MA58655B1 - Acide nucléique optimisé par codons qui code la protéine smn1 - Google Patents
Acide nucléique optimisé par codons qui code la protéine smn1Info
- Publication number
- MA58655B1 MA58655B1 MA58655A MA58655A MA58655B1 MA 58655 B1 MA58655 B1 MA 58655B1 MA 58655 A MA58655 A MA 58655A MA 58655 A MA58655 A MA 58655A MA 58655 B1 MA58655 B1 MA 58655B1
- Authority
- MA
- Morocco
- Prior art keywords
- encodes
- nucleic acid
- codon
- optimized nucleic
- smn1
- Prior art date
Links
- 102000039446 nucleic acids Human genes 0.000 title abstract 2
- 108020004707 nucleic acids Proteins 0.000 title abstract 2
- 150000007523 nucleic acids Chemical class 0.000 title abstract 2
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 title 1
- 102100021947 Survival motor neuron protein Human genes 0.000 title 1
- 101150081851 SMN1 gene Proteins 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 241000700605 Viruses Species 0.000 abstract 1
- 210000004027 cell Anatomy 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 210000002161 motor neuron Anatomy 0.000 abstract 1
- 230000032405 negative regulation of neuron apoptotic process Effects 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
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
- 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
- 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
- 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
-
- 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
Abstract
La présente invention se rapporte au domaine de la génétique, de la thérapie génique et de la biologie moléculaire. L'invention concerne plus précisément un acide nucléique optimisé par codons séparé qui code la protéine smn1 (protéine de survivance des neurones moteurs), une cassette d'expression et un vecteur à base de celle-ci, ainsi qu'un virus recombinant à base de aav9 (virus adéno-associé de sérotype 9) afin d'augmenter l'expression du gène smn1 dans des cellules cibles, ainsi que l'utilisation de celui-ci.
Applications Claiming Priority (2)
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 |
PCT/RU2021/000238 WO2021246909A1 (fr) | 2020-06-02 | 2021-06-02 | Acide nucléique optimisé par codons qui code la protéine smn1 |
Publications (2)
Publication Number | Publication Date |
---|---|
MA58655A1 MA58655A1 (fr) | 2023-06-28 |
MA58655B1 true MA58655B1 (fr) | 2023-12-29 |
Family
ID=74665909
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA58655A MA58655B1 (fr) | 2020-06-02 | 2021-06-02 | Acide nucléique optimisé par codons qui code la protéine smn1 |
Country Status (21)
Country | Link |
---|---|
US (1) | US20230212609A1 (fr) |
EP (1) | EP4159863A1 (fr) |
JP (1) | JP2023529855A (fr) |
KR (1) | KR20230019162A (fr) |
CN (1) | CN116234914A (fr) |
AR (1) | AR122500A1 (fr) |
AU (1) | AU2021282898A1 (fr) |
BR (1) | BR112022024708A2 (fr) |
CA (1) | CA3181288A1 (fr) |
CL (1) | CL2022003428A1 (fr) |
CO (1) | CO2022017334A2 (fr) |
CR (1) | CR20220619A (fr) |
EC (1) | ECSP22092206A (fr) |
IL (1) | IL298772A (fr) |
MA (1) | MA58655B1 (fr) |
MX (1) | MX2022015231A (fr) |
PE (1) | PE20240079A1 (fr) |
RU (1) | RU2742837C1 (fr) |
TW (1) | TW202214862A (fr) |
UY (1) | UY39245A (fr) |
WO (1) | WO2021246909A1 (fr) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN117210457A (zh) * | 2022-06-10 | 2023-12-12 | 拜奥卡德联合股份公司 | 具有启动子活性的核酸及其用途 |
WO2023246734A1 (fr) * | 2022-06-21 | 2023-12-28 | Skyline Therapeutics (Shanghai) Co., Ltd. | Vaa recombinant pour la thérapie génique de la maladie sma |
Family Cites Families (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
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 |
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 |
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 |
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 |
BR112018011975A2 (pt) * | 2015-12-14 | 2018-12-11 | Univ Pennsylvania | composições úteis no tratamento de atrofia muscular espinhal |
-
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 JP JP2022574544A patent/JP2023529855A/ja active Pending
- 2021-06-02 MA MA58655A patent/MA58655B1/fr unknown
- 2021-06-02 MX MX2022015231A patent/MX2022015231A/es unknown
- 2021-06-02 KR KR1020227046340A patent/KR20230019162A/ko unknown
- 2021-06-02 EP EP21817607.1A patent/EP4159863A1/fr active Pending
- 2021-06-02 BR BR112022024708A patent/BR112022024708A2/pt unknown
- 2021-06-02 PE PE2022002846A patent/PE20240079A1/es unknown
- 2021-06-02 WO PCT/RU2021/000238 patent/WO2021246909A1/fr active Application Filing
- 2021-06-02 IL IL298772A patent/IL298772A/en unknown
- 2021-06-02 CR CR20220619A patent/CR20220619A/es unknown
- 2021-06-02 CA CA3181288A patent/CA3181288A1/fr active Pending
- 2021-06-02 US US18/000,612 patent/US20230212609A1/en active Pending
- 2021-06-02 CN CN202180058177.5A patent/CN116234914A/zh active Pending
- 2021-06-02 AU AU2021282898A patent/AU2021282898A1/en active Pending
-
2022
- 2022-12-01 CO CONC2022/0017334A patent/CO2022017334A2/es unknown
- 2022-12-02 CL CL2022003428A patent/CL2022003428A1/es unknown
- 2022-12-02 EC ECSENADI202292206A patent/ECSP22092206A/es unknown
Also Published As
Publication number | Publication date |
---|---|
CL2022003428A1 (es) | 2023-06-09 |
MA58655A1 (fr) | 2023-06-28 |
PE20240079A1 (es) | 2024-01-16 |
US20230212609A1 (en) | 2023-07-06 |
MX2022015231A (es) | 2023-02-09 |
CN116234914A (zh) | 2023-06-06 |
CA3181288A1 (fr) | 2021-12-09 |
AU2021282898A1 (en) | 2023-02-02 |
AR122500A1 (es) | 2022-09-14 |
CR20220619A (es) | 2023-06-14 |
BR112022024708A2 (pt) | 2023-04-11 |
WO2021246909A1 (fr) | 2021-12-09 |
RU2742837C1 (ru) | 2021-02-11 |
UY39245A (es) | 2021-12-31 |
ECSP22092206A (es) | 2023-01-31 |
TW202214862A (zh) | 2022-04-16 |
JP2023529855A (ja) | 2023-07-12 |
IL298772A (en) | 2023-02-01 |
KR20230019162A (ko) | 2023-02-07 |
CO2022017334A2 (es) | 2023-04-17 |
EP4159863A1 (fr) | 2023-04-05 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
MA58655B1 (fr) | Acide nucléique optimisé par codons qui code la protéine smn1 | |
Acestor et al. | Trypanosoma brucei mitochondrial respiratome: composition and organization in procyclic form | |
Sun et al. | Ischaemic preconditioning preferentially increases protein S-nitrosylation in subsarcolemmal mitochondria | |
Dean et al. | A toolkit enabling efficient, scalable and reproducible gene tagging in trypanosomatids | |
Yao et al. | Localization of the small CAB-like proteins in photosystem II | |
Dröge | Oxidative stress and aging | |
Sun et al. | Heterogeneity within animal thioredoxin reductases: evidence for alternative first exon splicing | |
Benhar | Nitric oxide and the thioredoxin system: a complex interplay in redox regulation | |
Grossoehme et al. | Control of copper resistance and inorganic sulfur metabolism by paralogous regulators in Staphylococcus aureus | |
Vickers et al. | Trypanothione S-transferase activity in a trypanosomatid ribosomal elongation factor 1B | |
Safavi-Hemami et al. | Rapid expansion of the protein disulfide isomerase gene family facilitates the folding of venom peptides | |
Safavi-Hemami et al. | Identification of Conus peptidylprolyl cis-trans isomerases (PPIases) and assessment of their role in the oxidative folding of conotoxins | |
Ferro et al. | Cu, Zn superoxide dismutase genes in Tribolium castaneum: evolution, molecular characterisation, and gene expression during immune priming | |
Safavi-Hemami et al. | Modulation of conotoxin structure and function is achieved through a multienzyme complex in the venom glands of cone snails | |
Koch et al. | Unusual diversity of myoglobin genes in the lungfish | |
Nirujogi et al. | Moving from unsequenced to sequenced genome: reanalysis of the proteome of Leishmania donovani | |
MA43618A1 (fr) | Nouveaux peptides, association de peptides en tant que cibles et destinés à être utilisés en immunothérapie contre le cancer de la vésicule biliaire et le cholangiocarcinome et autres cancers | |
MA56142A1 (fr) | Protéine modifiée séparée vp1 de capside de aav5 | |
He et al. | Characterization and molecular cloning of a glutathione S-transferase gene from the tick, Boophilus microplus (Acari: Ixodidae) | |
Saraswathy et al. | Mitochondrial proteomics in experimental autoimmune uveitis oxidative stress | |
Drazic et al. | Role of cysteines in the stability and DNA-binding activity of the hypochlorite-specific transcription factor HypT | |
Manjunath et al. | Stop codon read-through of mammalian MTCH2 leading to an unstable isoform regulates mitochondrial membrane potential | |
MA62178A1 (fr) | Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale | |
Khoshnood et al. | A proteomics approach to identify targets of the ubiquitin-like molecule Urm1 in Drosophila melanogaster | |
Talbert et al. | Chromatin-based transcriptional punctuation |