KR20240052034A - 스플라이세오솜 구성요소의 동원을 통한 rna 편집 - Google Patents
스플라이세오솜 구성요소의 동원을 통한 rna 편집 Download PDFInfo
- Publication number
- KR20240052034A KR20240052034A KR1020247010496A KR20247010496A KR20240052034A KR 20240052034 A KR20240052034 A KR 20240052034A KR 1020247010496 A KR1020247010496 A KR 1020247010496A KR 20247010496 A KR20247010496 A KR 20247010496A KR 20240052034 A KR20240052034 A KR 20240052034A
- Authority
- KR
- South Korea
- Prior art keywords
- rna
- nucleotides
- composition
- molecule
- esnrna
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
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
- C12N15/11—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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- 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
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163240428P | 2021-09-03 | 2021-09-03 | |
| US63/240,428 | 2021-09-03 | ||
| US202263345660P | 2022-05-25 | 2022-05-25 | |
| US63/345,660 | 2022-05-25 | ||
| PCT/US2022/042486 WO2023034582A1 (en) | 2021-09-03 | 2022-09-02 | Rna editing via recruitment of spliceosome components |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20240052034A true KR20240052034A (ko) | 2024-04-22 |
Family
ID=85412903
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020247010496A Pending KR20240052034A (ko) | 2021-09-03 | 2022-09-02 | 스플라이세오솜 구성요소의 동원을 통한 rna 편집 |
Country Status (9)
| Country | Link |
|---|---|
| US (2) | US12152239B2 (https=) |
| EP (1) | EP4396350A4 (https=) |
| JP (1) | JP2024533163A (https=) |
| KR (1) | KR20240052034A (https=) |
| AU (1) | AU2022337146A1 (https=) |
| CA (1) | CA3230419A1 (https=) |
| IL (1) | IL311101A (https=) |
| MX (1) | MX2024002647A (https=) |
| WO (1) | WO2023034582A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2022220968A1 (en) * | 2021-04-15 | 2022-10-20 | U1 Bio, Inc. | High efficiency trans-splicing for replacement of targeted rna sequences in human cells |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
| EP4522758A4 (en) | 2022-05-13 | 2026-05-06 | Ascidian Therapeutics Inc | Abca4 trans-splicing molecules |
| WO2025085691A1 (en) | 2023-10-18 | 2025-04-24 | Tacit Therapeutics, Inc. | Trans-splicing nucleic acid molecules and methods of use |
| WO2025147110A1 (ko) * | 2024-01-02 | 2025-07-10 | 알지노믹스 주식회사 | Mecp2 전사체 특이적 트랜스-스플라이싱 리보자임 및 이의 용도 |
Family Cites Families (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20020193580A1 (en) | 1995-12-15 | 2002-12-19 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| IT1283876B1 (it) * | 1996-01-12 | 1998-05-07 | Univ Roma | Molecole chimeriche ribozima-snrna ad attivita' catalitica per rna a localizzazione nucleare |
| US20090148906A1 (en) | 1998-09-29 | 2009-06-11 | Shire Human Genetic Therapies, Inc. A Delaware Corporation | Optimized messenger rna |
| US20040214263A1 (en) * | 2002-03-20 | 2004-10-28 | Mitchell Lloyd G. | Spliceosome mediated RNA trans-splicing |
| FR2874384B1 (fr) * | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| EP2151248A1 (en) | 2008-07-30 | 2010-02-10 | Johann Bauer | Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses |
| US9303078B2 (en) | 2009-10-08 | 2016-04-05 | Association Institut De Myologie | Nucleic acid molecules and methods for exchanging exon(s) by transsplicing |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| IT1400425B1 (it) * | 2010-06-08 | 2013-05-31 | Amsterdam Molecular Therapeutics Bv | Modified snrnas for use in therapy. |
| IT1405793B1 (it) * | 2010-10-15 | 2014-01-24 | Univ Ferrara | Molecola di u1snrna umano modificato, gene codificante per la molecola di u1snrna umano modificato, vettore di espressione includente il gene, e loro uso in terapia genica |
| GB201219762D0 (en) | 2012-11-02 | 2012-12-19 | Bauer Johann | A RNA trans-splicing molecule (RTM) for use in the treatment of cancer |
| MX388221B (es) | 2013-04-20 | 2025-03-19 | Res Institute At Nationwide Children´S Hospital | ADMINISTRACIÓN DE VIRUS ADENO-ASOCIADO RECOMBINANTE DE CONSTRUCCIONES DE POLINUCLÉOTIDOS U7snRNA DIRIGIDA AL EXÓN 2. |
| CA3005474A1 (en) | 2015-11-19 | 2017-05-26 | The Trustees Of The University Of Pennsylvania | Compositions and methods for correction of heritable ocular disease |
| CA3005968A1 (en) | 2015-11-23 | 2017-06-01 | The Regents Of The University Of California | Tracking and manipulating cellular rna via nuclear delivery of crispr/cas9 |
| PL3386484T3 (pl) | 2015-12-10 | 2022-07-25 | Modernatx, Inc. | Kompozycje i sposoby dostarczania środków terapeutycznych |
| EP3436590A4 (en) * | 2016-04-01 | 2019-12-04 | National University of Singapore | TRANSSPLEISSEN OF RNA (TSRNA) |
| CN110612353A (zh) | 2017-03-03 | 2019-12-24 | 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 |
| US10476825B2 (en) | 2017-08-22 | 2019-11-12 | Salk Institue for Biological Studies | RNA targeting methods and compositions |
| KR102866133B1 (ko) | 2018-04-17 | 2025-09-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 트랜스-스플라이싱 분자 |
| KR102918544B1 (ko) | 2018-07-12 | 2026-01-27 | 로켓 파마슈티컬스, 리미티드 | 다논병을 치료하기 위한 유전자 요법 벡터 |
| US20220010333A1 (en) | 2018-09-06 | 2022-01-13 | The Regents Of The University Of California | Rna and dna base editing via engineered adar recruitment |
| US20210388351A1 (en) | 2018-09-28 | 2021-12-16 | The Jackson Laboratory | Artificial rna-guided splicing factors |
| WO2020160486A1 (en) | 2019-02-01 | 2020-08-06 | Spark Therapeutics, Inc. | Aav vector treatment methods for late infantile neuronal ceroid lipofuscinosis type 2 |
| WO2020205604A1 (en) | 2019-03-29 | 2020-10-08 | Salk Institute For Biological Studies | High-efficiency reconstitution of rna molecules |
| BR112021020539A2 (pt) | 2019-04-17 | 2022-01-04 | Univ Pennsylvania | Terminador de hélice tripla para trans-splicing de rna eficiente |
| CN115023499A (zh) | 2019-08-16 | 2022-09-06 | 麻省理工学院 | 使用CRISPR/Cas13的靶向反式剪接 |
| WO2021076656A1 (en) | 2019-10-15 | 2021-04-22 | University Of Massachusetts | Rna editor-enhanced rna trans-splicing |
| IL292904A (en) * | 2019-11-11 | 2022-07-01 | Salk Inst For Biological Studi | Compositions and methods for high-efficiency recombination of rna molecules |
| CN115777020A (zh) | 2020-04-22 | 2023-03-10 | 塑造治疗公司 | 使用snrna组分的组合物和方法 |
| CA3177380A1 (en) | 2020-05-26 | 2021-12-02 | Shape Therapeutics Inc. | Compositions and methods for modifying target rnas |
| EP4217010A4 (en) | 2020-09-28 | 2025-01-08 | Tacit Therapeutics, Inc. | TRANS-SPLICING SYSTEM FOR TISSUE-SPECIFIC REPLACEMENT OF RNA SEQUENCES |
| AU2022337146A1 (en) | 2021-09-03 | 2024-03-14 | Tacit Therapeutics, Inc. | Rna editing via recruitment of spliceosome components |
-
2022
- 2022-09-02 AU AU2022337146A patent/AU2022337146A1/en active Pending
- 2022-09-02 MX MX2024002647A patent/MX2024002647A/es unknown
- 2022-09-02 WO PCT/US2022/042486 patent/WO2023034582A1/en not_active Ceased
- 2022-09-02 EP EP22865613.8A patent/EP4396350A4/en active Pending
- 2022-09-02 KR KR1020247010496A patent/KR20240052034A/ko active Pending
- 2022-09-02 CA CA3230419A patent/CA3230419A1/en active Pending
- 2022-09-02 IL IL311101A patent/IL311101A/en unknown
- 2022-09-02 JP JP2024513942A patent/JP2024533163A/ja active Pending
-
2023
- 2023-07-31 US US18/362,698 patent/US12152239B2/en active Active
-
2024
- 2024-10-10 US US18/911,917 patent/US20250051764A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4396350A1 (en) | 2024-07-10 |
| WO2023034582A1 (en) | 2023-03-09 |
| US20240011026A1 (en) | 2024-01-11 |
| US20250051764A1 (en) | 2025-02-13 |
| MX2024002647A (es) | 2024-05-10 |
| CA3230419A1 (en) | 2023-03-09 |
| JP2024533163A (ja) | 2024-09-12 |
| AU2022337146A1 (en) | 2024-03-14 |
| EP4396350A4 (en) | 2026-01-14 |
| US12152239B2 (en) | 2024-11-26 |
| IL311101A (en) | 2024-04-01 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| PA0105 | International application |
St.27 status event code: A-0-1-A10-A15-nap-PA0105 |
|
| PG1501 | Laying open of application |
St.27 status event code: A-1-1-Q10-Q12-nap-PG1501 |
|
| E13 | Pre-grant limitation requested |
Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
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| E13-X000 | Pre-grant limitation requested |
St.27 status event code: A-2-3-E10-E13-lim-X000 |
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| P11 | Amendment of application requested |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
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| P11-X000 | Amendment of application requested |
St.27 status event code: A-2-2-P10-P11-nap-X000 |
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| P13 | Application amended |
Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE) |
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| P13-X000 | Application amended |
St.27 status event code: A-2-2-P10-P13-nap-X000 |