MX2021000343A - Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas. - Google Patents
Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas.Info
- Publication number
- MX2021000343A MX2021000343A MX2021000343A MX2021000343A MX2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A MX 2021000343 A MX2021000343 A MX 2021000343A
- Authority
- MX
- Mexico
- Prior art keywords
- guide rna
- traceless
- vesicles
- delivery
- production method
- Prior art date
Links
- 108020005004 Guide RNA Proteins 0.000 title abstract 3
- 101710163270 Nuclease Proteins 0.000 title 1
- 238000004519 manufacturing process Methods 0.000 title 1
- 108091032973 (ribonucleotides)n+m Proteins 0.000 abstract 1
- 108091033409 CRISPR Proteins 0.000 abstract 1
- 238000010354 CRISPR gene editing Methods 0.000 abstract 1
- 101710091045 Envelope protein Proteins 0.000 abstract 1
- 101710188315 Protein X Proteins 0.000 abstract 1
- 102000004389 Ribonucleoproteins Human genes 0.000 abstract 1
- 108010081734 Ribonucleoproteins Proteins 0.000 abstract 1
- 102100021696 Syncytin-1 Human genes 0.000 abstract 1
- 230000000799 fusogenic effect Effects 0.000 abstract 1
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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
-
- 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
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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
- C12N15/87—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
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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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
-
- 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]
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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 [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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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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
- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16023—Virus like particles [VLP]
-
- 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
- C12N2760/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssRNA viruses negative-sense
- C12N2760/00011—Details
- C12N2760/20011—Rhabdoviridae
- C12N2760/20211—Vesiculovirus, e.g. vesicular stomatitis Indiana virus
- C12N2760/20223—Virus like particles [VLP]
-
- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Medicinal Chemistry (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Virology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
La presente invención describe vesículas que llevan ARNg y/o complejos de ribonucleoproteína nucleasa (RNP) guiados por ARN asociado a CRISPR que se administran eficazmente a las células diana a través de proteínas fusogénicas de envoltura.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| IT201800007055 | 2018-07-10 | ||
| PCT/IB2019/055805 WO2020012335A1 (en) | 2018-07-10 | 2019-07-08 | Vesicles for traceless delivery of guide rna molecules and/or guide rna molecule/rna-guided nuclease complex(es) and a production method thereof |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2021000343A true MX2021000343A (es) | 2021-05-28 |
Family
ID=63684346
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021000343A MX2021000343A (es) | 2018-07-10 | 2019-07-08 | Vesículas para la administración sin trazas de moléculas de arn guía y/o complejos de nucleasa guiado por arn/molécula de arn guía y un método de producción de las mismas. |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20210261957A1 (es) |
| EP (1) | EP3820995A1 (es) |
| JP (1) | JP2021524272A (es) |
| KR (1) | KR20210044213A (es) |
| BR (1) | BR112021000408A2 (es) |
| CA (1) | CA3105925A1 (es) |
| MX (1) | MX2021000343A (es) |
| WO (1) | WO2020012335A1 (es) |
Families Citing this family (37)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| WO2019051097A1 (en) | 2017-09-08 | 2019-03-14 | The Regents Of The University Of California | RNA-GUIDED ENDONUCLEASE FUSION POLYPEPTIDES AND METHODS OF USING SAME |
| EP3820495A4 (en) | 2018-07-09 | 2022-07-20 | The Broad Institute Inc. | RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES |
| KR20240093924A (ko) | 2018-09-18 | 2024-06-24 | 브이엔브이 뉴코 인크. | Arc 기반 캡시드 및 이의 용도 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| AU2020242032A1 (en) | 2019-03-19 | 2021-10-07 | Massachusetts Institute Of Technology | Methods and compositions for editing nucleotide sequences |
| US20220195455A1 (en) * | 2019-04-03 | 2022-06-23 | University Of Georgia Research Foundation, Inc. | Delivery of crispr/mcas9 through extracellular vesicles for genome editing |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2020219713A1 (en) | 2019-04-23 | 2020-10-29 | Case Western Reserve University | Fusogenic particles and related methods for delivering therapeutic agents to cells |
| JP7808292B2 (ja) | 2019-06-13 | 2026-01-29 | ザ ジェネラル ホスピタル コーポレイション | 操作されたヒト内在性ウイルス様粒子および細胞への送達のためのその使用方法 |
| AU2021236683A1 (en) | 2020-03-19 | 2022-11-17 | Intellia Therapeutics, Inc. | Methods and compositions for directed genome editing |
| US20230365989A1 (en) * | 2020-03-20 | 2023-11-16 | The Broad Institute, Inc. | Compositions and methods for enhanced lentiviral production |
| TW202208626A (zh) * | 2020-04-24 | 2022-03-01 | 美商生命編輯公司 | Rna引導核酸酶及其活性片段與變體,以及使用方法 |
| MX2023001028A (es) | 2020-07-24 | 2023-04-24 | Massachusetts Gen Hospital | Particulas similares a virus potenciadas y metodos de uso de las mismas para suministro a celulas. |
| CN116348768A (zh) * | 2020-10-09 | 2023-06-27 | 亥姆霍兹慕尼黑中心-德国健康与环境研究中心(有限公司) | 确定是否存在中和病毒的抗体的方法和筛选化合物中和病毒能力的体外方法 |
| EP4247951A2 (en) * | 2020-11-19 | 2023-09-27 | Wake Forest University Health Sciences | Vectors, systems and methods for eukaryotic gene editing |
| WO2022152746A1 (en) | 2021-01-13 | 2022-07-21 | Alia Therapeutics Srl | K526d cas9 variants and applications thereof |
| WO2022261150A2 (en) | 2021-06-09 | 2022-12-15 | Scribe Therapeutics Inc. | Particle delivery systems |
| IL311218A (en) * | 2021-09-08 | 2024-05-01 | Flagship Pioneering Innovations Vi Llc | PAH Modulating Compositions and Methods |
| AU2022375820A1 (en) | 2021-11-01 | 2024-06-13 | Tome Biosciences, Inc. | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
| CN115725658B (zh) * | 2021-11-12 | 2025-03-04 | 中日友好医院(中日友好临床医学研究所) | 一种新型的基于水疱性口炎病毒的crispr rna递送系统及其应用 |
| EP4453196A1 (en) | 2021-12-21 | 2024-10-30 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
| WO2023194359A1 (en) | 2022-04-04 | 2023-10-12 | Alia Therapeutics Srl | Compositions and methods for treatment of usher syndrome type 2a |
| CN115261340A (zh) * | 2022-05-20 | 2022-11-01 | 四川大学 | 一种gh-1噬菌体病毒样颗粒、制备方法及应用 |
| WO2023225670A2 (en) | 2022-05-20 | 2023-11-23 | Tome Biosciences, Inc. | Ex vivo programmable gene insertion |
| WO2024020587A2 (en) | 2022-07-22 | 2024-01-25 | Tome Biosciences, Inc. | Pleiopluripotent stem cell programmable gene insertion |
| WO2024056880A2 (en) | 2022-09-16 | 2024-03-21 | Alia Therapeutics Srl | Enqp type ii cas proteins and applications thereof |
| EP4619535A1 (en) | 2022-11-16 | 2025-09-24 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
| CN116083488A (zh) * | 2023-01-03 | 2023-05-09 | 北京镁伽机器人科技有限公司 | 基因编辑方法、基因编辑方法得到的细胞、基因编辑系统及其应用 |
| CN116271102A (zh) * | 2023-01-09 | 2023-06-23 | 浙江大学 | 用于药物递送的含修饰蛋白的双层膜囊泡及其制备方法和应用 |
| CN120882867A (zh) | 2023-01-11 | 2025-10-31 | 阿利亚治疗有限公司 | Ii型cas蛋白及其应用 |
| WO2025003344A1 (en) | 2023-06-28 | 2025-01-02 | Alia Therapeutics Srl | Type ii cas proteins and applications thereof |
| WO2025021702A1 (en) | 2023-07-21 | 2025-01-30 | Alia Therapeutics S.R.L. | Cas9 orthologue nuclease and uses thereof |
| EP4600357A1 (en) * | 2024-02-12 | 2025-08-13 | Technische Universität München | Delivery system for gene editing tools |
| WO2025210147A1 (en) | 2024-04-04 | 2025-10-09 | Alia Therapeutics Srl | Type v cas proteins and applications thereof |
| WO2025224182A2 (en) | 2024-04-23 | 2025-10-30 | Basecamp Research Ltd | Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo |
| CN119662813A (zh) * | 2025-02-20 | 2025-03-21 | 聚芯堂(成都)生物科技有限公司 | 一种CRISPR技术检测AD靶标Micro RNA的检测试剂、试剂盒及其检测方法 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8697359B1 (en) * | 2012-12-12 | 2014-04-15 | The Broad Institute, Inc. | CRISPR-Cas systems and methods for altering expression of gene products |
| US9593356B2 (en) | 2013-06-11 | 2017-03-14 | Takara Bio Usa, Inc. | Protein enriched microvesicles and methods of making and using the same |
| WO2015191911A2 (en) | 2014-06-12 | 2015-12-17 | Clontech Laboratories, Inc. | Protein enriched microvesicles and methods of making and using the same |
| US20150376586A1 (en) | 2014-06-25 | 2015-12-31 | Caribou Biosciences, Inc. | RNA Modification to Engineer Cas9 Activity |
| US9816080B2 (en) * | 2014-10-31 | 2017-11-14 | President And Fellows Of Harvard College | Delivery of CAS9 via ARRDC1-mediated microvesicles (ARMMs) |
| CA2978314A1 (en) | 2015-03-03 | 2016-09-09 | The General Hospital Corporation | Engineered crispr-cas9 nucleases with altered pam specificity |
| CN108290933A (zh) * | 2015-06-18 | 2018-07-17 | 布罗德研究所有限公司 | 降低脱靶效应的crispr酶突变 |
| CN108350449B (zh) | 2015-08-28 | 2022-05-31 | 通用医疗公司 | 工程化的CRISPR-Cas9核酸酶 |
| WO2017068077A1 (en) * | 2015-10-20 | 2017-04-27 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods and products for genetic engineering |
-
2019
- 2019-07-08 EP EP19769219.7A patent/EP3820995A1/en not_active Withdrawn
- 2019-07-08 JP JP2021500606A patent/JP2021524272A/ja active Pending
- 2019-07-08 WO PCT/IB2019/055805 patent/WO2020012335A1/en not_active Ceased
- 2019-07-08 MX MX2021000343A patent/MX2021000343A/es unknown
- 2019-07-08 BR BR112021000408-4A patent/BR112021000408A2/pt unknown
- 2019-07-08 US US17/259,165 patent/US20210261957A1/en not_active Abandoned
- 2019-07-08 CA CA3105925A patent/CA3105925A1/en active Pending
- 2019-07-08 KR KR1020217004013A patent/KR20210044213A/ko not_active Ceased
Also Published As
| Publication number | Publication date |
|---|---|
| EP3820995A1 (en) | 2021-05-19 |
| US20210261957A1 (en) | 2021-08-26 |
| WO2020012335A1 (en) | 2020-01-16 |
| BR112021000408A2 (pt) | 2021-06-29 |
| CA3105925A1 (en) | 2020-01-16 |
| KR20210044213A (ko) | 2021-04-22 |
| JP2021524272A (ja) | 2021-09-13 |
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