EA202091707A1 - Хиноксалиноновые соединения, композиции, способы и наборы для повышения эффективности редактирования генома - Google Patents
Хиноксалиноновые соединения, композиции, способы и наборы для повышения эффективности редактирования геномаInfo
- Publication number
- EA202091707A1 EA202091707A1 EA202091707A EA202091707A EA202091707A1 EA 202091707 A1 EA202091707 A1 EA 202091707A1 EA 202091707 A EA202091707 A EA 202091707A EA 202091707 A EA202091707 A EA 202091707A EA 202091707 A1 EA202091707 A1 EA 202091707A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- methods
- dna
- genome editing
- kits
- compositions
- Prior art date
Links
- 238000000034 method Methods 0.000 title abstract 5
- 238000010362 genome editing Methods 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 2
- XSCHRSMBECNVNS-UHFFFAOYSA-N quinoxaline Chemical class N1=CC=NC2=CC=CC=C21 XSCHRSMBECNVNS-UHFFFAOYSA-N 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 230000037361 pathway Effects 0.000 abstract 2
- 150000001875 compounds Chemical class 0.000 abstract 1
- 230000011559 double-strand break repair via nonhomologous end joining Effects 0.000 abstract 1
- 239000003112 inhibitor Substances 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 229940043355 kinase inhibitor Drugs 0.000 abstract 1
- 239000003909 protein kinase inhibitor Substances 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D403/00—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
- C07D403/02—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
- C07D403/12—Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings linked by a chain containing hetero atoms as chain links
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
- A61K31/5375—1,4-Oxazines, e.g. morpholine
- A61K31/5377—1,4-Oxazines, e.g. morpholine not condensed and containing further heterocyclic rings, e.g. timolol
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/33—Heterocyclic compounds
- A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
- A61K31/535—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one oxygen as the ring hetero atoms, e.g. 1,2-oxazines
- A61K31/5375—1,4-Oxazines, e.g. morpholine
- A61K31/5386—1,4-Oxazines, e.g. morpholine spiro-condensed or forming part of bridged ring systems
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D241/00—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings
- C07D241/36—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings condensed with carbocyclic rings or ring systems
- C07D241/38—Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings condensed with carbocyclic rings or ring systems with only hydrogen or carbon atoms directly attached to the ring nitrogen atoms
- C07D241/40—Benzopyrazines
- C07D241/44—Benzopyrazines with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to carbon atoms of the hetero ring
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D405/00—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom
- C07D405/14—Heterocyclic compounds containing both one or more hetero rings having oxygen atoms as the only ring hetero atoms, and one or more rings having nitrogen as the only ring hetero atom containing three or more hetero rings
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
- C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
- C07D491/04—Ortho-condensed systems
- C07D491/044—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring
- C07D491/048—Ortho-condensed systems with only one oxygen atom as ring hetero atom in the oxygen-containing ring the oxygen-containing ring being five-membered
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D491/00—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00
- C07D491/02—Heterocyclic compounds containing in the condensed ring system both one or more rings having oxygen atoms as the only ring hetero atoms and one or more rings having nitrogen atoms as the only ring hetero atoms, not provided for by groups C07D451/00 - C07D459/00, C07D463/00, C07D477/00 or C07D489/00 in which the condensed system contains two hetero rings
- C07D491/08—Bridged systems
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D498/00—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms
- C07D498/02—Heterocyclic compounds containing in the condensed system at least one hetero ring having nitrogen and oxygen atoms as the only ring hetero atoms in which the condensed system contains two hetero rings
- C07D498/08—Bridged systems
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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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
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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
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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
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
- C12N9/22—Ribonucleases RNAses, DNAses
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
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- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Cell Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Mycology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Crystallography & Structural Chemistry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Plural Heterocyclic Compounds (AREA)
- Nitrogen Condensed Heterocyclic Rings (AREA)
Abstract
Предложены соединения, способы редактирования целевой геномной области(ей), способы репарации разрыва ДНК через путь HDR, способы ингибирования или подавления репарации разрыва ДНК, проходящей через путь NHEJ, и способы модификации экспрессии гена(ов) или белка(ов), которые включают введение в одну или несколько клеток, которые содержат одну или несколько целевых геномных областей, системы редактирования генома и ингибитора ДНК-протеинкиназы (ДНК-РК), которые описаны в настоящей заявке. Предложены также наборы и композиции для редактирования гена-мишени, которые содержат систему редактирования генома и ингибитор ДНК-РК, которые описаны в настоящей заявке.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201862618385P | 2018-01-17 | 2018-01-17 | |
PCT/US2019/013785 WO2019143677A1 (en) | 2018-01-17 | 2019-01-16 | Quinoxalinone compounds, compositions, methods, and kits for increasing genome editing efficiency |
Publications (1)
Publication Number | Publication Date |
---|---|
EA202091707A1 true EA202091707A1 (ru) | 2020-12-02 |
Family
ID=65516730
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
EA202091707A EA202091707A1 (ru) | 2018-01-17 | 2019-01-16 | Хиноксалиноновые соединения, композиции, способы и наборы для повышения эффективности редактирования генома |
Country Status (13)
Country | Link |
---|---|
US (1) | US20200361877A1 (ru) |
EP (1) | EP3740483B1 (ru) |
JP (1) | JP7483612B2 (ru) |
KR (1) | KR20200110687A (ru) |
CN (1) | CN111770921B (ru) |
AU (1) | AU2019209292B2 (ru) |
BR (1) | BR112020013626A2 (ru) |
CA (1) | CA3088791A1 (ru) |
EA (1) | EA202091707A1 (ru) |
ES (1) | ES2940786T3 (ru) |
IL (1) | IL276081B2 (ru) |
MA (1) | MA51623A (ru) |
WO (1) | WO2019143677A1 (ru) |
Families Citing this family (9)
Publication number | Priority date | Publication date | Assignee | Title |
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WO2018002812A1 (en) | 2016-06-29 | 2018-01-04 | Crispr Therapeutics Ag | Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders |
AU2019209293B2 (en) * | 2018-01-17 | 2023-07-27 | Vertex Pharmaceuticals Incorporated | DNA-PK inhibitors |
KR20220070443A (ko) | 2019-08-27 | 2022-05-31 | 버텍스 파마슈티칼스 인코포레이티드 | 반복성 dna와 연관된 장애의 치료용 조성물 및 방법 |
CN112750498B (zh) * | 2020-12-30 | 2022-06-24 | 同济大学 | 靶向逆转录引物结合位点从而抑制hiv病毒复制的方法 |
TW202246510A (zh) | 2021-02-26 | 2022-12-01 | 美商維泰克斯製藥公司 | 以crispr/slucas9治療第1型肌強直性營養不良之組合物及方法 |
WO2022182957A1 (en) | 2021-02-26 | 2022-09-01 | Vertex Pharmaceuticals Incorporated | Compositions and methods for treatment of myotonic dystrophy type 1 with crispr/sacas9 |
WO2023018637A1 (en) | 2021-08-09 | 2023-02-16 | Vertex Pharmaceuticals Incorporated | Gene editing of regulatory elements |
CN114685494B (zh) * | 2022-03-16 | 2024-04-16 | 中国人民解放军军事科学院军事医学研究院 | SpCas9抑制剂的合成制备方法及其应用 |
WO2023215991A1 (en) * | 2022-05-11 | 2023-11-16 | adMare Therapeutics Society | Dna-pk inhibitor compounds and uses thereof |
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2019
- 2019-01-16 US US16/962,443 patent/US20200361877A1/en active Pending
- 2019-01-16 JP JP2020539234A patent/JP7483612B2/ja active Active
- 2019-01-16 KR KR1020207023672A patent/KR20200110687A/ko not_active Application Discontinuation
- 2019-01-16 WO PCT/US2019/013785 patent/WO2019143677A1/en active Application Filing
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MA51623A (fr) | 2021-04-14 |
WO2019143677A1 (en) | 2019-07-25 |
JP2021511312A (ja) | 2021-05-06 |
IL276081A (en) | 2020-08-31 |
CN111770921B (zh) | 2024-03-22 |
IL276081B2 (en) | 2023-10-01 |
EP3740483B1 (en) | 2023-01-11 |
CN111770921A (zh) | 2020-10-13 |
EP3740483A1 (en) | 2020-11-25 |
IL276081B1 (en) | 2023-06-01 |
ES2940786T3 (es) | 2023-05-11 |
US20200361877A1 (en) | 2020-11-19 |
CA3088791A1 (en) | 2019-07-25 |
BR112020013626A2 (pt) | 2020-12-01 |
AU2019209292B2 (en) | 2023-10-05 |
AU2019209292A1 (en) | 2020-09-03 |
KR20200110687A (ko) | 2020-09-24 |
JP7483612B2 (ja) | 2024-05-15 |
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