MA62178A1 - Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale - Google Patents
Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinaleInfo
- Publication number
- MA62178A1 MA62178A1 MA62178A MA62178A MA62178A1 MA 62178 A1 MA62178 A1 MA 62178A1 MA 62178 A MA62178 A MA 62178A MA 62178 A MA62178 A MA 62178A MA 62178 A1 MA62178 A1 MA 62178A1
- Authority
- MA
- Morocco
- Prior art keywords
- nucleic acid
- smn1
- recombinant virus
- mir
- treatment
- Prior art date
Links
- 108091035591 miR-23a stem-loop Proteins 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
- 208000002320 spinal muscular atrophy Diseases 0.000 title 1
- 230000002195 synergetic effect Effects 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 abstract 4
- 102000039446 nucleic acids Human genes 0.000 abstract 4
- 150000007523 nucleic acids Chemical class 0.000 abstract 4
- 241000700605 Viruses Species 0.000 abstract 3
- 101150081851 SMN1 gene Proteins 0.000 abstract 2
- 108700005077 Viral Genes Proteins 0.000 abstract 1
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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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
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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
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
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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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
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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
- C12N2310/14—Type of nucleic acid interfering N.A.
- C12N2310/141—MicroRNAs, miRNAs
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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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
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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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/31—Combination therapy
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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
- 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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- 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
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- Biophysics (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physical Education & Sports Medicine (AREA)
- Gastroenterology & Hepatology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Toxicology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
La présente invention se rapporte aux domaines de la biotechnologie, de la virologie, de la génétique et de la biologie moléculaire. Plus particulièrement, la présente invention concerne un acide nucléique isolé servant à produire un produit viral de thérapie génique, ledit acide nucléique isolé comprenant un acide nucléique qui code la protéine smn1 ayant la séquence d'acides aminés de seq id no : 1, et un acide nucléique qui code le microarn mir-23a, une cassette d'expression et un vecteur basé sur cette dernière, ainsi qu'un virus recombinant à base d'aav9 pour exprimer le gène smn1 dans des cellules cibles, une composition pharmaceutique qui comprend ledit virus recombinant, et diverses utilisations du virus recombinant susmentionné et de la composition susmentionnée.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
RU2021102051A RU2021102051A (ru) | 2021-01-29 | Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии | |
PCT/RU2022/000025 WO2022164351A1 (fr) | 2021-01-29 | 2022-01-28 | Effet synergique <p> de smn1 et mir-23a dans le traitement de l'amyotrophie spinale </p> |
Publications (1)
Publication Number | Publication Date |
---|---|
MA62178A1 true MA62178A1 (fr) | 2023-12-29 |
Family
ID=82653732
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA62178A MA62178A1 (fr) | 2021-01-29 | 2022-01-28 | Effet synergique de smn1 et mir-23a dans le traitement de l'amyotrophie spinale |
Country Status (16)
Country | Link |
---|---|
US (1) | US20240091383A1 (fr) |
CN (1) | CN117545842A (fr) |
AR (1) | AR124736A1 (fr) |
AU (1) | AU2022213262A1 (fr) |
BR (1) | BR112023015177A2 (fr) |
CA (1) | CA3206671A1 (fr) |
CL (1) | CL2023002218A1 (fr) |
CO (1) | CO2023009633A2 (fr) |
CR (1) | CR20230363A (fr) |
EC (1) | ECSP23056133A (fr) |
IL (1) | IL304612A (fr) |
MA (1) | MA62178A1 (fr) |
MX (1) | MX2023008825A (fr) |
TW (1) | TW202246501A (fr) |
UY (1) | UY39621A (fr) |
WO (1) | WO2022164351A1 (fr) |
Families Citing this family (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2023215384A2 (fr) | 2022-05-04 | 2023-11-09 | Scholar Rock, Inc. | Utilisation d'un inhibiteur de la myostatine pour le traitement de l'amyotrophie spinale |
CN116693633B (zh) * | 2023-02-21 | 2023-12-22 | 广州派真生物技术有限公司 | 腺相关病毒突变体及其应用 |
Family Cites Families (2)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
MX356669B (es) * | 2009-05-02 | 2018-06-08 | Genzyme Corp | Terapia genica para trastornos neurodegenerativos. |
US11826433B2 (en) * | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
-
2022
- 2022-01-28 TW TW111104139A patent/TW202246501A/zh unknown
- 2022-01-28 CN CN202280025363.3A patent/CN117545842A/zh active Pending
- 2022-01-28 MX MX2023008825A patent/MX2023008825A/es unknown
- 2022-01-28 WO PCT/RU2022/000025 patent/WO2022164351A1/fr active Application Filing
- 2022-01-28 AU AU2022213262A patent/AU2022213262A1/en active Pending
- 2022-01-28 MA MA62178A patent/MA62178A1/fr unknown
- 2022-01-28 CR CR20230363A patent/CR20230363A/es unknown
- 2022-01-28 BR BR112023015177A patent/BR112023015177A2/pt unknown
- 2022-01-28 CA CA3206671A patent/CA3206671A1/fr active Pending
- 2022-01-28 US US18/274,496 patent/US20240091383A1/en active Pending
- 2022-01-31 AR ARP220100193A patent/AR124736A1/es unknown
- 2022-01-31 UY UY0001039621A patent/UY39621A/es unknown
-
2023
- 2023-07-19 CO CONC2023/0009633A patent/CO2023009633A2/es unknown
- 2023-07-20 IL IL304612A patent/IL304612A/en unknown
- 2023-07-24 EC ECSENADI202356133A patent/ECSP23056133A/es unknown
- 2023-07-27 CL CL2023002218A patent/CL2023002218A1/es unknown
Also Published As
Publication number | Publication date |
---|---|
MX2023008825A (es) | 2023-08-10 |
ECSP23056133A (es) | 2023-08-31 |
TW202246501A (zh) | 2022-12-01 |
US20240091383A1 (en) | 2024-03-21 |
AR124736A1 (es) | 2023-04-26 |
CO2023009633A2 (es) | 2023-12-20 |
CA3206671A1 (fr) | 2022-08-04 |
CR20230363A (es) | 2024-02-20 |
BR112023015177A2 (pt) | 2023-11-14 |
CN117545842A (zh) | 2024-02-09 |
AU2022213262A1 (en) | 2023-08-24 |
CL2023002218A1 (es) | 2024-02-02 |
WO2022164351A1 (fr) | 2022-08-04 |
IL304612A (en) | 2023-09-01 |
UY39621A (es) | 2022-08-31 |
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