CO2021015542A2 - Molécula oligomérica de ácido nucleico y uso de esta en el tratamiento de la porfiria aguda intermitente - Google Patents
Molécula oligomérica de ácido nucleico y uso de esta en el tratamiento de la porfiria aguda intermitenteInfo
- Publication number
- CO2021015542A2 CO2021015542A2 CONC2021/0015542A CO2021015542A CO2021015542A2 CO 2021015542 A2 CO2021015542 A2 CO 2021015542A2 CO 2021015542 A CO2021015542 A CO 2021015542A CO 2021015542 A2 CO2021015542 A2 CO 2021015542A2
- Authority
- CO
- Colombia
- Prior art keywords
- nucleic acid
- acid molecule
- hmbs gene
- treatment
- acid strand
- Prior art date
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- 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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- A61K31/00—Medicinal preparations containing organic active ingredients
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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- A61P3/00—Drugs for disorders of the metabolism
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- 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
- C12N15/1137—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 against enzymes
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- C12Y205/01—Transferases transferring alkyl or aryl groups, other than methyl groups (2.5) transferring alkyl or aryl groups, other than methyl groups (2.5.1)
- C12Y205/01061—Hydroxymethylbilane synthase (2.5.1.61)
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- Bioinformatics & Cheminformatics (AREA)
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Abstract
La presente invención se relaciona con composiciones de moléculas de ácido nucleico activador pequeño para aumentar la expresión del gen HMBS y un uso de estas. La molécula de ácido nucleico activador pequeño puede ser una molécula bicatenaria o monocatenaria de ARN dirigida a la región promotora del gen HMBS. La primera cadena de ácido nucleico y la segunda cadena de ácido nucleico contienen una región complementaria cada una, y las regiones complementarias pueden formar una estructura bicatenaria de ácido nucleico, que puede promover la expresión del gen HMBS. La primera cadena de ácido nucleico o la segunda cadena de ácido nucleico tienen independientemente una longitud de 16 a 35 nucleótidos. El extremo 3' de las dos cadenas oligonucleotídicas puede tener una prolongación de 0 a 6 nucleótidos de longitud. La molécula de ácido nucleico activador pequeño del gen HMBS se puede usar para aumentar las expresiones del ARNm y de la proteína del gen HMBS en una célula y promover la actividad enzimática de esta.
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
CN201910364093.9A CN111849968A (zh) | 2019-04-30 | 2019-04-30 | 寡核苷酸分子及其在急性间歇性卟啉症治疗中的应用 |
PCT/CN2020/087844 WO2020221309A1 (zh) | 2019-04-30 | 2020-04-29 | 寡聚核酸分子及其在急性间歇性卟啉症治疗中的应用 |
Publications (1)
Publication Number | Publication Date |
---|---|
CO2021015542A2 true CO2021015542A2 (es) | 2021-12-10 |
Family
ID=72965191
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CONC2021/0015542A CO2021015542A2 (es) | 2019-04-30 | 2021-11-18 | Molécula oligomérica de ácido nucleico y uso de esta en el tratamiento de la porfiria aguda intermitente |
Country Status (12)
Country | Link |
---|---|
US (1) | US20220211739A1 (es) |
EP (1) | EP3964582A4 (es) |
JP (1) | JP2022530937A (es) |
KR (1) | KR20220002896A (es) |
CN (2) | CN111849968A (es) |
AU (1) | AU2020265062A1 (es) |
BR (1) | BR112021021614A2 (es) |
CA (1) | CA3129976A1 (es) |
CO (1) | CO2021015542A2 (es) |
IL (1) | IL287615A (es) |
MX (1) | MX2021013361A (es) |
WO (1) | WO2020221309A1 (es) |
Families Citing this family (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2022166815A1 (zh) * | 2021-02-07 | 2022-08-11 | 中美瑞康核酸技术(南通)研究院有限公司 | 用于治疗增殖性玻璃体视网膜病变的双链核酸分子及其应用 |
Family Cites Families (11)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CN101210267A (zh) * | 2006-12-31 | 2008-07-02 | 国营武昌造船厂 | 编码多小片段rna载体在基因干扰与基因激活中的应用 |
US9045751B2 (en) * | 2010-04-28 | 2015-06-02 | The Regents Of The University Of California | Modified small activating RNA molecules and methods of use |
WO2012065143A1 (en) * | 2010-11-12 | 2012-05-18 | The General Hospital Corporation | Polycomb-associated non-coding rnas |
CN102657878B (zh) * | 2012-05-01 | 2014-04-16 | 浙江大学 | Ints6基因小激活rna在制备抗***癌药物中的应用 |
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 |
CA3005090A1 (en) * | 2015-12-14 | 2017-06-22 | Cold Spring Harbor Laboratory | Compositions and methods for treatment of liver diseases |
CN106244588B (zh) * | 2016-07-30 | 2019-07-12 | 内蒙古医科大学附属人民医院 | 激活肺癌细胞中RUNX3表达的saRNA及其应用 |
CN106480028B (zh) * | 2016-10-12 | 2019-04-05 | 上海市第七人民医院 | TPO基因的saRNA分子及其应用 |
CN106929508B (zh) * | 2017-02-17 | 2020-08-21 | 张灏 | 一种激活PTPRO基因表达的saRNA及其转运载体 |
CN108103063B (zh) * | 2017-12-06 | 2021-08-13 | 张灏 | 一种saRNA及其用途 |
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2019
- 2019-04-30 CN CN201910364093.9A patent/CN111849968A/zh active Pending
-
2020
- 2020-04-29 EP EP20798178.8A patent/EP3964582A4/en active Pending
- 2020-04-29 KR KR1020217033428A patent/KR20220002896A/ko unknown
- 2020-04-29 JP JP2021555183A patent/JP2022530937A/ja active Pending
- 2020-04-29 CN CN202080006273.0A patent/CN113260702B/zh active Active
- 2020-04-29 BR BR112021021614A patent/BR112021021614A2/pt unknown
- 2020-04-29 WO PCT/CN2020/087844 patent/WO2020221309A1/zh unknown
- 2020-04-29 CA CA3129976A patent/CA3129976A1/en active Pending
- 2020-04-29 MX MX2021013361A patent/MX2021013361A/es unknown
- 2020-04-29 US US17/594,740 patent/US20220211739A1/en active Pending
- 2020-04-29 AU AU2020265062A patent/AU2020265062A1/en not_active Abandoned
-
2021
- 2021-10-27 IL IL287615A patent/IL287615A/en unknown
- 2021-11-18 CO CONC2021/0015542A patent/CO2021015542A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP3964582A4 (en) | 2023-01-18 |
CN113260702B (zh) | 2022-02-22 |
AU2020265062A1 (en) | 2021-11-18 |
BR112021021614A2 (pt) | 2021-12-21 |
CA3129976A1 (en) | 2020-11-05 |
CN113260702A (zh) | 2021-08-13 |
IL287615A (en) | 2021-12-01 |
KR20220002896A (ko) | 2022-01-07 |
JP2022530937A (ja) | 2022-07-05 |
WO2020221309A1 (zh) | 2020-11-05 |
EP3964582A1 (en) | 2022-03-09 |
US20220211739A1 (en) | 2022-07-07 |
CN111849968A (zh) | 2020-10-30 |
MX2021013361A (es) | 2021-11-18 |
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