CA3005249A1 - Compositions et methodes de traitement de maladies renales - Google Patents
Compositions et methodes de traitement de maladies renales Download PDFInfo
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- CA3005249A1 CA3005249A1 CA3005249A CA3005249A CA3005249A1 CA 3005249 A1 CA3005249 A1 CA 3005249A1 CA 3005249 A CA3005249 A CA 3005249A CA 3005249 A CA3005249 A CA 3005249A CA 3005249 A1 CA3005249 A1 CA 3005249A1
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- C—CHEMISTRY; METALLURGY
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
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- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Biomedical Technology (AREA)
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- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Urology & Nephrology (AREA)
- Public Health (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
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Abstract
L'invention concerne des procédés et des compositions pour augmenter l'expression d'une protéine, et pour traiter un sujet en ayant besoin, par exemple un sujet souffrant d'une déficience d'expression de protéine ou un sujet atteint d'une maladie rénale.
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562267242P | 2015-12-14 | 2015-12-14 | |
| US62/267,242 | 2015-12-14 | ||
| PCT/US2016/066576 WO2017106292A1 (fr) | 2015-12-14 | 2016-12-14 | Compositions et méthodes de traitement de maladies rénales |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CA3005249A1 true CA3005249A1 (fr) | 2017-06-22 |
Family
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Family Applications (1)
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| CA3005249A Pending CA3005249A1 (fr) | 2015-12-14 | 2016-12-14 | Compositions et methodes de traitement de maladies renales |
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| JP (2) | JP7049247B2 (fr) |
| CA (1) | CA3005249A1 (fr) |
| WO (1) | WO2017106292A1 (fr) |
Families Citing this family (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CA2963288A1 (fr) | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Augmentation ciblee de la production de genes nucleaires |
| AU2016334804B2 (en) | 2015-10-09 | 2022-03-31 | University Of Southampton | Modulation of gene expression and screening for deregulated protein expression |
| WO2017106377A1 (fr) | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Oligomères antisens destinés au traitement du retard mental dominant autosomique 5 et du syndrome de dravet |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| WO2018136702A1 (fr) | 2017-01-23 | 2018-07-26 | Regeneron Pharmaceuticals, Inc. | Variants de la 17-bêta-hydroxystéroïde déshydrogénase 13 (hsd17b13) et leurs utilisations |
| EP3610029A1 (fr) | 2017-04-11 | 2020-02-19 | Regeneron Pharmaceuticals, Inc. | Dosage de criblage de l'activité des modulateurs de membres de la famille des hydroxystéroïde (17-bêta) déshydrogénase (hsd17b) |
| LT3673080T (lt) | 2017-08-25 | 2023-12-27 | Stoke Therapeutics, Inc. | Priešprasminiai oligomerai, skirti būklėms ir ligoms gydyti |
| CN111183234A (zh) | 2017-10-11 | 2020-05-19 | 雷杰纳荣制药公司 | 在表达pnpla3 i148m变异的患者的肝病治疗中对hsd17b13的抑制 |
| BR112020018758A2 (pt) | 2018-03-21 | 2021-01-26 | Regeneron Pharmaceuticals, Inc. | agente de ácido ribonucleico de fita dupla, célula, vetor, composição farmacêutica, e, métodos para inibição da expressão de 17¿-hidroxiesteroide desidrogenases tipo 13, para tratamento de um indivíduo, para prevenção de um sintoma em um indivíduo, para redução do risco de desenvolver doença hepática crônica, para inibição da progressão de esteatose, para inibição do acúmulo de gotículas de lipídios |
| BR112020022512A2 (pt) | 2018-05-04 | 2021-05-04 | Stoke Therapeutics, Inc. | métodos e composições para tratamento de doença de armazenamento de éster de colesteril |
| US12163129B2 (en) | 2018-06-08 | 2024-12-10 | University Of Massachusetts | Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells |
| EP3861018A4 (fr) | 2018-10-05 | 2022-10-26 | Seattle Children's Hospital d/b/a Seattle Children's Research Institute | Dépistage de nouveau-né au sujet d'immunodéficiences primaires, de la cystinose et de la maladie de wilson |
| AU2020210924A1 (en) * | 2019-01-23 | 2021-09-16 | The Florey Institute Of Neuroscience And Mental Health | Antisense oligonucleotides targeting SCN2A retained introns |
| JP2022544702A (ja) * | 2019-08-19 | 2022-10-20 | ストーク セラピューティクス，インク． | スプライシングおよびタンパク質発現を調節するための組成物および方法 |
| EP4574971A2 (fr) | 2020-03-31 | 2025-06-25 | Seattle Children's Hospital d/b/a Seattle Children's Research Institute | Criblage protéomique de maladies lysosomales |
| EP4150092A4 (fr) | 2020-05-11 | 2024-11-06 | Stoke Therapeutics, Inc. | Oligomères antisens opa1 pour le traitement de pathologies et de maladies |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030224514A1 (en) * | 2002-05-31 | 2003-12-04 | Isis Pharmaceuticals Inc. | Antisense modulation of PPAR-delta expression |
| CA2588646A1 (fr) * | 2004-11-19 | 2006-05-26 | Acadia Pharmaceuticals Inc. | Outils permettant l'identification de ligands pour recepteurs nucleaires hormonaux |
| US20110269735A1 (en) * | 2010-04-19 | 2011-11-03 | Celera Corporation | Genetic polymorphisms associated with statin response and cardiovascular diseases, methods of detection and uses thereof |
| US20140128449A1 (en) * | 2011-04-07 | 2014-05-08 | The Board Of Regents Of The University Of Texas System | Oligonucleotide modulation of splicing |
| US9909128B2 (en) * | 2012-11-15 | 2018-03-06 | The Regents Of The University Of California | Splice modulating oligonucleotides that inhibit cancer |
| PL3041958T3 (pl) * | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
| CA2963288A1 (fr) * | 2014-10-03 | 2016-04-07 | Cold Spring Harbor Laboratory | Augmentation ciblee de la production de genes nucleaires |
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2016
- 2016-12-14 CA CA3005249A patent/CA3005249A1/fr active Pending
- 2016-12-14 JP JP2018529237A patent/JP7049247B2/ja not_active Expired - Fee Related
- 2016-12-14 EP EP16876548.5A patent/EP3390666A4/fr not_active Withdrawn
- 2016-12-14 WO PCT/US2016/066576 patent/WO2017106292A1/fr not_active Ceased
-
2022
- 2022-02-01 JP JP2022013876A patent/JP2022062143A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| JP7049247B2 (ja) | 2022-04-06 |
| EP3390666A4 (fr) | 2019-08-07 |
| EP3390666A1 (fr) | 2018-10-24 |
| WO2017106292A1 (fr) | 2017-06-22 |
| JP2019500346A (ja) | 2019-01-10 |
| JP2022062143A (ja) | 2022-04-19 |
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