CN113748207A - 脆性x智力低下蛋白干扰寡核苷酸及其使用方法 - Google Patents

脆性x智力低下蛋白干扰寡核苷酸及其使用方法 Download PDF

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CN113748207A
CN113748207A CN202080031598.4A CN202080031598A CN113748207A CN 113748207 A CN113748207 A CN 113748207A CN 202080031598 A CN202080031598 A CN 202080031598A CN 113748207 A CN113748207 A CN 113748207A
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antisense oligonucleotide
fmrp
linkages
seq
antisense
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F·维蒂
S·贝林维亚
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Nogra Pharma Ltd
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Nogra Pharma Ltd
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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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    • 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
    • C12N15/1135—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 oncogenes or tumor suppressor genes
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    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine

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CN202080031598.4A 2019-02-26 2020-02-26 脆性x智力低下蛋白干扰寡核苷酸及其使用方法 Pending CN113748207A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962810697P 2019-02-26 2019-02-26
US62/810,697 2019-02-26
PCT/EP2020/055071 WO2020174023A1 (en) 2019-02-26 2020-02-26 Fragile x mental retardation protein interfering oligonucleotides and methods of using same

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CN113748207A true CN113748207A (zh) 2021-12-03

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US (1) US20220145303A1 (https=)
EP (1) EP3931324A1 (https=)
JP (1) JP2022521502A (https=)
KR (1) KR20210132678A (https=)
CN (1) CN113748207A (https=)
AU (1) AU2020228134A1 (https=)
BR (1) BR112021016907A2 (https=)
CA (1) CA3130854A1 (https=)
EA (1) EA202192330A1 (https=)
MX (1) MX2021010255A (https=)
SG (1) SG11202109112WA (https=)
WO (1) WO2020174023A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121109315A (zh) * 2025-08-28 2025-12-12 同济大学 脆性x智力障碍1号基因在治疗或缓解骨关节炎中的应用

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025501755A (ja) * 2021-12-23 2025-01-23 ユニバーシティー オブ マサチューセッツ 脆弱ｘ関連障害に対する治療的処置

Citations (4)

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Publication number Priority date Publication date Assignee Title
CN102449171A (zh) * 2009-03-24 2012-05-09 奥斯瑞根公司 对fmr1和fmr2基因5’非翻译区进行表征的pcr方法
US20120231015A1 (en) * 2009-11-06 2012-09-13 Emory University Fragile x mental retardation protein (fmrp), compositions, and methods related thereto
CN103981253A (zh) * 2014-03-27 2014-08-13 江苏佰龄全基因生物医学技术有限公司 一种用于检测脆性x染色体综合征的cgg重复数及agg插入信息的pcr试剂盒
US20180256749A1 (en) * 2015-09-17 2018-09-13 University Of Massachusetts Compositions and methods for modulating fmr1 expression

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RU2593939C2 (ru) 2008-11-13 2016-08-10 Ногра Фарма Лимитед Антисмысловые композиции и способы их получения и применения
EP2488665B1 (en) * 2009-10-16 2017-12-13 National University of Singapore Screening method for trinucleotide repeat sequences
AU2011273360A1 (en) * 2010-07-02 2013-02-07 Vib Vzw The role of Fragile X mental retardation gene and protein in cancer metastasis
EP3368089B1 (en) * 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
WO2019154884A1 (en) * 2018-02-07 2019-08-15 Ecole Polytechnique Federale De Lausanne (Epfl) Method for determining cancer invasiveness and patient prognosis
CN108531576A (zh) * 2018-04-12 2018-09-14 北京信诺佰世医学检验所有限公司 检测脆性x染色体综合征的试剂盒和系统

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102449171A (zh) * 2009-03-24 2012-05-09 奥斯瑞根公司 对fmr1和fmr2基因5’非翻译区进行表征的pcr方法
US20120231015A1 (en) * 2009-11-06 2012-09-13 Emory University Fragile x mental retardation protein (fmrp), compositions, and methods related thereto
CN103981253A (zh) * 2014-03-27 2014-08-13 江苏佰龄全基因生物医学技术有限公司 一种用于检测脆性x染色体综合征的cgg重复数及agg插入信息的pcr试剂盒
US20180256749A1 (en) * 2015-09-17 2018-09-13 University Of Massachusetts Compositions and methods for modulating fmr1 expression

Non-Patent Citations (4)

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Title
AHMAD M. KHALIL等: "A Novel RNA Transcript with Antiapoptotic Function Is Silenced in Fragile X Syndrome", PLOS ONE, vol. 3, no. 1, 23 January 2008 (2008-01-23), pages 1486 *
THOMAS MAURIN等: "Fragile X Syndrome: From molecular pathology to therapy", NEUROSCIENCE AND BIOBEHAVIORAL REVIEWS, vol. 46, 22 January 2014 (2014-01-22), pages 242 *
XIN JIN等: "FXR1 is elevated in colorectal cancer and acts as an oncogene", TUMOR BIOL., vol. 37, no. 2, 24 September 2015 (2015-09-24), XP036218869, DOI: 10.1007/s13277-015-4068-9 *
石玉衡;: "基因敲除模型小鼠在疾病研究中的应用", 中国组织工程研究与临床康复, no. 33, 13 August 2011 (2011-08-13), pages 6239 - 6242 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121109315A (zh) * 2025-08-28 2025-12-12 同济大学 脆性x智力障碍1号基因在治疗或缓解骨关节炎中的应用

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WO2020174023A1 (en) 2020-09-03
EA202192330A1 (ru) 2021-12-13
CA3130854A1 (en) 2020-09-03
EP3931324A1 (en) 2022-01-05
MX2021010255A (es) 2021-12-15
BR112021016907A2 (pt) 2021-11-03
US20220145303A1 (en) 2022-05-12
JP2022521502A (ja) 2022-04-08
SG11202109112WA (en) 2021-09-29
AU2020228134A1 (en) 2021-10-14
KR20210132678A (ko) 2021-11-04

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Application publication date: 20211203