IL312508A - Polynucleotides, compounds and methods for genome editing - Google Patents
Polynucleotides, compounds and methods for genome editingInfo
- Publication number
- IL312508A IL312508A IL312508A IL31250824A IL312508A IL 312508 A IL312508 A IL 312508A IL 312508 A IL312508 A IL 312508A IL 31250824 A IL31250824 A IL 31250824A IL 312508 A IL312508 A IL 312508A
- Authority
- IL
- Israel
- Prior art keywords
- polynucleotides
- compositions
- methods
- genome editing
- editing
- Prior art date
Links
- 238000010362 genome editing Methods 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 239000000203 mixture Substances 0.000 title 1
- 102000040430 polynucleotide Human genes 0.000 title 1
- 108091033319 polynucleotide Proteins 0.000 title 1
- 239000002157 polynucleotide Substances 0.000 title 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
- 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 [RNase]; Deoxyribonucleases [DNase]
-
- 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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
- A61K48/0016—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
- A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
- A61K9/51—Nanocapsules; Nanoparticles
- A61K9/5107—Excipients; Inactive ingredients
- A61K9/5123—Organic compounds, e.g. fats, sugars
-
- 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
- C12N15/63—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
- 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
- C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
-
- 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
- C12N15/87—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
-
- 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
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
- C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
-
- 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
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
-
- 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)
- C12Y305/04005—Cytidine deaminase (3.5.4.5)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Medicinal Chemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Nanotechnology (AREA)
- Optics & Photonics (AREA)
- Cell Biology (AREA)
- Mycology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163275425P | 2021-11-03 | 2021-11-03 | |
| US202263352158P | 2022-06-14 | 2022-06-14 | |
| PCT/US2022/079124 WO2023081689A2 (en) | 2021-11-03 | 2022-11-02 | Polynucleotides, compositions, and methods for genome editing |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL312508A true IL312508A (en) | 2024-07-01 |
Family
ID=84537879
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL312508A IL312508A (en) | 2021-11-03 | 2022-11-02 | Polynucleotides, compounds and methods for genome editing |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240301377A1 (enExample) |
| EP (1) | EP4426822A2 (enExample) |
| JP (1) | JP2024542995A (enExample) |
| KR (1) | KR20240114296A (enExample) |
| AU (1) | AU2022382975A1 (enExample) |
| CA (1) | CA3237303A1 (enExample) |
| CL (1) | CL2024001332A1 (enExample) |
| CO (1) | CO2024007019A2 (enExample) |
| IL (1) | IL312508A (enExample) |
| MX (1) | MX2024005242A (enExample) |
| TW (1) | TW202325848A (enExample) |
| WO (1) | WO2023081689A2 (enExample) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN115516773A (zh) * | 2020-05-08 | 2022-12-23 | 瑞典爱立信有限公司 | 多功能aas接收机 |
| WO2021228349A1 (en) | 2020-05-11 | 2021-11-18 | Telefonaktiebolaget Lm Ericsson (Publ) | Efficient prach scheduling |
| WO2025081064A2 (en) * | 2023-10-11 | 2025-04-17 | Illumina, Inc. | Thermophilic deaminase and methods for identifying modified cytosine |
| WO2025137301A1 (en) * | 2023-12-20 | 2025-06-26 | Intellia Therapeutics, Inc. | Methods for rapid engineering of cells |
Family Cites Families (27)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5585481A (en) | 1987-09-21 | 1996-12-17 | Gen-Probe Incorporated | Linking reagents for nucleotide probes |
| US5378825A (en) | 1990-07-27 | 1995-01-03 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogs |
| JP3131222B2 (ja) | 1991-12-24 | 2001-01-31 | アイシス・ファーマシューティカルス・インコーポレーテッド | ギャップを有する２′修飾オリゴヌクレオチド |
| JPH10500310A (ja) | 1994-05-19 | 1998-01-13 | ダコ アクティーゼルスカブ | 淋菌及びトラコーマ クラミジアの検出のためのｐｎａ プローブ |
| US20060051405A1 (en) | 2004-07-19 | 2006-03-09 | Protiva Biotherapeutics, Inc. | Compositions for the delivery of therapeutic agents and uses thereof |
| DK2931891T3 (da) | 2012-12-17 | 2019-08-19 | Harvard College | Rna-styret modificering af menneskelige genomer |
| CN107879960B (zh) | 2013-03-08 | 2021-06-22 | 诺华股份有限公司 | 用于传递活性成分的脂质和脂质组合物 |
| CN112301024A (zh) | 2013-03-15 | 2021-02-02 | 通用医疗公司 | 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性 |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| EP3083556B1 (en) | 2013-12-19 | 2019-12-25 | Novartis AG | Lipids and lipid compositions for the delivery of active agents |
| CN106794141B (zh) | 2014-07-16 | 2021-05-28 | 诺华股份有限公司 | 将核酸包封在脂质纳米粒主体中的方法 |
| CN120842296A (zh) | 2015-09-21 | 2025-10-28 | 垂林克生物技术有限公司 | 用于合成5’-加帽rna的组合物和方法 |
| LT3436077T (lt) | 2016-03-30 | 2025-06-25 | Intellia Therapeutics, Inc. | Lipidų nanodalelių vaisto formos, skirtos crispr/cas komponentams |
| WO2018067447A1 (en) | 2016-10-03 | 2018-04-12 | Itellia Therapeutics, Inc. | Improved methods for identifying double strand break sites |
| KR20240116572A (ko) * | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| WO2019067910A1 (en) | 2017-09-29 | 2019-04-04 | Intellia Therapeutics, Inc. | POLYNUCLEOTIDES, COMPOSITIONS AND METHODS FOR GENOMIC EDITION |
| US20230140670A1 (en) | 2017-09-29 | 2023-05-04 | Intellia Therapeutics, Inc. | Formulations |
| IL317505A (en) | 2017-11-10 | 2025-02-01 | Univ Massachusetts | CRISPR-directed delivery platforms |
| WO2020051562A2 (en) * | 2018-09-07 | 2020-03-12 | Beam Therapeutics Inc. | Compositions and methods for improving base editing |
| US20220009878A1 (en) | 2018-10-02 | 2022-01-13 | Intellia Therapeutics, Inc. | Ionizable amine lipids |
| IL282267B2 (en) * | 2018-10-15 | 2026-03-01 | Univ Massachusetts | Programmed DNA editing by chimeric deaminase-nme2cas9 proteins |
| BR112021010853A2 (pt) | 2018-12-05 | 2021-08-31 | Intellia Therapeutics, Inc. | Lipídios de amina modificados |
| MX2021011757A (es) | 2019-03-28 | 2021-12-10 | Intellia Therapeutics Inc | Polinucleótidos, composiciones y métodos para la expresión de polipéptidos. |
| CN114127044A (zh) | 2019-04-25 | 2022-03-01 | 英特利亚治疗股份有限公司 | 可电离的胺类脂质和脂质纳米颗粒 |
| AR124555A1 (es) * | 2019-06-11 | 2023-04-12 | Pairwise Plants Services Inc | Métodos para producir plantas con desarrollo de frutas alteradas y plantas derivas de las mismas |
| EP4028063A1 (en) * | 2019-09-13 | 2022-07-20 | Regeneron Pharmaceuticals, Inc. | Transcription modulation in animals using crispr/cas systems delivered by lipid nanoparticles |
| IL303506A (en) * | 2020-12-11 | 2023-08-01 | Intellia Therapeutics Inc | Polynucleotides, compositions, and methods for genome editing involving deamination |
-
2022
- 2022-11-02 CA CA3237303A patent/CA3237303A1/en active Pending
- 2022-11-02 MX MX2024005242A patent/MX2024005242A/es unknown
- 2022-11-02 TW TW111141875A patent/TW202325848A/zh unknown
- 2022-11-02 EP EP22826533.6A patent/EP4426822A2/en active Pending
- 2022-11-02 JP JP2024525233A patent/JP2024542995A/ja active Pending
- 2022-11-02 AU AU2022382975A patent/AU2022382975A1/en active Pending
- 2022-11-02 KR KR1020247018182A patent/KR20240114296A/ko active Pending
- 2022-11-02 IL IL312508A patent/IL312508A/en unknown
- 2022-11-02 WO PCT/US2022/079124 patent/WO2023081689A2/en not_active Ceased
-
2024
- 2024-04-30 CL CL2024001332A patent/CL2024001332A1/es unknown
- 2024-05-01 US US18/652,180 patent/US20240301377A1/en active Pending
- 2024-05-31 CO CONC2024/0007019A patent/CO2024007019A2/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP4426822A2 (en) | 2024-09-11 |
| KR20240114296A (ko) | 2024-07-23 |
| US20240301377A1 (en) | 2024-09-12 |
| TW202325848A (zh) | 2023-07-01 |
| JP2024542995A (ja) | 2024-11-19 |
| CO2024007019A2 (es) | 2024-06-07 |
| CL2024001332A1 (es) | 2024-11-15 |
| AU2022382975A1 (en) | 2024-05-02 |
| CA3237303A1 (en) | 2023-05-11 |
| WO2023081689A2 (en) | 2023-05-11 |
| WO2023081689A3 (en) | 2023-06-08 |
| MX2024005242A (es) | 2024-07-02 |
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