CO2021017544A2 - Composiciones y métodos para la regulación génica selectiva - Google Patents
Composiciones y métodos para la regulación génica selectivaInfo
- Publication number
- CO2021017544A2 CO2021017544A2 CONC2021/0017544A CO2021017544A CO2021017544A2 CO 2021017544 A2 CO2021017544 A2 CO 2021017544A2 CO 2021017544 A CO2021017544 A CO 2021017544A CO 2021017544 A2 CO2021017544 A2 CO 2021017544A2
- Authority
- CO
- Colombia
- Prior art keywords
- compositions
- methods
- gene regulation
- selective gene
- selective
- Prior art date
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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
- 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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- 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
- A61P25/08—Antiepileptics; Anticonvulsants
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
- C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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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
- C12N15/62—DNA sequences coding for fusion proteins
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- C—CHEMISTRY; METALLURGY
- 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
- C12N15/63—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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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
- C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2319/00—Fusion polypeptide
- C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
- C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10344—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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- C12N2740/00—Reverse transcribing RNA viruses
- C12N2740/00011—Details
- C12N2740/10011—Retroviridae
- C12N2740/16011—Human Immunodeficiency Virus, HIV
- C12N2740/16041—Use of virus, viral particle or viral elements as a vector
- C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- 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
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14151—Methods of production or purification of viral material
- C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/007—Vectors comprising a special translation-regulating system cell or tissue specific
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
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- Wood Science & Technology (AREA)
- Biotechnology (AREA)
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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Abstract
En la presente se proporcionan factores de transcripción modificados para la regulación por aumento selectiva de SCN1a y usos de los mismos para tratar enfermedades y trastornos, tal como, síndrome de Dravet. También se proporcionan sitios de unión de microARN y usos de los mismos para la expresión selectiva en neuronas de parvalbúmina
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962854238P | 2019-05-29 | 2019-05-29 | |
US201962857727P | 2019-06-05 | 2019-06-05 | |
US202063008569P | 2020-04-10 | 2020-04-10 | |
PCT/US2020/035431 WO2020243651A1 (en) | 2019-05-29 | 2020-05-29 | Compositions and methods for selective gene regulation |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2021017544A2 true CO2021017544A2 (es) | 2022-01-17 |
Family
ID=73552430
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2021/0017544A CO2021017544A2 (es) | 2019-05-29 | 2021-12-21 | Composiciones y métodos para la regulación génica selectiva |
Country Status (15)
Country | Link |
---|---|
US (1) | US20220136009A1 (es) |
EP (1) | EP3976798A4 (es) |
JP (1) | JP7432621B2 (es) |
KR (1) | KR20220066225A (es) |
CN (1) | CN114174520A (es) |
AU (2) | AU2020282352B2 (es) |
BR (1) | BR112021024055A2 (es) |
CA (1) | CA3141900C (es) |
CL (1) | CL2021003151A1 (es) |
CO (1) | CO2021017544A2 (es) |
IL (1) | IL288313A (es) |
MX (1) | MX2021014460A (es) |
SG (1) | SG11202113048SA (es) |
TW (1) | TW202113084A (es) |
WO (1) | WO2020243651A1 (es) |
Families Citing this family (6)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AU2019346447A1 (en) | 2018-09-26 | 2021-04-29 | California Institute Of Technology | Adeno-associated virus compositions for targeted gene therapy |
EP3952924A4 (en) * | 2019-04-12 | 2023-05-24 | Encoded Therapeutics, Inc. | COMPOSITIONS AND METHODS OF ADMINISTRATION OF THERAPEUTIC PRODUCTS |
CN117980484A (zh) * | 2021-09-16 | 2024-05-03 | 诺华股份有限公司 | 新颖的转录因子 |
EP4151736A1 (en) * | 2021-09-21 | 2023-03-22 | SVAR Life Science AB | Novel system for the quantification of anti-aav neutralizing antibodies |
WO2023129940A1 (en) * | 2021-12-30 | 2023-07-06 | Regel Therapeutics, Inc. | Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof |
CN114507693A (zh) * | 2022-02-09 | 2022-05-17 | 中国人民解放军陆军军医大学第一附属医院 | 重组腺相关病毒表达载体及其应用 |
Family Cites Families (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2046806A4 (en) * | 2006-07-05 | 2009-10-21 | Scripps Research Inst | CHIMERIC RECOMBINASES WITH ZINC FINGERS OPTIMIZED FOR CATALYSIS BY DIRECT EVOLUTION |
US9526784B2 (en) * | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
WO2017180915A2 (en) * | 2016-04-13 | 2017-10-19 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
CN109890962A (zh) * | 2016-09-07 | 2019-06-14 | 旗舰创业股份有限公司 | 用于调节基因表达的方法和组合物 |
JP2018088888A (ja) * | 2016-12-06 | 2018-06-14 | 国立研究開発法人理化学研究所 | Scn1a遺伝子の発現増強法 |
SG11201909203WA (en) * | 2017-04-03 | 2019-11-28 | Encoded Therapeutics Inc | Tissue selective transgene expression |
JP7401432B2 (ja) * | 2017-12-01 | 2023-12-19 | エンコーデッド セラピューティクス, インコーポレイテッド | 操作されたdna結合タンパク質 |
WO2019224864A1 (ja) * | 2018-05-21 | 2019-11-28 | 国立研究開発法人理化学研究所 | Scn1a遺伝子の発現増強法とそれによるドラベ症候群の治療法 |
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2020
- 2020-05-29 BR BR112021024055A patent/BR112021024055A2/pt unknown
- 2020-05-29 WO PCT/US2020/035431 patent/WO2020243651A1/en active Application Filing
- 2020-05-29 TW TW109118224A patent/TW202113084A/zh unknown
- 2020-05-29 SG SG11202113048SA patent/SG11202113048SA/en unknown
- 2020-05-29 AU AU2020282352A patent/AU2020282352B2/en active Active
- 2020-05-29 KR KR1020217043048A patent/KR20220066225A/ko not_active Application Discontinuation
- 2020-05-29 CN CN202080054248.XA patent/CN114174520A/zh active Pending
- 2020-05-29 JP JP2021570376A patent/JP7432621B2/ja active Active
- 2020-05-29 MX MX2021014460A patent/MX2021014460A/es unknown
- 2020-05-29 EP EP20815541.6A patent/EP3976798A4/en active Pending
- 2020-05-29 CA CA3141900A patent/CA3141900C/en active Active
-
2021
- 2021-11-10 US US17/523,627 patent/US20220136009A1/en active Pending
- 2021-11-22 IL IL288313A patent/IL288313A/en unknown
- 2021-11-26 CL CL2021003151A patent/CL2021003151A1/es unknown
- 2021-12-21 CO CONC2021/0017544A patent/CO2021017544A2/es unknown
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2023
- 2023-07-10 AU AU2023204555A patent/AU2023204555A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
AU2020282352B2 (en) | 2023-05-18 |
IL288313A (en) | 2022-01-01 |
US20220136009A1 (en) | 2022-05-05 |
MX2021014460A (es) | 2022-02-11 |
AU2023204555A1 (en) | 2023-08-03 |
CA3141900A1 (en) | 2020-12-03 |
CN114174520A (zh) | 2022-03-11 |
AU2020282352A1 (en) | 2021-12-23 |
EP3976798A1 (en) | 2022-04-06 |
BR112021024055A2 (pt) | 2022-02-08 |
SG11202113048SA (en) | 2021-12-30 |
CL2021003151A1 (es) | 2022-07-29 |
JP2022535749A (ja) | 2022-08-10 |
JP7432621B2 (ja) | 2024-02-16 |
CA3141900C (en) | 2023-06-06 |
EP3976798A4 (en) | 2023-07-12 |
TW202113084A (zh) | 2021-04-01 |
WO2020243651A1 (en) | 2020-12-03 |
KR20220066225A (ko) | 2022-05-24 |
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