ES2698522T3 - Oligonucleótidos para la realización de un cambio en la secuencia de una molécula de ARN diana presente en una célula viva - Google Patents
Oligonucleótidos para la realización de un cambio en la secuencia de una molécula de ARN diana presente en una célula viva Download PDFInfo
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- ES2698522T3 ES2698522T3 ES16166821T ES16166821T ES2698522T3 ES 2698522 T3 ES2698522 T3 ES 2698522T3 ES 16166821 T ES16166821 T ES 16166821T ES 16166821 T ES16166821 T ES 16166821T ES 2698522 T3 ES2698522 T3 ES 2698522T3
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| US8563573B2 (en) | 2007-11-02 | 2013-10-22 | Vertex Pharmaceuticals Incorporated | Azaindole derivatives as CFTR modulators |
| US8802868B2 (en) | 2010-03-25 | 2014-08-12 | Vertex Pharmaceuticals Incorporated | Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide |
| CA2797118C (en) | 2010-04-22 | 2021-03-30 | Vertex Pharmaceuticals Incorporated | Process of producing cycloalkylcarboxamido-indole compounds |
| SI2852668T1 (sl) | 2012-07-12 | 2016-09-30 | Proqr Therapeutics Ii B.V. | Oligonukleotidi za izvedbo spremembe v sekvenci tarčne molekule rna, prisotne v živeči celici |
| RS62140B1 (sr) | 2014-04-15 | 2021-08-31 | Vertex Pharma | Farmaceutske kompozicije za lečenje bolesti posredovanih transmembranskim regulatorom provodljivosti cistične fibroze |
| EP3142673A4 (en) | 2014-05-14 | 2018-01-03 | Targlmmune Therapeutics AG | Improved polyethyleneimine polyethyleneglycol vectors |
| KR20170063954A (ko) | 2014-10-07 | 2017-06-08 | 버텍스 파마슈티칼스 인코포레이티드 | 낭성 섬유증 막횡단 전도도 조절자의 조정제의 공-결정 |
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| JP6997623B2 (ja) * | 2014-12-12 | 2022-02-04 | エム． ウルフ、トッド | オリゴヌクレオチドを利用して細胞内の核酸を編集するための組成物および方法 |
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| US20210401869A1 (en) * | 2015-02-20 | 2021-12-30 | Rosalind Franklin University Of Medicine And Science | Antisense Compounds Targeting Genes Associated with Cystic Fibrosis |
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| CA3001723A1 (en) | 2015-10-15 | 2017-04-20 | City Of Hope | Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof |
| WO2017193053A1 (en) * | 2016-05-06 | 2017-11-09 | Woolf Tod M | Improved methods for genome editing with and without programmable nucleases |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| WO2018134301A1 (en) | 2017-01-19 | 2018-07-26 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in rna editing |
| CN118416088A (zh) | 2017-03-03 | 2024-08-02 | 加利福尼亚大学董事会 | 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向 |
| CN107267516B (zh) * | 2017-07-28 | 2020-09-29 | 佛山科学技术学院 | 双sgRNA介导的基因精确修饰方法及应用 |
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| EP3615085A4 (en) * | 2018-02-09 | 2021-01-13 | Ohio State Innovation Foundation | RNA NANOSTRUCTURES, THEIR MANUFACTURING PROCESSES AND THEIR USES |
| AU2019243946B2 (en) | 2018-03-27 | 2025-03-06 | Proqr Therapeutics Ii B.V. | Nucleic acid molecules for pseudouridylation |
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| TWI859184B (zh) * | 2019-01-22 | 2024-10-21 | 美商科羅生物公司 | Ｒｎａ編輯之寡核苷酸及其用途 |
| CA3159944A1 (en) | 2019-12-02 | 2021-06-10 | David HUSS | Therapeutic editing |
| CN113397996A (zh) * | 2021-07-23 | 2021-09-17 | 河南省人民医院 | 一种抗菌漱口水及其制备方法 |
| CN114917227B (zh) * | 2022-05-19 | 2023-10-31 | 中国人民解放军海军军医大学 | 依伐卡托在制备抗蜱传脑炎病毒、西尼罗病毒、黄热病毒和基孔肯雅热病毒感染药中的应用 |
| EP4432289A1 (en) | 2023-03-15 | 2024-09-18 | Shape Therapeutics Inc. | Generative sequence screening with conditional gans, diffusion models, and denoising diffusion conditional gans |
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| HRP930935A2 (en) * | 1992-06-11 | 1994-12-31 | Astra Ab | New dna sequences |
| CA2162361C (en) | 1993-05-11 | 2008-10-21 | Ryszard Kole | Antisense oligonucleotides which combat aberrant splicing and methods of using the same |
| US6207455B1 (en) | 1997-05-01 | 2001-03-27 | Lung-Ji Chang | Lentiviral vectors |
| JP4390860B2 (ja) | 1997-05-13 | 2009-12-24 | ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | レンチウイルスをベースにした遺伝子転移ベクター |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| US6218181B1 (en) | 1998-03-18 | 2001-04-17 | The Salk Institute For Biological Studies | Retroviral packaging cell line |
| US6683173B2 (en) | 1998-04-03 | 2004-01-27 | Epoch Biosciences, Inc. | Tm leveling methods |
| DE19909769A1 (de) | 1999-03-05 | 2000-09-07 | Bundesrepublik Deutschland Let | Von SIVagm abgeleitete lentivirale Vektoren, Verfahren zu ihrer Herstellung und ihre Verwendung zur Genübertragung in Säugerzellen |
| WO2005094370A2 (en) | 2004-03-29 | 2005-10-13 | The General Hospital Corporation | Oligonucleotide complex compositions and methods of use as gene alteration tools |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| JP2013518603A (ja) | 2010-02-08 | 2013-05-23 | 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 | 反復伸張に関連する疾患または病態の治療に有用な方法および組成物 |
| CA3098080A1 (en) * | 2010-04-23 | 2011-10-27 | Arrowhead Research Corporation | Organic compositions to treat beta-enac-related diseases |
| NZ603606A (en) | 2010-05-28 | 2015-06-26 | Sarepta Therapeutics Inc | Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups |
| WO2014011050A1 (en) | 2012-07-12 | 2014-01-16 | Proqr Therapeutics B.V. | Exon replacement with stabilized artificial rnas |
| SI2852668T1 (sl) * | 2012-07-12 | 2016-09-30 | Proqr Therapeutics Ii B.V. | Oligonukleotidi za izvedbo spremembe v sekvenci tarčne molekule rna, prisotne v živeči celici |
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