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 intermitente

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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
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Colombia
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nucleic acid
acid molecule
hmbs gene
treatment
acid strand
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CONC2021/0015542A
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English (en)
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Longcheng Li
Moorim Kang
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Ractigen Therapeutics
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Publication of CO2021015542A2 publication Critical patent/CO2021015542A2/es

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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.
CONC2021/0015542A 2019-04-30 2021-11-18 Molécula oligomérica de ácido nucleico y uso de esta en el tratamiento de la porfiria aguda intermitente CO2021015542A2 (es)

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

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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)

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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

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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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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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