DK3353299T3 - Fremgangsmåder og stoffer til målrettet rna-editering - Google Patents
Fremgangsmåder og stoffer til målrettet rna-editering Download PDFInfo
- Publication number
- DK3353299T3 DK3353299T3 DK16766834.2T DK16766834T DK3353299T3 DK 3353299 T3 DK3353299 T3 DK 3353299T3 DK 16766834 T DK16766834 T DK 16766834T DK 3353299 T3 DK3353299 T3 DK 3353299T3
- Authority
- DK
- Denmark
- Prior art keywords
- materials
- methods
- rna editing
- targeted rna
- targeted
- Prior art date
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04004—Adenosine deaminase (3.5.4.4)
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Crystallography & Structural Chemistry (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Enzymes And Modification Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DE102015012522.2A DE102015012522B3 (de) | 2015-09-26 | 2015-09-26 | Verfahren und Substanzen zur gerichteten RNA-Editierung |
| PCT/DE2016/000309 WO2017050306A1 (de) | 2015-09-26 | 2016-08-09 | Verfahren und substanzen zur gerichteten rna-editierung |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| DK3353299T3 true DK3353299T3 (da) | 2020-06-08 |
Family
ID=55968076
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DK16766834.2T DK3353299T3 (da) | 2015-09-26 | 2016-08-09 | Fremgangsmåder og stoffer til målrettet rna-editering |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20190093098A1 (da) |
| EP (1) | EP3353299B1 (da) |
| DE (1) | DE102015012522B3 (da) |
| DK (1) | DK3353299T3 (da) |
| WO (1) | WO2017050306A1 (da) |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3712269A1 (en) | 2014-12-17 | 2020-09-23 | ProQR Therapeutics II B.V. | Targeted rna editing |
| US11390865B2 (en) | 2015-07-14 | 2022-07-19 | Fukuoka University | Method for introducing site-directed RNA mutation, target editing guide RNA used in the method and target RNA-target editing guide RNA complex |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| US10941402B2 (en) | 2016-09-01 | 2021-03-09 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded RNA-editing oligonucleotides |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| GB2574769A (en) | 2017-03-03 | 2019-12-18 | Univ California | RNA Targeting of mutations via suppressor tRNAs and deaminases |
| IL316453A (en) * | 2017-10-06 | 2024-12-01 | Univ Oregon Health & Science | RNA editing agents and methods |
| WO2019111957A1 (ja) * | 2017-12-06 | 2019-06-13 | 学校法人福岡大学 | オリゴヌクレオチド、その製造方法及び標的ｒｎａの部位特異的編集方法 |
| CN112020557B (zh) | 2018-03-27 | 2025-02-11 | 罗切斯特大学 | 用于假尿苷化的核酸分子 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| US20220073915A1 (en) | 2018-06-29 | 2022-03-10 | Eberhard-Karls-Universität-Tübingen | Artificial nucleic acids for rna editing |
| CA3115864A1 (en) * | 2018-10-12 | 2020-04-16 | Peking University | Methods and compositions for editing rnas |
| CN113573717A (zh) | 2019-01-22 | 2021-10-29 | 科罗生物公司 | Rna编辑的寡核苷酸及其用途 |
| JP2022519184A (ja) | 2019-01-22 | 2022-03-22 | コロ バイオ， インコーポレイテッド | Ｒｎａ編集オリゴヌクレオチド及びその使用 |
| CA3126947A1 (en) | 2019-01-22 | 2020-07-30 | Korro Bio, Inc. | Rna-editing oligonucleotides and uses thereof |
| WO2020211780A1 (en) * | 2019-04-15 | 2020-10-22 | Edigene Inc. | Methods and compositions for editing rnas |
| WO2021008447A1 (en) | 2019-07-12 | 2021-01-21 | Peking University | Targeted rna editing by leveraging endogenous adar using engineered rnas |
| JPWO2021020550A1 (da) * | 2019-08-01 | 2021-02-04 | ||
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| EP4074825A4 (en) | 2019-12-09 | 2025-01-22 | Astellas Pharma Inc. | ANTISENSE GUIDE RNA WITH ADDED FUNCTIONAL REGION FOR TARGET RNA EDITING |
| JP7755837B2 (ja) * | 2020-03-12 | 2025-10-17 | 学校法人福岡大学 | オリゴヌクレオチド及び標的ｒｎａの部位特異的編集方法 |
| US20240011028A9 (en) * | 2020-04-22 | 2024-01-11 | Shape Therapeutics Inc. | Compositions and Methods Using SNRNA Components |
| WO2022078569A1 (en) | 2020-10-12 | 2022-04-21 | Eberhard Karls Universität Tübingen | Artificial nucleic acids for rna editing |
| IL278401A (en) | 2020-10-29 | 2022-05-01 | Yeda Res & Dev | Polynucleotides for editing RNA and a method for using them |
| US20240150754A1 (en) | 2020-12-25 | 2024-05-09 | Astellas Pharma Inc. | Guide rna for editing polyadenylation signal sequence of target rna |
| GB202107553D0 (en) * | 2021-05-27 | 2021-07-14 | Univ Oxford Innovation Ltd | Method |
| WO2023152371A1 (en) | 2022-02-14 | 2023-08-17 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| WO2024013360A1 (en) | 2022-07-15 | 2024-01-18 | Proqr Therapeutics Ii B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| AU2023306595A1 (en) | 2022-07-15 | 2025-02-27 | Proqr Therapeutics Ii B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| CN120476207A (zh) | 2022-11-24 | 2025-08-12 | ProQR治疗上市公司Ⅱ | 反义寡核苷酸治疗遗传性hfe血色素沉着症 |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| EP4630553A1 (en) | 2022-12-09 | 2025-10-15 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of cardiovascular disease |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024175550A1 (en) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| CN121039282A (zh) | 2023-03-24 | 2025-11-28 | ProQR治疗上市公司Ⅱ | 用于治疗神经系统病症的反义寡核苷酸 |
| AU2024246572A1 (en) | 2023-03-27 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| TW202516003A (zh) | 2023-06-16 | 2025-04-16 | 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ | 用於治療神經退化性疾病之反義寡核苷酸 |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2025104239A1 (en) | 2023-11-16 | 2025-05-22 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of classic galactosemia |
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| GB202404661D0 (en) | 2024-04-02 | 2024-05-15 | Proqr Therapeutics Ii Bv | Antisense oligoncleotides for the treatment of liver disease |
| WO2025224230A1 (en) | 2024-04-25 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of fatty liver disease |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9650627B1 (en) | 2012-07-19 | 2017-05-16 | University Of Puerto Rico | Site-directed RNA editing |
| EP3712269A1 (en) | 2014-12-17 | 2020-09-23 | ProQR Therapeutics II B.V. | Targeted rna editing |
| US11390865B2 (en) * | 2015-07-14 | 2022-07-19 | Fukuoka University | Method for introducing site-directed RNA mutation, target editing guide RNA used in the method and target RNA-target editing guide RNA complex |
-
2015
- 2015-09-26 DE DE102015012522.2A patent/DE102015012522B3/de active Active
-
2016
- 2016-08-09 US US15/763,351 patent/US20190093098A1/en active Pending
- 2016-08-09 EP EP16766834.2A patent/EP3353299B1/de active Active
- 2016-08-09 DK DK16766834.2T patent/DK3353299T3/da active
- 2016-08-09 WO PCT/DE2016/000309 patent/WO2017050306A1/de not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| WO2017050306A1 (de) | 2017-03-30 |
| DE102015012522B3 (de) | 2016-06-02 |
| US20190093098A1 (en) | 2019-03-28 |
| EP3353299B1 (de) | 2020-03-04 |
| EP3353299A1 (de) | 2018-08-01 |
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