BR112021021614A2 - Molécula de ácido nucleico oligomérico e uso da mesma no tratamento de porfiria aguda intermitente - Google Patents

Molécula de ácido nucleico oligomérico e uso da mesma no tratamento de porfiria aguda intermitente

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Publication number
BR112021021614A2
BR112021021614A2 BR112021021614A BR112021021614A BR112021021614A2 BR 112021021614 A2 BR112021021614 A2 BR 112021021614A2 BR 112021021614 A BR112021021614 A BR 112021021614A BR 112021021614 A BR112021021614 A BR 112021021614A BR 112021021614 A2 BR112021021614 A2 BR 112021021614A2
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Prior art keywords
nucleic acid
acid molecule
treatment
hmbs gene
acute intermittent
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BR112021021614A
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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 BR112021021614A2 publication Critical patent/BR112021021614A2/pt

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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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • 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
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    • 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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Abstract

molécula de ácido nucleico oligomérico e uso da mesma no tratamento de porfiria aguda intermitente. a presente invenção refere-se a composições de moléculas de pequeno ácido nucleico de ativação para aumentar a expressão do gene hmbs e a um uso das mesmas. a molécula de pequeno ácido nucleico de ativação pode ser uma molécula de rna de fita dupla ou de fita simples, direcionada à região promotora do gene hmbs. a primeira fita de ácido nucleico e a segunda fita de ácido nucleico contêm, cada uma, uma região complementar, e as regiões complementares podem formar uma estrutura de ácido nucleico de fita dupla, que pode promover a expressão do gene hmbs. a primeira fita de ácido nucleico ou a segunda fita de ácido nucleico têm, independentemente, um comprimento de 16 a 35 nucleotídeos. o terminal 3? das duas fitas de oligonucleotídeo pode ter uma saliência de 0 a 6 nucleotídeos de comprimento. a molécula de pequeno ácido nucleico de ativação para o gene hmbs pode ser usada para suprarregular as expressões de mrna e de proteína do gene hmbs em uma célula e promover a atividade enzimática do mesmo.
BR112021021614A 2019-04-30 2020-04-29 Molécula de ácido nucleico oligomérico e uso da mesma no tratamento de porfiria aguda intermitente BR112021021614A2 (pt)

Applications Claiming Priority (2)

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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 (pt)
EP (1) EP3964582A4 (pt)
JP (1) JP2022530937A (pt)
KR (1) KR20220002896A (pt)
CN (2) CN111849968A (pt)
AU (1) AU2020265062A1 (pt)
BR (1) BR112021021614A2 (pt)
CA (1) CA3129976A1 (pt)
CO (1) CO2021015542A2 (pt)
IL (1) IL287615A (pt)
MX (1) MX2021013361A (pt)
WO (1) WO2020221309A1 (pt)

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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
AU2011325956B2 (en) * 2010-11-12 2016-07-14 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在制备抗前列腺癌药物中的应用
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
US20180305689A1 (en) * 2015-04-22 2018-10-25 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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US20220211739A1 (en) 2022-07-07
MX2021013361A (es) 2021-11-18
CN113260702A (zh) 2021-08-13
KR20220002896A (ko) 2022-01-07
EP3964582A4 (en) 2023-01-18
JP2022530937A (ja) 2022-07-05
IL287615A (en) 2021-12-01
CN113260702B (zh) 2022-02-22
WO2020221309A1 (zh) 2020-11-05
CA3129976A1 (en) 2020-11-05
CO2021015542A2 (es) 2021-12-10
CN111849968A (zh) 2020-10-30
AU2020265062A1 (en) 2021-11-18
EP3964582A1 (en) 2022-03-09

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