AR124736A1 - EFECTO SINÉRGICO DE SMN1 Y miR-23a EN EL TRATAMIENTO DE LA ATROFIA MUSCULAR ESPINAL - Google Patents
EFECTO SINÉRGICO DE SMN1 Y miR-23a EN EL TRATAMIENTO DE LA ATROFIA MUSCULAR ESPINALInfo
- Publication number
- AR124736A1 AR124736A1 ARP220100193A ARP220100193A AR124736A1 AR 124736 A1 AR124736 A1 AR 124736A1 AR P220100193 A ARP220100193 A AR P220100193A AR P220100193 A ARP220100193 A AR P220100193A AR 124736 A1 AR124736 A1 AR 124736A1
- Authority
- AR
- Argentina
- Prior art keywords
- nucleic acid
- smn1
- recombinant virus
- aav9
- mir
- Prior art date
Links
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 title abstract 3
- 102100021947 Survival motor neuron protein Human genes 0.000 title abstract 3
- 108091035591 miR-23a stem-loop Proteins 0.000 title abstract 3
- 208000002320 spinal muscular atrophy Diseases 0.000 title 1
- 230000002195 synergetic effect Effects 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 abstract 10
- 102000039446 nucleic acids Human genes 0.000 abstract 10
- 150000007523 nucleic acids Chemical class 0.000 abstract 10
- 241000700605 Viruses Species 0.000 abstract 6
- 101150081851 SMN1 gene Proteins 0.000 abstract 3
- 210000004027 cell Anatomy 0.000 abstract 3
- 239000008194 pharmaceutical composition Substances 0.000 abstract 3
- 108700011259 MicroRNAs Proteins 0.000 abstract 2
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 2
- 238000001415 gene therapy Methods 0.000 abstract 2
- 239000002679 microRNA Substances 0.000 abstract 2
- 108090000623 proteins and genes Proteins 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 230000003612 virological effect Effects 0.000 abstract 2
- 241000702421 Dependoparvovirus Species 0.000 abstract 1
- 210000000234 capsid Anatomy 0.000 abstract 1
- 239000013604 expression vector Substances 0.000 abstract 1
- 239000000203 mixture Substances 0.000 abstract 1
- 210000002161 motor neuron Anatomy 0.000 abstract 1
- 230000032405 negative regulation of neuron apoptotic process Effects 0.000 abstract 1
- 239000000546 pharmaceutical excipient Substances 0.000 abstract 1
- 239000013598 vector 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
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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
- C12N15/63—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
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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
- 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)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Neurology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Toxicology (AREA)
- General Chemical & Material Sciences (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Abstract
La presente solicitud está relacionada con los campos de la biotecnología, la virología, la genética y la biología molecular. Más específicamente, la presente invención se refiere a un ácido nucleico aislado para producir un producto viral para terapia génica; dicho ácido nucleico aislado comprende un ácido nucleico que codifica la proteína SMN1 que tiene la secuencia de aminoácidos de SEQ ID Nº 1, y un ácido nucleico que codifica el microARN miR-23a, un casete de expresión y un vector basado en el mismo, así como un virus recombinante basado en AAV9 para expresar el gen SMN1 en células diana, una composición farmacéutica que incluye dicho virus recombinante, y diversos usos del virus recombinante anterior y de la composición anterior. Reivindicación 1: Un ácido nucleico aislado para producir un producto viral para terapia génica; dicho ácido nucleico aislado comprende un ácido nucleico que codifica la proteína SMN1 (proteína de supervivencia de motoneuronas) que tiene la secuencia de aminoácidos de SEQ ID Nº 1, y un ácido nucleico que codifica el microARN miR-23a. Reivindicación 5: Un casete de expresión que comprende el ácido nucleico según cualquiera de las reivindicaciones 1 a 4. Reivindicación 8: Un vector de expresión que comprende el ácido nucleico según una cualquiera de las reivindicaciones 1 a 4 o el casete según una cualquiera de las reivindicaciones 5 a 7. Reivindicación 9: Un virus recombinante basado en AAV9 (virus adeno-asociado de serotipo 9) para la expresión del gen SMN1 en células diana; dicho virus recombinante basado en AAV9 comprende una cápside y el casete de expresión según cualquiera de las reivindicaciones 5 a 7. Reivindicación 15: Una composición farmacéutica para entregar el gen SMN1 a las células diana; dicha composición farmacéutica comprende el virus recombinante basado en AAV9 según las reivindicaciones 9 a 14 en combinación con uno o más excipientes farmacéuticamente aceptables.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| RU2021102051A RU2021102051A (ru) | 2021-01-29 | Синергетическое действие SMN1 и miR-23a при лечении спинальной мышечной атрофии |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AR124736A1 true AR124736A1 (es) | 2023-04-26 |
Family
ID=82653732
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ARP220100193A AR124736A1 (es) | 2021-01-29 | 2022-01-31 | EFECTO SINÉRGICO DE SMN1 Y miR-23a EN EL TRATAMIENTO DE LA ATROFIA MUSCULAR ESPINAL |
Country Status (16)
| Country | Link |
|---|---|
| US (1) | US20240091383A1 (es) |
| CN (1) | CN117545842A (es) |
| AR (1) | AR124736A1 (es) |
| AU (1) | AU2022213262A1 (es) |
| BR (1) | BR112023015177A2 (es) |
| CA (1) | CA3206671A1 (es) |
| CL (1) | CL2023002218A1 (es) |
| CO (1) | CO2023009633A2 (es) |
| CR (1) | CR20230363A (es) |
| EC (1) | ECSP23056133A (es) |
| IL (1) | IL304612A (es) |
| MA (1) | MA62178A1 (es) |
| MX (1) | MX2023008825A (es) |
| TW (1) | TW202246501A (es) |
| UY (1) | UY39621A (es) |
| WO (1) | WO2022164351A1 (es) |
Families Citing this family (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023215384A2 (en) | 2022-05-04 | 2023-11-09 | Scholar Rock, Inc. | Use of myostatin inhibitor for treating spinal muscular atrophy |
| 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 |
|---|---|---|---|---|
| JP5879256B2 (ja) * | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | 神経変性障害のための遺伝子治療 |
| 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 AU AU2022213262A patent/AU2022213262A1/en active Pending
- 2022-01-28 CN CN202280025363.3A patent/CN117545842A/zh active Pending
- 2022-01-28 WO PCT/RU2022/000025 patent/WO2022164351A1/en active Application Filing
- 2022-01-28 MA MA62178A patent/MA62178A1/fr unknown
- 2022-01-28 TW TW111104139A patent/TW202246501A/zh unknown
- 2022-01-28 MX MX2023008825A patent/MX2023008825A/es unknown
- 2022-01-28 BR BR112023015177A patent/BR112023015177A2/pt unknown
- 2022-01-28 CR CR20230363A patent/CR20230363A/es unknown
- 2022-01-28 CA CA3206671A patent/CA3206671A1/en active Pending
- 2022-01-28 US US18/274,496 patent/US20240091383A1/en active Pending
- 2022-01-31 UY UY0001039621A patent/UY39621A/es unknown
- 2022-01-31 AR ARP220100193A patent/AR124736A1/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 |
|---|---|
| TW202246501A (zh) | 2022-12-01 |
| CO2023009633A2 (es) | 2023-12-20 |
| MX2023008825A (es) | 2023-08-10 |
| BR112023015177A2 (pt) | 2023-11-14 |
| MA62178A1 (fr) | 2023-12-29 |
| CR20230363A (es) | 2024-02-20 |
| ECSP23056133A (es) | 2023-08-31 |
| WO2022164351A1 (en) | 2022-08-04 |
| IL304612A (en) | 2023-09-01 |
| CN117545842A (zh) | 2024-02-09 |
| CL2023002218A1 (es) | 2024-02-02 |
| US20240091383A1 (en) | 2024-03-21 |
| UY39621A (es) | 2022-08-31 |
| AU2022213262A1 (en) | 2023-08-24 |
| CA3206671A1 (en) | 2022-08-04 |
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