BR112022011214A2 - Rnas-guia modificados para edição de gene - Google Patents
Rnas-guia modificados para edição de geneInfo
- Publication number
- BR112022011214A2 BR112022011214A2 BR112022011214A BR112022011214A BR112022011214A2 BR 112022011214 A2 BR112022011214 A2 BR 112022011214A2 BR 112022011214 A BR112022011214 A BR 112022011214A BR 112022011214 A BR112022011214 A BR 112022011214A BR 112022011214 A2 BR112022011214 A2 BR 112022011214A2
- Authority
- BR
- Brazil
- Prior art keywords
- gene editing
- guide rnas
- modified guide
- modified
- rnas
- Prior art date
Links
Classifications
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- 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
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- 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
-
- 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
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- 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
- 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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- 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 [CRISPRs]
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
-
- 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
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Crystallography & Structural Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Saccharide Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
RNAS-GUIA MODIFICADOS PARA EDIÇÃO DE GENE. A presente invenção refere-se a RNAs-guia modificados tendo atividade melhorada in vitro e in vivo em métodos de edição de gene.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962946905P | 2019-12-11 | 2019-12-11 | |
PCT/US2020/064250 WO2021119275A1 (en) | 2019-12-11 | 2020-12-10 | Modified guide rnas for gene editing |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112022011214A2 true BR112022011214A2 (pt) | 2022-08-23 |
Family
ID=74181290
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022011214A BR112022011214A2 (pt) | 2019-12-11 | 2020-12-10 | Rnas-guia modificados para edição de gene |
Country Status (13)
Country | Link |
---|---|
US (1) | US20220372483A1 (pt) |
EP (1) | EP4073249A1 (pt) |
JP (1) | JP2023506482A (pt) |
KR (1) | KR20220126725A (pt) |
CN (1) | CN115176001A (pt) |
AU (1) | AU2020401206A1 (pt) |
BR (1) | BR112022011214A2 (pt) |
CA (1) | CA3164192A1 (pt) |
CO (1) | CO2022009562A2 (pt) |
IL (1) | IL293569A (pt) |
MX (1) | MX2022006950A (pt) |
TW (1) | TW202136509A (pt) |
WO (1) | WO2021119275A1 (pt) |
Families Citing this family (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA3224995A1 (en) * | 2021-06-22 | 2022-12-29 | Intellia Therapeutics, Inc. | Methods for in vivo editing of a liver gene |
AU2022381173A1 (en) * | 2021-11-03 | 2024-05-02 | Intellia Therapeutics, Inc. | Modified guide rnas for gene editing |
WO2023081200A2 (en) | 2021-11-03 | 2023-05-11 | Intellia Therapeutics, Inc. | Cd38 compositions and methods for immunotherapy |
WO2023245108A2 (en) | 2022-06-16 | 2023-12-21 | Intellia Therapeutics, Inc. | Compositions and methods for reducing mhc class i in a cell |
WO2023245113A1 (en) | 2022-06-16 | 2023-12-21 | Intellia Therapeutics, Inc. | Methods and compositions for genetically modifying a cell |
WO2024006955A1 (en) | 2022-06-29 | 2024-01-04 | Intellia Therapeutics, Inc. | Engineered t cells |
Family Cites Families (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP3756313B2 (ja) | 1997-03-07 | 2006-03-15 | 武 今西 | 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体 |
DE04020014T1 (de) | 1997-09-12 | 2006-01-26 | Exiqon A/S | Bi-zyklische - Nukleosid,Nnukleotid und Oligonukleotid-Analoga |
EP1272840A1 (en) | 2000-04-03 | 2003-01-08 | The Board Of Trustees Of The Leland Stanford Junior University | A method for protein structure alignment |
US7774185B2 (en) | 2004-09-14 | 2010-08-10 | International Business Machines Corporation | Protein structure alignment using cellular automata |
CN113563476A (zh) | 2013-03-15 | 2021-10-29 | 通用医疗公司 | 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向 |
WO2015200555A2 (en) * | 2014-06-25 | 2015-12-30 | Caribou Biosciences, Inc. | Rna modification to engineer cas9 activity |
WO2016010840A1 (en) | 2014-07-16 | 2016-01-21 | Novartis Ag | Method of encapsulating a nucleic acid in a lipid nanoparticle host |
CA2969619A1 (en) * | 2014-12-03 | 2016-06-09 | Agilent Technologies, Inc. | Guide rna with chemical modifications |
EP3280803B1 (en) * | 2015-04-06 | 2021-05-26 | The Board of Trustees of the Leland Stanford Junior University | Chemically modified guide rnas for crispr/cas-mediated gene regulation |
EP3551757A1 (en) * | 2016-12-08 | 2019-10-16 | Intellia Therapeutics, Inc. | Modified guide rnas |
BR112020005287A2 (pt) * | 2017-09-29 | 2020-09-24 | Intellia Therapeutics, Inc. | composições e métodos para edição de gene ttr e tratar amiloidose attr |
AU2018339089A1 (en) | 2017-09-29 | 2020-04-09 | Intellia Therapeutics, Inc. | Polynucleotides, compositions, and methods for genome editing |
LT3688162T (lt) * | 2017-09-29 | 2024-05-27 | Intellia Therapeutics, Inc. | Preparatai |
AU2019282824A1 (en) * | 2018-06-08 | 2021-01-07 | Intellia Therapeutics, Inc. | Modified guide RNAS for gene editing |
-
2020
- 2020-12-10 WO PCT/US2020/064250 patent/WO2021119275A1/en active Application Filing
- 2020-12-10 KR KR1020227023444A patent/KR20220126725A/ko unknown
- 2020-12-10 AU AU2020401206A patent/AU2020401206A1/en active Pending
- 2020-12-10 TW TW109143632A patent/TW202136509A/zh unknown
- 2020-12-10 EP EP20839432.0A patent/EP4073249A1/en active Pending
- 2020-12-10 CN CN202080094797.XA patent/CN115176001A/zh active Pending
- 2020-12-10 BR BR112022011214A patent/BR112022011214A2/pt unknown
- 2020-12-10 JP JP2022535572A patent/JP2023506482A/ja active Pending
- 2020-12-10 CA CA3164192A patent/CA3164192A1/en active Pending
- 2020-12-10 IL IL293569A patent/IL293569A/en unknown
- 2020-12-10 MX MX2022006950A patent/MX2022006950A/es unknown
-
2022
- 2022-06-09 US US17/836,265 patent/US20220372483A1/en active Pending
- 2022-07-07 CO CONC2022/0009562A patent/CO2022009562A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP4073249A1 (en) | 2022-10-19 |
TW202136509A (zh) | 2021-10-01 |
CA3164192A1 (en) | 2021-06-17 |
JP2023506482A (ja) | 2023-02-16 |
CN115176001A (zh) | 2022-10-11 |
US20220372483A1 (en) | 2022-11-24 |
KR20220126725A (ko) | 2022-09-16 |
WO2021119275A1 (en) | 2021-06-17 |
CO2022009562A2 (es) | 2022-08-30 |
AU2020401206A1 (en) | 2022-07-14 |
MX2022006950A (es) | 2022-11-07 |
IL293569A (en) | 2022-08-01 |
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