PE20212248A1 - Molecula oligomerica de acido nucleico y uso de esta - Google Patents
Molecula oligomerica de acido nucleico y uso de estaInfo
- Publication number
- PE20212248A1 PE20212248A1 PE2021001108A PE2021001108A PE20212248A1 PE 20212248 A1 PE20212248 A1 PE 20212248A1 PE 2021001108 A PE2021001108 A PE 2021001108A PE 2021001108 A PE2021001108 A PE 2021001108A PE 20212248 A1 PE20212248 A1 PE 20212248A1
- Authority
- PE
- Peru
- Prior art keywords
- nucleic acid
- acid molecule
- small
- target gene
- promoter
- Prior art date
Links
- 108020004707 nucleic acids Proteins 0.000 title abstract 10
- 150000007523 nucleic acids Chemical class 0.000 title abstract 10
- 102000039446 nucleic acids Human genes 0.000 title abstract 10
- 239000012190 activator Substances 0.000 abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 230000000692 anti-sense effect Effects 0.000 abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 239000008194 pharmaceutical composition Substances 0.000 abstract 2
- 108700039691 Genetic Promoter Regions Proteins 0.000 abstract 1
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 abstract 1
- 108091034117 Oligonucleotide Proteins 0.000 abstract 1
- 102100021947 Survival motor neuron protein Human genes 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 239000003937 drug carrier Substances 0.000 abstract 1
- 208000002320 spinal muscular atrophy Diseases 0.000 abstract 1
Classifications
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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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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
- A01K67/027—New or modified breeds of vertebrates
- A01K67/0275—Genetically modified vertebrates, e.g. transgenic
-
- 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
-
- 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
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
- A01K2217/07—Animals genetically altered by homologous recombination
- A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
- A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
-
- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/0306—Animal model for genetic diseases
- A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
-
- 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
- C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
- C12N2310/122—Hairpin
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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
- C12N2320/10—Applications; Uses in screening processes
- C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Epidemiology (AREA)
- Environmental Sciences (AREA)
- Biodiversity & Conservation Biology (AREA)
- Animal Husbandry (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
Abstract
La presente invencion se relaciona con una molecula de acido nucleico activador pequeno para tratar la atrofia muscular espinal y el uso de esta. La molecula de acido nucleico activador pequeno comprende una cadena de acido nucleico sentido y una cadena de acido nucleico antisentido, en donde la cadena de acido nucleico sentido y la cadena de acido nucleico antisentido son independientemente una cadena oligonucleotidica de 16 a 35 nucleotidos de longitud, en la que una cadena nucleotidica tiene al menos 75 % de homologia o complementariedad de bases con respecto a una diana seleccionada de una region promotora de un gen diana SMN2. La presente invencion tambien se relaciona con una composicion farmaceutica que comprende la molecula de acido nucleico activador pequeno descrita en la presente y, opcionalmente, un transportador farmaceuticamente aceptable, y un metodo para aumentar la expresion de un gen diana en celulas y metodos para tratar una enfermedad inducida por la expresion insuficiente de un gen diana con la molecula de acido nucleico activador pequeno o la composicion farmaceutica que comprende la molecula de acido nucleico activador pequeno descrita en la presente.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN201811634268 | 2018-12-29 | ||
PCT/CN2019/129025 WO2020135677A1 (zh) | 2018-12-29 | 2019-12-27 | 寡聚核酸分子及其应用 |
Publications (1)
Publication Number | Publication Date |
---|---|
PE20212248A1 true PE20212248A1 (es) | 2021-11-24 |
Family
ID=71125998
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PE2021001108A PE20212248A1 (es) | 2018-12-29 | 2019-12-27 | Molecula oligomerica de acido nucleico y uso de esta |
Country Status (13)
Country | Link |
---|---|
US (1) | US20220064642A1 (es) |
EP (1) | EP3904517A4 (es) |
JP (1) | JP2022515881A (es) |
KR (1) | KR20210110310A (es) |
CN (1) | CN112996913B (es) |
AU (1) | AU2019414608A1 (es) |
BR (1) | BR112021012422A2 (es) |
CA (1) | CA3120534A1 (es) |
IL (1) | IL284333A (es) |
MX (1) | MX2021007932A (es) |
PE (1) | PE20212248A1 (es) |
SG (1) | SG11202106327QA (es) |
WO (1) | WO2020135677A1 (es) |
Families Citing this family (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20230287416A1 (en) * | 2020-07-31 | 2023-09-14 | Ractigen Therapeutics | Combinatory treatment of sma with sarna and mrna modulators |
CN112779252B (zh) * | 2020-12-31 | 2023-05-26 | 首都儿科研究所 | 靶向SMN2启动子区MeCP2结合的关键甲基化区域的反义寡核苷酸 |
CN117377764A (zh) * | 2021-02-08 | 2024-01-09 | 中美瑞康核酸技术(南通)研究院有限公司 | 多价寡核苷酸试剂及其使用方法 |
Family Cites Families (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2006005042A2 (en) * | 2004-06-30 | 2006-01-12 | Cemines, Inc. | Methods and compositions for the diagnosis,prognosis,and treatment of cancer |
US7838657B2 (en) * | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
CN1904900A (zh) * | 2005-07-28 | 2007-01-31 | 中国科学院生物物理研究所 | 人类的内源性siRNA序列及其应用和筛选方法 |
US20080187512A1 (en) * | 2007-02-07 | 2008-08-07 | Academia Sinica | Treatment for spinal muscular atrophy |
JP2015518714A (ja) * | 2012-05-16 | 2015-07-06 | ラナ セラピューティクス インコーポレイテッド | 遺伝子発現を調節するための組成物及び方法 |
JP2016534035A (ja) * | 2013-10-04 | 2016-11-04 | ラナ セラピューティクス インコーポレイテッド | 筋萎縮性側索硬化症を治療するための組成物及び方法 |
CN104630219B (zh) * | 2013-11-15 | 2019-05-07 | 中美瑞康核酸技术(南通)研究院有限公司 | 抑癌基因IRX1的saRNA分子及其组合物和其应用 |
CN106032532A (zh) * | 2015-03-17 | 2016-10-19 | 中国医学科学院北京协和医院 | 一种小激活rna及其制备方法和应用 |
EP3286318A2 (en) * | 2015-04-22 | 2018-02-28 | Mina Therapeutics Limited | Sarna compositions and methods of use |
WO2017075030A1 (en) * | 2015-10-26 | 2017-05-04 | Rana Therapeutics, Inc. | Methods and compositions for increasing smn expression |
WO2017087486A1 (en) * | 2015-11-16 | 2017-05-26 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using survival motor neuron (smn) protein |
US9669109B1 (en) * | 2015-11-24 | 2017-06-06 | Universita Di Ferrara | Modified human U1snRNA molecule, a gene encoding for the modified human U1snRNA molecule, an expression vector including the gene, and the use thereof in gene therapy of familial dysautonomia and spinal muscular atrophy |
WO2017218884A1 (en) * | 2016-06-16 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of smn expression |
WO2018017991A1 (en) * | 2016-07-21 | 2018-01-25 | The General Hospital Corporation | Extracellular mrna markers of muscular dystrophies in human urine |
-
2019
- 2019-12-27 US US17/419,569 patent/US20220064642A1/en active Pending
- 2019-12-27 CA CA3120534A patent/CA3120534A1/en active Pending
- 2019-12-27 EP EP19906151.6A patent/EP3904517A4/en active Pending
- 2019-12-27 PE PE2021001108A patent/PE20212248A1/es unknown
- 2019-12-27 KR KR1020217020377A patent/KR20210110310A/ko unknown
- 2019-12-27 CN CN201980076095.6A patent/CN112996913B/zh active Active
- 2019-12-27 WO PCT/CN2019/129025 patent/WO2020135677A1/zh active Application Filing
- 2019-12-27 AU AU2019414608A patent/AU2019414608A1/en not_active Abandoned
- 2019-12-27 BR BR112021012422-5A patent/BR112021012422A2/pt unknown
- 2019-12-27 MX MX2021007932A patent/MX2021007932A/es unknown
- 2019-12-27 SG SG11202106327QA patent/SG11202106327QA/en unknown
- 2019-12-27 JP JP2021538368A patent/JP2022515881A/ja active Pending
-
2021
- 2021-06-23 IL IL284333A patent/IL284333A/en unknown
Also Published As
Publication number | Publication date |
---|---|
CN112996913B (zh) | 2022-01-04 |
US20220064642A1 (en) | 2022-03-03 |
EP3904517A1 (en) | 2021-11-03 |
BR112021012422A2 (pt) | 2021-10-26 |
JP2022515881A (ja) | 2022-02-22 |
IL284333A (en) | 2021-08-31 |
WO2020135677A1 (zh) | 2020-07-02 |
SG11202106327QA (en) | 2021-07-29 |
KR20210110310A (ko) | 2021-09-07 |
EP3904517A4 (en) | 2022-12-28 |
CA3120534A1 (en) | 2020-07-02 |
AU2019414608A1 (en) | 2021-07-22 |
MX2021007932A (es) | 2021-08-16 |
CN112996913A (zh) | 2021-06-18 |
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