MX2021007932A - Molecula oligomerica de acido nucleico y uso de esta. - Google Patents
Molecula oligomerica de acido nucleico y uso de esta.Info
- Publication number
- MX2021007932A MX2021007932A MX2021007932A MX2021007932A MX2021007932A MX 2021007932 A MX2021007932 A MX 2021007932A MX 2021007932 A MX2021007932 A MX 2021007932A MX 2021007932 A MX2021007932 A MX 2021007932A MX 2021007932 A MX2021007932 A MX 2021007932A
- Authority
- MX
- Mexico
- Prior art keywords
- nucleic acid
- acid molecule
- small
- activating
- application
- Prior art date
Links
- 150000007523 nucleic acids Chemical class 0.000 title abstract 10
- 108020004707 nucleic acids Proteins 0.000 title abstract 8
- 102000039446 nucleic acids Human genes 0.000 title abstract 8
- 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
- 108090000623 proteins and genes Proteins 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
- 230000014509 gene expression Effects 0.000 abstract 1
- 208000002320 spinal muscular atrophy Diseases 0.000 abstract 1
- 230000009452 underexpressoin Effects 0.000 abstract 1
- 230000003827 upregulation Effects 0.000 abstract 1
Classifications
-
- 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
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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
-
- 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)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Physical Education & Sports Medicine (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Epidemiology (AREA)
- Environmental Sciences (AREA)
- Animal Husbandry (AREA)
- Biodiversity & Conservation Biology (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 invención se relaciona con una molécula de ácido nucleico activador pequeño para tratar la atrofia muscular espinal y el uso de esta; la molécula de ácido nucleico activador pequeño comprende una cadena de ácido nucleico sentido y una cadena de ácido nucleico antisentido, en donde la cadena de ácido nucleico sentido y la cadena de ácido nucleico antisentido son independientemente una cadena oligonucleotídica de 16 a 35 nucleótidos de longitud, en la que una cadena nucleotídica tiene al menos 75 % de homología o complementariedad de bases con respecto a una diana seleccionada de una región promotora de un gen diana SMN2; la presente invención también se relaciona con una composición farmacéutica que comprende la molécula de ácido nucleico activador pequeño descrita en la presente y, opcionalmente, un transportador farmacéuticamente aceptable, y un método para aumentar la expresión de un gen diana en células y métodos para tratar una enfermedad inducida por la expresión insuficiente de un gen diana con la molécula de ácido nucleico activador pequeño o la composición farmacéutica que comprende la molécula de ácido nucleico activador pequeño 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 |
|---|---|
| MX2021007932A true MX2021007932A (es) | 2021-08-16 |
Family
ID=71125998
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2021007932A MX2021007932A (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 |
|---|---|---|---|---|
| EP4189091A1 (en) * | 2020-07-31 | 2023-06-07 | 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 |
| KR20230154026A (ko) * | 2021-02-08 | 2023-11-07 | 락티젠 세러퓨틱스 | 다가 올리고뉴클레오티드 작용제 및 이의 사용 방법 |
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 |
| AU2013262663A1 (en) * | 2012-05-16 | 2015-01-22 | The General Hospital Corporation D/B/A Massachusetts General Hospital | Compositions and methods for modulating gene expression |
| WO2015051283A1 (en) * | 2013-10-04 | 2015-04-09 | Rana Therapeutics, Inc. | Compositions and methods for treating amyotrophic lateral sclerosis |
| 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 |
| WO2016170348A2 (en) * | 2015-04-22 | 2016-10-27 | Mina Therapeutics Limited | Sarna compositions and methods of use |
| EP3368671A1 (en) * | 2015-10-26 | 2018-09-05 | Translate Bio Ma, 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 |
| EP3471779A4 (en) * | 2016-06-16 | 2020-07-08 | Ionis Pharmaceuticals, Inc. | COMBINATIONS FOR MODULATING THE SMN EXPRESSION |
| CA3034385A1 (en) * | 2016-07-21 | 2018-01-25 | The General Hospital Corporation | Extracellular mrna markers of muscular dystrophies in human urine |
-
2019
- 2019-12-27 BR BR112021012422-5A patent/BR112021012422A2/pt unknown
- 2019-12-27 AU AU2019414608A patent/AU2019414608A1/en not_active Abandoned
- 2019-12-27 CA CA3120534A patent/CA3120534A1/en active Pending
- 2019-12-27 MX MX2021007932A patent/MX2021007932A/es unknown
- 2019-12-27 KR KR1020217020377A patent/KR20210110310A/ko unknown
- 2019-12-27 JP JP2021538368A patent/JP2022515881A/ja active Pending
- 2019-12-27 SG SG11202106327QA patent/SG11202106327QA/en unknown
- 2019-12-27 PE PE2021001108A patent/PE20212248A1/es unknown
- 2019-12-27 EP EP19906151.6A patent/EP3904517A4/en active Pending
- 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 US US17/419,569 patent/US20220064642A1/en active Pending
-
2021
- 2021-06-23 IL IL284333A patent/IL284333A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| PE20212248A1 (es) | 2021-11-24 |
| SG11202106327QA (en) | 2021-07-29 |
| CN112996913B (zh) | 2022-01-04 |
| US20220064642A1 (en) | 2022-03-03 |
| EP3904517A4 (en) | 2022-12-28 |
| CN112996913A (zh) | 2021-06-18 |
| EP3904517A1 (en) | 2021-11-03 |
| KR20210110310A (ko) | 2021-09-07 |
| IL284333A (en) | 2021-08-31 |
| JP2022515881A (ja) | 2022-02-22 |
| AU2019414608A1 (en) | 2021-07-22 |
| BR112021012422A2 (pt) | 2021-10-26 |
| WO2020135677A1 (zh) | 2020-07-02 |
| CA3120534A1 (en) | 2020-07-02 |
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| MX2021007932A (es) | Molecula oligomerica de acido nucleico y uso de esta. | |
| ES2558962T3 (es) | Polinucleótido bicatenario | |
| MX2023005326A (es) | Composiciones de oligonucleótidos y métodos de las mismas. | |
| WO2009142822A3 (en) | 2-f modified rna interference agents | |
| MX2021012126A (es) | Composiciones y métodos para inhibir la expresión génica en el sistema nervioso central. | |
| SG10201903290YA (en) | Modified double-stranded rna agents | |
| WO2005089287A3 (en) | Methods and compositions for the specific inhibition of gene expression by double-stranded rna | |
| PH12021551122A1 (en) | Method for treating muscular dystrophy by targeting utrophin gene | |
| BRPI0811170B8 (pt) | oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila | |
| ATE460481T1 (de) | Verfahren zur hemmung der expression eines zielgens und medikament zur therapie einer tumorerkrankung | |
| WO2006121464A3 (en) | Compositions for treating respiratory viral infections and their use | |
| WO2008116860A3 (en) | Dsrna compositions and methods for treating hpv infections | |
| IL166546A (en) | Double-stranded ribonucleic acid, preparations containing it, is used in drug preparation and in vitro method to inhibit the expression of a target gene | |
| DE60325016D1 (de) | Oligonukleotide mit alternierenden segmenten und deren verwendungen | |
| MX2018012695A (es) | Oligomeros antisentido y metodos de uso de los mismos para tratar enfermedades relacionadas con el gen alpha-glucosidasa acido. | |
| ATE528008T1 (de) | Rnai-modulation von aha und ihre therapeutische verwendung | |
| WO2010006973A3 (en) | Compositions and methods for inhibiting expression of tgf-beta receptor genes | |
| WO2008036638A3 (en) | Rnai modulation of scap and therapeutic uses thereof | |
| AR123420A1 (es) | Inhibidores de dux4 y métodos de uso de estos | |
| JP2007530431A5 (es) | ||
| MX2021014478A (es) | Metodo para tratar distrofia muscular por direccionamiento del gen dmpk. | |
| PT1175489E (pt) | Oligonucleotidos contendo uma sequencia antisense estabilizada por uma estrutura secundaria e composicoes farmaceuticas contendo as mesmas | |
| US11072795B2 (en) | Antisense oligonucleotides | |
| WO2007137220A3 (en) | Compositions and methods for inhibiting expression of ikk-b gene | |
| HK1159988A1 (en) | Compositions and methods for inhibiting expression of factor v leiden mutant gene |