1 expression |
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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 |
| CA2145535C (en) | 1992-09-25 | 2007-07-17 | Axel Kahn | 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 |
| GB0326578D0 (en) * | 2003-11-14 | 2003-12-17 | Univ Belfast | Cancer diagnosis and therapy |
| WO2007047913A2 (en) | 2005-10-20 | 2007-04-26 | Isis Pharmaceuticals, Inc | Compositions and methods for modulation of lmna expression |
| US20080280848A1 (en) | 2005-10-28 | 2008-11-13 | Volker Patzel | Structures of Active Guide Rna Molecules and Method of Selection |
| WO2007048629A2 (en) | 2005-10-28 | 2007-05-03 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Modulation of rna silencing efficiency by argonaute proteins |
| WO2009082607A2 (en) | 2007-12-04 | 2009-07-02 | Alnylam Pharmaceuticals, Inc. | Targeting lipids |
| MX361732B (es) | 2009-06-17 | 2018-12-14 | Cold Spring Harbor Laboratory | Composiciones y metodos para la modulacion de division de smn2 en un sujeto. |
| 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 |
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| US20040102401A1 (en) * | 2002-11-22 | 2004-05-27 | Isis Pharmaceuticals Inc. | Modulation of jagged 1 expression |
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| CA3005245A1 (en) | 2017-06-22 |
| JP2022106803A (ja) | 2022-07-20 |
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| WO2017106210A1 (en) | 2017-06-22 |
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