MX2023005541A - Nuevo transcripto de arn. - Google Patents
Nuevo transcripto de arn.Info
- Publication number
- MX2023005541A MX2023005541A MX2023005541A MX2023005541A MX2023005541A MX 2023005541 A MX2023005541 A MX 2023005541A MX 2023005541 A MX2023005541 A MX 2023005541A MX 2023005541 A MX2023005541 A MX 2023005541A MX 2023005541 A MX2023005541 A MX 2023005541A
- Authority
- MX
- Mexico
- Prior art keywords
- mrna
- compound
- spliced
- intronic sequence
- induced
- Prior art date
Links
- 150000001875 compounds Chemical class 0.000 abstract 5
- 108020004999 messenger RNA Proteins 0.000 abstract 5
- 150000003384 small molecules Chemical class 0.000 abstract 2
- ICZNVPJUHBURBG-UHFFFAOYSA-N 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)triazolo[4,5-c]pyridazin-6-yl]-5-(triazol-2-yl)phenol Chemical compound N1C(CC(CC1(C)C)N1C2=C(N=N1)C=C(C1=CC=C(N3N=CC=N3)C=C1O)N=N2)(C)C ICZNVPJUHBURBG-UHFFFAOYSA-N 0.000 abstract 1
- 208000023105 Huntington disease Diseases 0.000 abstract 1
- 108091027974 Mature messenger RNA Proteins 0.000 abstract 1
- 108020004485 Nonsense Codon Proteins 0.000 abstract 1
- 108700026244 Open Reading Frames Proteins 0.000 abstract 1
- -1 e.g. Chemical class 0.000 abstract 1
- 230000037433 frameshift Effects 0.000 abstract 1
- 230000001404 mediated effect Effects 0.000 abstract 1
- 230000002028 premature Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
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
- C12N15/11—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
- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
Landscapes
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Saccharide Compounds (AREA)
- Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Abstract
Se describe una secuencia intrónica empalmada alternativamente, cuyo empalme puede inducirse en presencia de una molécula pequeña, como se describe en el presente documento.
Applications Claiming Priority (8)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063113182P | 2020-11-12 | 2020-11-12 | |
| US202063113826P | 2020-11-13 | 2020-11-13 | |
| US202163192203P | 2021-05-24 | 2021-05-24 | |
| US202163245927P | 2021-09-19 | 2021-09-19 | |
| US202163261467P | 2021-09-21 | 2021-09-21 | |
| US202163261495P | 2021-09-22 | 2021-09-22 | |
| US202163255745P | 2021-10-14 | 2021-10-14 | |
| PCT/US2021/059010 WO2022103980A1 (en) | 2020-11-12 | 2021-11-11 | Novel rna transcript |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023005541A true MX2023005541A (es) | 2023-06-15 |
Family
ID=78845036
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023005541A MX2023005541A (es) | 2020-11-12 | 2021-11-11 | Nuevo transcripto de arn. |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20220162610A1 (es) |
| EP (1) | EP4244362A1 (es) |
| AU (1) | AU2021380758A1 (es) |
| CA (1) | CA3199442A1 (es) |
| IL (1) | IL302839A (es) |
| MX (1) | MX2023005541A (es) |
| WO (1) | WO2022103980A1 (es) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR20200033249A (ko) | 2017-06-05 | 2020-03-27 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
| US12103926B2 (en) | 2018-03-27 | 2024-10-01 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| BR112020026545A2 (pt) | 2018-06-27 | 2021-03-23 | Ptc Therapeutics, Inc. | Compostos heterocíclicos e de heteroarila para o tratamento da doença de huntington |
| EA202092899A1 (ru) | 2018-06-27 | 2021-05-14 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Гетероарильные соединения для лечения болезни гентингтона |
| WO2025065630A1 (zh) * | 2023-09-28 | 2025-04-03 | 北京大学 | 利用rna剪接调节剂调控基因表达的核酸分子 |
Family Cites Families (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EA201800367A1 (ru) | 2015-12-10 | 2019-02-28 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы лечения болезни хантингтона |
| KR20200033249A (ko) | 2017-06-05 | 2020-03-27 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 화합물 |
| JP2020523365A (ja) * | 2017-06-14 | 2020-08-06 | ピーティーシー セラピューティクス，インコーポレーテッド | Ｒｎａスプライシングを改変する方法 |
| CA3067591A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Methods for treating huntington's disease |
| US11395822B2 (en) | 2017-06-28 | 2022-07-26 | Ptc Therapeutics, Inc. | Methods for treating Huntington's disease |
| BR112020026545A2 (pt) | 2018-06-27 | 2021-03-23 | Ptc Therapeutics, Inc. | Compostos heterocíclicos e de heteroarila para o tratamento da doença de huntington |
| WO2020190793A1 (en) * | 2019-03-15 | 2020-09-24 | Skyhawk Therapeutics, Inc. | Compositions and methods for correction of aberrant splicing |
| AU2020377204A1 (en) * | 2019-11-01 | 2022-06-02 | Novartis Ag | The use of a splicing modulator for a treatment slowing progression of huntington's disease |
-
2021
- 2021-11-11 AU AU2021380758A patent/AU2021380758A1/en active Pending
- 2021-11-11 MX MX2023005541A patent/MX2023005541A/es unknown
- 2021-11-11 EP EP21824134.7A patent/EP4244362A1/en active Pending
- 2021-11-11 US US17/524,586 patent/US20220162610A1/en not_active Abandoned
- 2021-11-11 WO PCT/US2021/059010 patent/WO2022103980A1/en not_active Ceased
- 2021-11-11 CA CA3199442A patent/CA3199442A1/en active Pending
- 2021-11-11 IL IL302839A patent/IL302839A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| IL302839A (en) | 2023-07-01 |
| US20220162610A1 (en) | 2022-05-26 |
| AU2021380758A1 (en) | 2023-06-15 |
| WO2022103980A9 (en) | 2022-08-04 |
| WO2022103980A1 (en) | 2022-05-19 |
| CA3199442A1 (en) | 2022-05-19 |
| EP4244362A1 (en) | 2023-09-20 |
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