UY39245A - Ácido nucleico codón-optimizado que codifica la proteína smn1 y su uso - Google Patents
Ácido nucleico codón-optimizado que codifica la proteína smn1 y su usoInfo
- Publication number
- UY39245A UY39245A UY0001039245A UY39245A UY39245A UY 39245 A UY39245 A UY 39245A UY 0001039245 A UY0001039245 A UY 0001039245A UY 39245 A UY39245 A UY 39245A UY 39245 A UY39245 A UY 39245A
- Authority
- UY
- Uruguay
- Prior art keywords
- smn1
- nucleic acid
- acid encoding
- codon
- optimized nucleic
- Prior art date
Links
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
- 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
-
- 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
- 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
-
- 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
- C07K14/4707—Muscular dystrophy
-
- 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
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
-
- 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
-
- 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
- C07K14/475—Growth factors; Growth regulators
-
- 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
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
-
- 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
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Biophysics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Gastroenterology & Hepatology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Toxicology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurosurgery (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Epidemiology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Abstract
La presente solicitud se refiere a los campos de la genética, la terapia génica y la biología molecular. Más específicamente, la presente invención se refiere a un ácido nucleico codón-optimizado aislado que codifica la proteína SMN1 (proteína de la neurona motora de supervivencia), un casete de expresión y un vector basado en el mismo, así como un virus recombinante basado en AAV9 (virus adenoasociado de serotipo 9) para aumentar la expresión del gen SMN1 en células diana, y su uso.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
RU2020118148A RU2742837C1 (ru) | 2020-06-02 | 2020-06-02 | Кодон-оптимизированная нуклеиновая кислота, которая кодирует белок SMN1, и ее применение |
Publications (1)
Publication Number | Publication Date |
---|---|
UY39245A true UY39245A (es) | 2021-12-31 |
Family
ID=74665909
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
UY0001039245A UY39245A (es) | 2020-06-02 | 2021-06-01 | Ácido nucleico codón-optimizado que codifica la proteína smn1 y su uso |
Country Status (22)
Country | Link |
---|---|
US (1) | US20230212609A1 (es) |
EP (1) | EP4159863A4 (es) |
JP (1) | JP2023529855A (es) |
KR (1) | KR20230019162A (es) |
CN (1) | CN116234914A (es) |
AR (1) | AR122500A1 (es) |
AU (1) | AU2021282898A1 (es) |
BR (1) | BR112022024708A2 (es) |
CA (1) | CA3181288A1 (es) |
CL (1) | CL2022003428A1 (es) |
CO (1) | CO2022017334A2 (es) |
CR (1) | CR20220619A (es) |
EC (1) | ECSP22092206A (es) |
IL (1) | IL298772A (es) |
MA (1) | MA58655B1 (es) |
MX (1) | MX2022015231A (es) |
PE (1) | PE20240079A1 (es) |
RU (1) | RU2742837C1 (es) |
TW (1) | TW202214862A (es) |
UY (1) | UY39245A (es) |
WO (1) | WO2021246909A1 (es) |
ZA (1) | ZA202213092B (es) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AR129552A1 (es) * | 2022-06-10 | 2024-09-04 | Joint Stock Company Biocad | Ácido nucleico con actividad promotora y uso del mismo |
WO2023246734A1 (en) * | 2022-06-21 | 2023-12-28 | Skyline Therapeutics (Shanghai) Co., Ltd. | Recombinant aav for the gene therapy of sma disease |
Family Cites Families (13)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US4634665A (en) | 1980-02-25 | 1987-01-06 | The Trustees Of Columbia University In The City Of New York | Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials |
US5179017A (en) | 1980-02-25 | 1993-01-12 | The Trustees Of Columbia University In The City Of New York | Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials |
US4399216A (en) | 1980-02-25 | 1983-08-16 | The Trustees Of Columbia University | Processes for inserting DNA into eucaryotic cells and for producing proteinaceous materials |
DE3431140A1 (de) | 1984-08-24 | 1986-03-06 | Behringwerke Ag, 3550 Marburg | Enhancer fuer eukaryotische expressionssysteme |
GB8717430D0 (en) | 1987-07-23 | 1987-08-26 | Celltech Ltd | Recombinant dna product |
RU2312896C2 (ru) * | 2001-09-20 | 2007-12-20 | Глаксо Груп Лимитед | Последовательность нуклеиновой кислоты, кодирующая белок gag вич-1, способ получения указанной последовательности, вектор, содержащий ее, белок, кодируемый ею, фармацевтическая композиция и их применение для профилактики и/или лечения вич-инфекции и спида |
WO2014178863A1 (en) * | 2013-05-01 | 2014-11-06 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
AU2015283954B2 (en) * | 2014-07-02 | 2020-11-12 | Translate Bio, Inc. | Encapsulation of messenger RNA |
MX2017005834A (es) * | 2014-11-05 | 2017-11-17 | Voyager Therapeutics Inc | Polinucleotidos aad para el tratamiento de la enfermedad de parkinson. |
KR20180086266A (ko) * | 2015-12-14 | 2018-07-30 | 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 | 척수성 근위축증의 치료에 유용한 아데노-관련 바이러스 벡터 |
WO2017191274A2 (en) * | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
JOP20190200A1 (ar) * | 2017-02-28 | 2019-08-27 | Univ Pennsylvania | تركيبات نافعة في معالجة ضمور العضل النخاعي |
US20200370069A1 (en) * | 2017-07-08 | 2020-11-26 | Genethon | Treatment of spinal muscular atrophy |
-
2020
- 2020-06-02 RU RU2020118148A patent/RU2742837C1/ru active
-
2021
- 2021-06-01 AR ARP210101493A patent/AR122500A1/es unknown
- 2021-06-01 TW TW110119867A patent/TW202214862A/zh unknown
- 2021-06-01 UY UY0001039245A patent/UY39245A/es unknown
- 2021-06-02 US US18/000,612 patent/US20230212609A1/en active Pending
- 2021-06-02 CR CR20220619A patent/CR20220619A/es unknown
- 2021-06-02 BR BR112022024708A patent/BR112022024708A2/pt unknown
- 2021-06-02 PE PE2022002846A patent/PE20240079A1/es unknown
- 2021-06-02 MA MA58655A patent/MA58655B1/fr unknown
- 2021-06-02 AU AU2021282898A patent/AU2021282898A1/en active Pending
- 2021-06-02 CN CN202180058177.5A patent/CN116234914A/zh active Pending
- 2021-06-02 EP EP21817607.1A patent/EP4159863A4/en active Pending
- 2021-06-02 MX MX2022015231A patent/MX2022015231A/es unknown
- 2021-06-02 JP JP2022574544A patent/JP2023529855A/ja active Pending
- 2021-06-02 KR KR1020227046340A patent/KR20230019162A/ko unknown
- 2021-06-02 CA CA3181288A patent/CA3181288A1/en active Pending
- 2021-06-02 WO PCT/RU2021/000238 patent/WO2021246909A1/ru active Application Filing
- 2021-06-02 IL IL298772A patent/IL298772A/en unknown
-
2022
- 2022-12-01 CO CONC2022/0017334A patent/CO2022017334A2/es unknown
- 2022-12-02 ZA ZA2022/13092A patent/ZA202213092B/en unknown
- 2022-12-02 EC ECSENADI202292206A patent/ECSP22092206A/es unknown
- 2022-12-02 CL CL2022003428A patent/CL2022003428A1/es unknown
Also Published As
Publication number | Publication date |
---|---|
KR20230019162A (ko) | 2023-02-07 |
EP4159863A1 (en) | 2023-04-05 |
CO2022017334A2 (es) | 2023-04-17 |
MA58655B1 (fr) | 2023-12-29 |
MA58655A1 (fr) | 2023-06-28 |
CA3181288A1 (en) | 2021-12-09 |
IL298772A (en) | 2023-02-01 |
CN116234914A (zh) | 2023-06-06 |
RU2742837C1 (ru) | 2021-02-11 |
PE20240079A1 (es) | 2024-01-16 |
BR112022024708A2 (pt) | 2023-04-11 |
ZA202213092B (en) | 2024-09-25 |
CR20220619A (es) | 2023-06-14 |
EP4159863A4 (en) | 2024-08-28 |
TW202214862A (zh) | 2022-04-16 |
WO2021246909A1 (ru) | 2021-12-09 |
MX2022015231A (es) | 2023-02-09 |
CL2022003428A1 (es) | 2023-06-09 |
US20230212609A1 (en) | 2023-07-06 |
ECSP22092206A (es) | 2023-01-31 |
JP2023529855A (ja) | 2023-07-12 |
AR122500A1 (es) | 2022-09-14 |
AU2021282898A1 (en) | 2023-02-02 |
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