JP2018533362A - 薬物候補の有効性プロファイルを決定するための方法 - Google Patents
薬物候補の有効性プロファイルを決定するための方法 Download PDFInfo
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022517803A (ja) * | 2019-01-17 | 2022-03-10 | エフ.ホフマン-ラ ロシュ アーゲー | E3ユビキチンリガーゼ(ube3a)のタンパク質標的 |
Families Citing this family (30)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| NZ714887A (en) | 2009-09-11 | 2019-07-26 | Ionis Pharmaceuticals Inc | Modulation of huntingtin expression |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| WO2014004572A2 (en) | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| US10400243B2 (en) | 2014-11-25 | 2019-09-03 | Ionis Pharmaceuticals, Inc. | Modulation of UBE3A-ATS expression |
| LT3374509T (lt) | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| KR102723989B1 (ko) | 2017-12-01 | 2024-11-01 | 더 텍사스 에이 & 엠 유니버시티 시스템 | 엔젤만 증후군 안티센스 치료 |
| WO2019157531A1 (en) | 2018-02-12 | 2019-08-15 | Ionis Pharmaceuticals, Inc. | Modified compounds and uses thereof |
| CN108795935B (zh) * | 2018-05-23 | 2022-06-21 | 中国农业科学院上海兽医研究所(中国动物卫生与流行病学中心上海分中心) | 日本血吸虫SjELAV-like 1基因的siRNA及其应用 |
| WO2020061528A1 (en) * | 2018-09-21 | 2020-03-26 | University Of Connecticut | Compositions and methods to restore paternal ube3a gene expression in human angelman syndrome |
| TW202102516A (zh) * | 2019-02-20 | 2021-01-16 | 丹麥商羅氏創新中心哥本哈根有限公司 | 膦醯基乙酸酯間隙子寡核苷酸 |
| PT3947684T (pt) * | 2019-03-29 | 2025-05-19 | Ionis Pharmaceuticals Inc | Compostos e métodos para modular ube3a‑ats |
| US12465655B2 (en) * | 2019-05-22 | 2025-11-11 | The University Of North Carolina At Chapel Hill | UBE3A genes and expression cassettes and their use |
| EP3976765A1 (en) | 2019-05-28 | 2022-04-06 | F. Hoffmann-La Roche AG | Method to generate monocytic progenitor cells |
| TW202204607A (zh) * | 2020-04-16 | 2022-02-01 | 英商羅斯林科技有限公司 | 誘導性多功能幹細胞(iPSC)之誘導 |
| US20230212572A1 (en) * | 2020-06-09 | 2023-07-06 | Roche Innovation Center Copenhagen A/S | Guanosine Analogues for Use in Therapeutics Polynucleotides |
| EP4282651A4 (en) | 2021-01-21 | 2024-12-18 | Fujimori Kogyo Co., Ltd. | MULTILAYER FILM AND PACKAGING BAGS |
| CN119173632A (zh) | 2022-05-10 | 2024-12-20 | 豪夫迈·罗氏有限公司 | 靶向cfp-elk1基因间区域的反义寡核苷酸 |
| EP4705457A2 (en) | 2023-05-04 | 2026-03-11 | F. Hoffmann-La Roche AG | Oligonucleotides capable of upregulating glucocerebrosidase expression |
| WO2025100980A1 (ko) * | 2023-11-08 | 2025-05-15 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| WO2025125542A1 (en) | 2023-12-15 | 2025-06-19 | F. Hoffmann-La Roche Ag | Intrathecal dosage method |
| WO2025213123A1 (en) * | 2024-04-05 | 2025-10-09 | Encoded Therapeutics, Inc. | Methods and compositions for increasing expression of ube3a |
| WO2025217466A1 (en) * | 2024-04-11 | 2025-10-16 | Ultragenyx Pharmaceutical Inc. | Dosing regimens for antisense treatment of angelman syndrome |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015143342A1 (en) * | 2014-03-21 | 2015-09-24 | Cellular Dynamics International, Inc. | Production of midbrain dopaminergic neurons and methods for the use thereof |
Family Cites Families (58)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4373084A (en) | 1981-07-07 | 1983-02-08 | E. I. Du Pont De Nemours And Company | Sulfur-modified polypropylene ether glycols, a method for preparing them, and polyurethanes prepared therefrom |
| DE69233046T2 (de) | 1991-10-24 | 2004-03-04 | Isis Pharmaceuticals, Inc., Carlsfeld | Derivatisierte oligonukleotide mit verbessertem aufnahmevermögen |
| JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
| US6617162B2 (en) | 2001-12-18 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Antisense modulation of estrogen receptor alpha expression |
| JP4236812B2 (ja) | 1997-09-12 | 2009-03-11 | エクシコン エ/エス | オリゴヌクレオチド類似体 |
| US6238921B1 (en) | 1998-03-26 | 2001-05-29 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of human mdm2 expression |
| CA2702148C (en) * | 1999-01-06 | 2014-03-04 | Genenews Inc. | Method of profiling gene expression in a human subject having an infectious disease |
| ES2234563T5 (es) | 1999-02-12 | 2018-01-17 | Daiichi Sankyo Company, Limited | Nuevos análogos de nucleósidos y oligonucleótidos |
| US7053207B2 (en) | 1999-05-04 | 2006-05-30 | Exiqon A/S | L-ribo-LNA analogues |
| US6617442B1 (en) | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| EP1234174A1 (en) | 1999-12-02 | 2002-08-28 | Myriad Genetics, Inc. | Protein-protein interactions |
| US6300132B1 (en) | 1999-12-17 | 2001-10-09 | Isis Pharmaceuticals, Inc. | Antisense inhibition of telomeric repeat binding factor 2 expression |
| WO2001092582A1 (en) * | 2000-06-01 | 2001-12-06 | Genaissance Pharmaceuticals, Inc. | Haplotypes of the ube3a gene |
| US20030087855A1 (en) | 2001-09-13 | 2003-05-08 | Isis Pharmaceuticals Inc. | Antisense modulation of protein kinase R expression |
| EP2221376B1 (en) | 2001-06-21 | 2012-11-21 | Isis Pharmaceuticals, Inc. | Antisense modulation of superoxide dismutase 1, soluble expression |
| US20050124572A1 (en) * | 2002-06-17 | 2005-06-09 | Freier Susan M. | Compositions and their uses directed to signal tranducers |
| WO2004016754A2 (en) | 2002-08-14 | 2004-02-26 | Pharmacia Corporation | ANTISENSE MODULATION OF Nav1.3 EXPRESSION |
| JP2006500066A (ja) * | 2002-09-25 | 2006-01-05 | ファルマシア・コーポレーション | ミクロソームのプロスタグランジンe2シンターゼ発現のアンチセンス調節 |
| WO2004046160A2 (en) | 2002-11-18 | 2004-06-03 | Santaris Pharma A/S | Amino-lna, thio-lna and alpha-l-oxy-ln |
| KR20060115351A (ko) * | 2003-08-29 | 2006-11-08 | 위스콘신 얼럼나이 리서어치 화운데이션 | 영장류 배아 줄기 세포 기원의 신경 줄기 세포, 운동 뉴런및 도파민 뉴런의 시험관내 분화 방법 |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| JP5713377B2 (ja) | 2005-12-28 | 2015-05-07 | ザ スクリプス リサーチ インスティテュート | 薬物標的としての天然アンチセンスおよび非コードrna転写物 |
| WO2007090071A2 (en) | 2006-01-27 | 2007-08-09 | Isis Pharmaceuticals, Inc. | 6-modified bicyclic nucleic acid analogs |
| JP2009536222A (ja) | 2006-05-05 | 2009-10-08 | アイシス ファーマシューティカルズ, インコーポレーテッド | Pcsk9の発現を調節するための化合物および方法 |
| AU2007249349B2 (en) | 2006-05-11 | 2012-03-08 | Isis Pharmaceuticals, Inc. | 5'-Modified bicyclic nucleic acid analogs |
| US7666854B2 (en) | 2006-05-11 | 2010-02-23 | Isis Pharmaceuticals, Inc. | Bis-modified bicyclic nucleic acid analogs |
| DK2149605T3 (da) | 2007-03-22 | 2013-09-30 | Santaris Pharma As | Korte RNA antagonist forbindelser til modulering af det ønskede mRNA |
| PE20090064A1 (es) | 2007-03-26 | 2009-03-02 | Novartis Ag | Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende |
| CA2688321A1 (en) | 2007-05-30 | 2008-12-11 | Isis Pharmaceuticals, Inc. | N-substituted-aminomethylene bridged bicyclic nucleic acid analogs |
| DK2173760T4 (en) | 2007-06-08 | 2016-02-08 | Isis Pharmaceuticals Inc | Carbocyclic bicyclic nukleinsyreanaloge |
| AU2008272918B2 (en) | 2007-07-05 | 2012-09-13 | Isis Pharmaceuticals, Inc. | 6-disubstituted bicyclic nucleic acid analogs |
| WO2009067647A1 (en) | 2007-11-21 | 2009-05-28 | Isis Pharmaceuticals, Inc. | Carbocyclic alpha-l-bicyclic nucleic acid analogs |
| WO2009090182A1 (en) | 2008-01-14 | 2009-07-23 | Santaris Pharma A/S | C4'-substituted - dna nucleotide gapmer oligonucleotides |
| US9290534B2 (en) | 2008-04-04 | 2016-03-22 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside |
| DK2356129T3 (da) | 2008-09-24 | 2013-05-13 | Isis Pharmaceuticals Inc | Substituerede alpha-L-bicykliske nukleosider |
| WO2010096496A2 (en) * | 2009-02-17 | 2010-08-26 | Memorial Sloan-Kettering Cancer Center | Methods of neural conversion of human embryonic stem cells |
| US9012421B2 (en) | 2009-08-06 | 2015-04-21 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| JP5843775B2 (ja) * | 2009-10-13 | 2016-01-13 | ステムセル テクノロジーズ インコーポレーティッド | 幹細胞を分化させるための重量オスモル濃度の操作法 |
| CN102869776B (zh) | 2009-12-23 | 2017-06-23 | 库尔纳公司 | 通过抑制肝细胞生长因子(hgf)的天然反义转录物而治疗hgf相关疾病 |
| EP2580228B1 (en) | 2010-06-08 | 2016-03-23 | Ionis Pharmaceuticals, Inc. | Substituted 2'-amino and 2'-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom |
| JP5998131B2 (ja) | 2010-07-14 | 2016-09-28 | カッパーアールエヌエー,インコーポレイテッド | Discslargehomolog(dlg)dlg1への天然アンチセンス転写物の阻害によるdlg関連疾患の治療 |
| EP2595663A4 (en) | 2010-07-19 | 2014-03-05 | Isis Pharmaceuticals Inc | MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION |
| CA2807226A1 (en) * | 2010-08-19 | 2012-02-23 | F.Hoffmann-La Roche Ag | Conversion of somatic cells to induced reprogrammed neural stem cells (irnscs) |
| US9714427B2 (en) | 2010-11-11 | 2017-07-25 | The University Of North Carolina At Chapel Hill | Methods and compositions for unsilencing imprinted genes |
| CA2817960C (en) | 2010-11-17 | 2020-06-09 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| DK2694644T3 (en) * | 2011-03-30 | 2018-04-16 | Cellular Dynamics Int Inc | Priming of pluripotent stem cells for neural differentiation |
| RU2013150331A (ru) | 2011-04-20 | 2015-05-27 | Рош Гликарт Аг | СПОСОБ И УСТРОЙСТВА ДЛЯ рН-ЗАВИСИМОГО ПРОХОЖДЕНИЯ ГЕМАТОЭНЦЕФАЛИЧЕСКОГО БАРЬЕРА |
| WO2013033230A1 (en) | 2011-08-29 | 2013-03-07 | Isis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| EP2753631A1 (en) | 2011-09-07 | 2014-07-16 | Marina Biotech, Inc. | Synthesis and uses of nucleic acid compounds with conformationally restricted monomers |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| WO2014004572A2 (en) * | 2012-06-25 | 2014-01-03 | Isis Pharmaceuticals, Inc. | Modulation of ube3a-ats expression |
| CN104837996A (zh) | 2012-11-15 | 2015-08-12 | 罗氏创新中心哥本哈根有限公司 | 抗apob反义缀合物化合物 |
| WO2014077693A1 (en) | 2012-11-16 | 2014-05-22 | Academisch Ziekenhuis Leiden H.O.D.N. Lumc | Means and methods for reducing an effect of aging in a mammalian cell |
| LT3374509T (lt) * | 2015-11-12 | 2021-03-10 | F. Hoffmann-La Roche Ag | Oligonukleotidai, skirti sukelti iš tėvo paveldėto ube3a raišką |
| BR112019027692A2 (pt) | 2017-06-28 | 2020-11-24 | University Of South Florida | gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman |
| KR102723989B1 (ko) | 2017-12-01 | 2024-11-01 | 더 텍사스 에이 & 엠 유니버시티 시스템 | 엔젤만 증후군 안티센스 치료 |
| AU2019212849B2 (en) | 2018-01-26 | 2024-02-29 | F. Hoffmann-La Roche Ag | Radiolabelled oligonucleotides and process for their preparation |
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Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015143342A1 (en) * | 2014-03-21 | 2015-09-24 | Cellular Dynamics International, Inc. | Production of midbrain dopaminergic neurons and methods for the use thereof |
Non-Patent Citations (3)
| Title |
|---|
| CARTER R. L. ET AL.: "Reversal of cellular phenotypes in neural cells derived from Huntington's disease monkey-induced plu", STEM CELL REPORTS, vol. 3, no. 4, JPN6020025859, 2014, pages 585 - 593, XP055345080, ISSN: 0004462212, DOI: 10.1016/j.stemcr.2014.07.011 * |
| SIRENKO O. ET AL.: "High-content high-throughput assays for characterizing the viability and morphology of human iPSC-de", ASSAY AND DRUG DEVELOPMENT TECHNOLOGIES, vol. vol. 12, no9/10, JPN6020025861, 2014, pages 536 - 547, ISSN: 0004307410 * |
| SUNDBERG M. ET AL.: "Improved cell therapy protocols for Parkinson's disease based on differentiation efficiency and safe", STEM CELLS, vol. 31, no. 8, JPN6020025860, 2013, pages 1548 - 1562, XP055189032, ISSN: 0004462213, DOI: 10.1002/stem.1415 * |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022517803A (ja) * | 2019-01-17 | 2022-03-10 | エフ.ホフマン-ラ ロシュ アーゲー | E3ユビキチンリガーゼ(ube3a)のタンパク質標的 |
| JP7241185B2 (ja) | 2019-01-17 | 2023-03-16 | エフ. ホフマン-ラ ロシュ アーゲー | E3ユビキチンリガーゼ(ube3a)のタンパク質標的 |
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