BR112021020406A2 - Sistemas de edição de genes para modificar um gene scn9a ou scn10a e métodos de uso dos mesmos - Google Patents
Sistemas de edição de genes para modificar um gene scn9a ou scn10a e métodos de uso dos mesmosInfo
- Publication number
- BR112021020406A2 BR112021020406A2 BR112021020406A BR112021020406A BR112021020406A2 BR 112021020406 A2 BR112021020406 A2 BR 112021020406A2 BR 112021020406 A BR112021020406 A BR 112021020406A BR 112021020406 A BR112021020406 A BR 112021020406A BR 112021020406 A2 BR112021020406 A2 BR 112021020406A2
- Authority
- BR
- Brazil
- Prior art keywords
- gene
- editing systems
- scn9a
- scn10a
- modifying
- Prior art date
Links
- 238000010362 genome editing Methods 0.000 title abstract 5
- 108090000623 proteins and genes Proteins 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 2
- 108010053752 Voltage-Gated Sodium Channels Proteins 0.000 abstract 3
- 102000016913 Voltage-Gated Sodium Channels Human genes 0.000 abstract 2
- 102000007260 Deoxyribonuclease I Human genes 0.000 abstract 1
- 108010008532 Deoxyribonuclease I Proteins 0.000 abstract 1
- 108020005004 Guide RNA Proteins 0.000 abstract 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal 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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- 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
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/04—Centrally acting analgesics, e.g. opioids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
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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
- C12N15/11—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
- C12N15/1138—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 against receptors or cell surface proteins
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- 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
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- 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
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2320/00—Applications; Uses
- C12N2320/10—Applications; Uses in screening processes
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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- C12N2800/00—Nucleic acids vectors
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- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
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- Wood Science & Technology (AREA)
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- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Pain & Pain Management (AREA)
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Abstract
sistemas de edição de genes para modificar um gene scn9a ou scn10a e métodos de uso dos mesmos. são divulgados neste documento sistemas de edição de genes altamente eficientes para editar um gene de canal de sódio controlado por voltagem, tal como subunidade alfa 9 do canal de sódio controlado por voltagem (scn9a) ou subunidade alfa 10 do canal de sódio controlado por voltagem (scn10a), seja in vitro ou in vivo. os sistemas de edição de genes divulgados neste documento compreendem endonuclease de dna guiada por rna e rnas guia específicos. também são fornecidos neste documento os usos dos sistemas de edição de genes para modificar o gene alvo, aliviando, assim, a dor.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962833523P | 2019-04-12 | 2019-04-12 | |
PCT/US2020/027690 WO2020210640A1 (en) | 2019-04-12 | 2020-04-10 | Gene-editing systems for modifying a scn9a or scn10a gene and methods of use thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112021020406A2 true BR112021020406A2 (pt) | 2021-12-07 |
Family
ID=70416602
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112021020406A BR112021020406A2 (pt) | 2019-04-12 | 2020-04-10 | Sistemas de edição de genes para modificar um gene scn9a ou scn10a e métodos de uso dos mesmos |
Country Status (15)
Country | Link |
---|---|
US (1) | US20200354741A1 (pt) |
EP (1) | EP3953460A1 (pt) |
JP (1) | JP2022526670A (pt) |
KR (1) | KR20210151110A (pt) |
CN (1) | CN113994009A (pt) |
AU (1) | AU2020272031A1 (pt) |
BR (1) | BR112021020406A2 (pt) |
CA (1) | CA3136700A1 (pt) |
CO (1) | CO2021014015A2 (pt) |
EA (1) | EA202192806A1 (pt) |
IL (1) | IL287149A (pt) |
JO (1) | JOP20210276A1 (pt) |
MX (1) | MX2021012493A (pt) |
SG (1) | SG11202111270SA (pt) |
WO (1) | WO2020210640A1 (pt) |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN109843914B (zh) | 2016-07-06 | 2024-03-15 | 沃泰克斯药物股份有限公司 | 用于治疗疼痛相关病症的材料和方法 |
CA3029132A1 (en) | 2016-07-06 | 2018-01-11 | Crispr Therapeutics Ag | Materials and methods for treatment of pain related disorders |
US11898179B2 (en) * | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
-
2020
- 2020-04-10 MX MX2021012493A patent/MX2021012493A/es unknown
- 2020-04-10 SG SG11202111270SA patent/SG11202111270SA/en unknown
- 2020-04-10 WO PCT/US2020/027690 patent/WO2020210640A1/en active Application Filing
- 2020-04-10 JO JOP/2021/0276A patent/JOP20210276A1/ar unknown
- 2020-04-10 BR BR112021020406A patent/BR112021020406A2/pt unknown
- 2020-04-10 JP JP2021560111A patent/JP2022526670A/ja active Pending
- 2020-04-10 US US16/845,280 patent/US20200354741A1/en active Pending
- 2020-04-10 CN CN202080038231.5A patent/CN113994009A/zh active Pending
- 2020-04-10 KR KR1020217034109A patent/KR20210151110A/ko unknown
- 2020-04-10 EA EA202192806A patent/EA202192806A1/ru unknown
- 2020-04-10 AU AU2020272031A patent/AU2020272031A1/en active Pending
- 2020-04-10 EP EP20721089.9A patent/EP3953460A1/en active Pending
- 2020-04-10 CA CA3136700A patent/CA3136700A1/en active Pending
-
2021
- 2021-10-10 IL IL287149A patent/IL287149A/en unknown
- 2021-10-20 CO CONC2021/0014015A patent/CO2021014015A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP3953460A1 (en) | 2022-02-16 |
IL287149A (en) | 2021-12-01 |
KR20210151110A (ko) | 2021-12-13 |
US20200354741A1 (en) | 2020-11-12 |
CA3136700A1 (en) | 2020-10-15 |
CN113994009A (zh) | 2022-01-28 |
CO2021014015A2 (es) | 2021-10-29 |
SG11202111270SA (en) | 2021-11-29 |
JOP20210276A1 (ar) | 2023-01-30 |
JP2022526670A (ja) | 2022-05-25 |
MX2021012493A (es) | 2022-01-06 |
AU2020272031A1 (en) | 2021-11-18 |
WO2020210640A1 (en) | 2020-10-15 |
EA202192806A1 (ru) | 2022-02-03 |
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BR112021020406A2 (pt) | Sistemas de edição de genes para modificar um gene scn9a ou scn10a e métodos de uso dos mesmos |