DK3103872T3 - Oligonukleotider for at lave en ændring i sekvensen af et mål-rna-molekyle i en levende celle - Google Patents
Oligonukleotider for at lave en ændring i sekvensen af et mål-rna-molekyle i en levende celle Download PDFInfo
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- DK3103872T3 DK3103872T3 DK16166821.5T DK16166821T DK3103872T3 DK 3103872 T3 DK3103872 T3 DK 3103872T3 DK 16166821 T DK16166821 T DK 16166821T DK 3103872 T3 DK3103872 T3 DK 3103872T3
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- oligonucleotide
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Claims (10)
1. En enkelt strandet oligonukleotid for målretning af en mål-RNA-sekvens omfattende en mutation, til brug i behandlingen af en generisk forstyrrelse vedrørende mutationen i nævnte mål-RNA-sekvens, hvor oligonukleotiden omfatter en sekvens, som supplerer mål-RNA-sekvensen, og koder vildtypesekvensen, og hvor alle oligonukleocidens nukleosider er 2'-O-alkyl ribonukleosider.
2. En oligonukleotid til anvendelse ifølge krav 1, hvor alle oligonukleotidens nukleosider er 2'-O-metyl rigonukleosider.
3. En oligonukleotid til anvendelse ifølge krav 1 eller 2, hvor oligonukleotiden omfatter ændrede intemukleosidforbindelser valgt blandt gruppen bestående af fosforotioatforbindelser og metylfosfornatforbindelser.
4. En oligonukleotid til anvendelse ifølge ethvert af kravene 1 til 3, hvor nævnte mutation er en substitution, sletning eller indsætning.
5. En oligonukleotid til anvendelse ifølge ethvert af kravene 1 til 4, hvor oligonukleotiden har en længde på 15 til 100 nukleotider, hvoraf mindst 10 supplerer mål-RNA-sekvensen.
6. En oligonukleotid til anvendelse ifølge ethvert af kravene 1 til 5, hvor nævnte generiske forstyrrelse er valgt blandt gruppen bestående af cystisk fibrose, albinisme, alfa-1-antitrypsinmangel, Alzheimers sygdom, amyotrofisk lateral sklerose, astma, β- thalassæmi, Cadasil-syndrom, Charcot-Marie-tandsygdom, kronisk obstruktiv lungesygdom (COPD), distal spinal muskulatrofi (DSMA), Duchenne/Becker muskeldystrofi, Dystrofisk epidermolysis bullosa, epidormylosis bullosa, Fabrys sygdom, familiær adenomatøs, polyposis, galaktosæmi, Gauchers sygdom, glucose-6-fosfat dehydrogenase, hæmofili, arvelig hematochromatose, Hunters syndrom, Huntingtons sygdom, Hurler syndrom, inflammatoriske tarmsygdomme (IBD), nedarvet polyagglutination syndrom, Lesch-Nyhan syndrom, Lynch, Marfan syndrom, mucopolysaccaridosis, muskelsvind, myotonisk dystrofi type I og II, Niemann-Pick sygdom type A, B og C, NY-esol relateret cancer, Parkinsons sygdom, Peutz-Jeghers syndrom, fenylketonuri, Pompes sygdom, primær ciliær sygdom, pulmonal hypertension, retinitis pigmentosa, Sandhoff sygdom, svær kombineret immuninsufficiens syndrom (SCID), seglcelleanæmi, spinal muskelatrofi, Stargardts sygdom, Tay-Sachs sygdom og X-bunden immundefekt.
7. En oligonukleotid til anvendelse ifølge krav 6, hvor nævnte genetiske forstyrrelse er cystisk fibrose, og mål-RNA-sekvensen koder menneskelig CFTR omfattende en AF508 mutation.
8. En oligonukleotid til anvendelse ifølge krav 7, hvor oligonukleotiden supperer en sekvens med mindst 75 %, 80 %, 85 %, 90 %, 95 % eller 99 % sekvensidentitet til nukleotider 16-30 og 34-48 af SEQ ID NO: 5.
9. En oligonukleotid til anvendelse ifølge krav 7, hvor oligonukleotiden omfatter sekvensen af SEQ ID NO: 1.
10. En farmaceutisk sammensætning omfattende en oligonukleotid ifølge ethvert af kravene 1 til 9, og en farmaceutisk acceptabel bærer og/eller en hypertonisk fysiologisk og farmaceutisk acceptabel saltopløsning.
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| EP13760133.2A EP2852668B1 (en) | 2012-07-12 | 2013-07-12 | Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell |
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| DK3103872T3 true DK3103872T3 (da) | 2018-10-22 |
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| DK13760133.2T DK2852668T3 (da) | 2012-07-12 | 2013-07-12 | Oligonukleotider til at gennemføre en ændring i sekvensen af et rna-målmolekyle der er til stede i en levende celle |
| DK16166821.5T DK3103872T3 (da) | 2012-07-12 | 2013-07-12 | Oligonukleotider for at lave en ændring i sekvensen af et mål-rna-molekyle i en levende celle |
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| DK13760133.2T DK2852668T3 (da) | 2012-07-12 | 2013-07-12 | Oligonukleotider til at gennemføre en ændring i sekvensen af et rna-målmolekyle der er til stede i en levende celle |
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| HUE049976T2 (hu) | 2005-12-28 | 2020-11-30 | Vertex Pharma | N-[2,4-bisz(1,1-dimetil-etil)-5-hidroxi-fenil]-1,4-dihidro-4-oxo-kinolin-3-karboxamid amorf alakjának gyógyászati kompozíciói |
| 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 |
| MX2012012204A (es) | 2010-04-22 | 2012-12-05 | Vertex Pharma | Proceso para producir compuestos de cicloalquilcarboxamido-indol. |
| EP2852668B1 (en) | 2012-07-12 | 2016-04-27 | ProQR Therapeutics II B.V. | Oligonucleotides for making a change in the sequence of a target rna molecule present in a living cell |
| ES2957761T3 (es) | 2014-04-15 | 2024-01-25 | Vertex Pharma | Composiciones farmacéuticas para el tratamiento de enfermedades mediadas por el regulador de la conductancia transmembrana de fibrosis quística |
| SG10201911695SA (en) | 2014-05-14 | 2020-01-30 | Targimmune Therapeutics Ag | Improved polyethyleneimine polyethyleneglycol vectors |
| CN107250113B (zh) | 2014-10-07 | 2019-03-29 | 弗特克斯药品有限公司 | 囊性纤维化跨膜传导调节蛋白的调节剂的共晶 |
| GB201418892D0 (en) * | 2014-10-23 | 2014-12-10 | Proqr Therapeutics B V | DNA editing |
| US20210395729A1 (en) * | 2014-12-12 | 2021-12-23 | Tod M. Woolf | Compositions and methods for Editing Nucleic Acids in Cells Utilizing Oligonucleotides |
| CN107109413B (zh) * | 2014-12-17 | 2021-03-09 | ProQR治疗上市公司Ⅱ | 靶向的rna编辑 |
| GB201507926D0 (en) * | 2015-05-08 | 2015-06-24 | Proqr Therapeutics N V | Improved treatments using oligonucleotides |
| EP3359667A1 (en) | 2015-10-05 | 2018-08-15 | ProQR Therapeutics II B.V. | Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion |
| EP3364984B1 (en) | 2015-10-15 | 2021-11-17 | City of Hope | Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof |
| WO2017220751A1 (en) * | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| KR102501980B1 (ko) * | 2016-09-01 | 2023-02-20 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드 |
| US11274300B2 (en) | 2017-01-19 | 2022-03-15 | Proqr Therapeutics Ii B.V. | Oligonucleotide complexes for use in RNA editing |
| EP3589751A4 (en) | 2017-03-03 | 2021-11-17 | The Regents of The University of California | RNA TARGETING OF MUTATIONS VIA SUPPRESSOR RNA AND DEAMINASES |
| 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 |
| EP3697904A1 (en) | 2017-10-17 | 2020-08-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Combination treatment for cystic fibrosis |
| US11866702B2 (en) | 2018-03-27 | 2024-01-09 | University Of Rochester | Nucleic acid molecules for pseudouridylation |
| CA3127241A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| 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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| US8314226B2 (en) * | 2004-03-29 | 2012-11-20 | 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 |
| WO2011097641A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Methods and compositions useful in treatment of diseases or conditions related to repeat expansion |
| EA034363B1 (ru) * | 2010-04-23 | 2020-01-30 | Эрроухед Фармасьютикалс, Инк. | Фармацевтическая композиция для ингибирования экспрессии гена beta-enac и ее применение |
| TWI620756B (zh) | 2010-05-28 | 2018-04-11 | 薩羅塔治療公司 | 具有經修飾之單元間連結及／或末端基團之寡核苷酸類似物 |
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| US20150209448A1 (en) | 2012-07-12 | 2015-07-30 | Proqr Therapeutics N.V. | Exon replacement with stabilized artificial rnas |
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