KR20210132678A - 취약 x 정신 지체 단백질 간섭 올리고뉴클레오타이드 및 이를 사용하는 방법 - Google Patents

취약 x 정신 지체 단백질 간섭 올리고뉴클레오타이드 및 이를 사용하는 방법 Download PDF

Info

Publication number
KR20210132678A
KR20210132678A KR1020217030136A KR20217030136A KR20210132678A KR 20210132678 A KR20210132678 A KR 20210132678A KR 1020217030136 A KR1020217030136 A KR 1020217030136A KR 20217030136 A KR20217030136 A KR 20217030136A KR 20210132678 A KR20210132678 A KR 20210132678A
Authority
KR
South Korea
Prior art keywords
linkage
antisense oligonucleotide
fmrp
seq
antisense
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
Application number
KR1020217030136A
Other languages
English (en)
Korean (ko)
Inventor
프란세스카 비티
살바토레 벨린비아
Original Assignee
노그라 파마 리미티드
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 노그라 파마 리미티드 filed Critical 노그라 파마 리미티드
Publication of KR20210132678A publication Critical patent/KR20210132678A/ko
Abandoned legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1135Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • C12N2310/3125Methylphosphonates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
KR1020217030136A 2019-02-26 2020-02-26 취약 x 정신 지체 단백질 간섭 올리고뉴클레오타이드 및 이를 사용하는 방법 Abandoned KR20210132678A (ko)

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

Publications (1)

Publication Number Publication Date
KR20210132678A true KR20210132678A (ko) 2021-11-04

Family

ID=69723929

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217030136A Abandoned KR20210132678A (ko) 2019-02-26 2020-02-26 취약 x 정신 지체 단백질 간섭 올리고뉴클레오타이드 및 이를 사용하는 방법

Country Status (12)

Country Link
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=)

Families Citing this family (2)

* 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
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

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2593939C2 (ru) 2008-11-13 2016-08-10 Ногра Фарма Лимитед Антисмысловые композиции и способы их получения и применения
US8679757B2 (en) * 2009-03-24 2014-03-25 Asuragen, Inc. PCR methods for characterizing the 5′ untranslated region of the FMR1 and FMR2 genes
EP2488665B1 (en) * 2009-10-16 2017-12-13 National University of Singapore Screening method for trinucleotide repeat sequences
US20120231015A1 (en) * 2009-11-06 2012-09-13 Emory University Fragile x mental retardation protein (fmrp), compositions, and methods related thereto
AU2011273360A1 (en) * 2010-07-02 2013-02-07 Vib Vzw The role of Fragile X mental retardation gene and protein in cancer metastasis
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
WO2017049192A1 (en) * 2015-09-17 2017-03-23 University Of Massachusetts Compositions and methods for modulating fmr1 expression
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

Also Published As

Publication number Publication date
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
CN113748207A (zh) 2021-12-03
US20220145303A1 (en) 2022-05-12
JP2022521502A (ja) 2022-04-08
SG11202109112WA (en) 2021-09-29
AU2020228134A1 (en) 2021-10-14

Similar Documents

Publication Publication Date Title
CN109312341B (zh) 微小rna及其使用方法
US11788145B2 (en) Epigeneiic silencing of NMT2
KR102232623B1 (ko) 결장직장암의 치료 방법
KR20220024153A (ko) 안지오포이에틴 유사 7(angptl7) 관련 질환의 치료
KR20150003846A (ko) 마이크로rna 화합물 및 mir-21 활성 조절 방법
WO2016201450A2 (en) Cancer treatment and diagnosis
US10006029B2 (en) Methods of treating colorectal cancer
KR20210132678A (ko) 취약 x 정신 지체 단백질 간섭 올리고뉴클레오타이드 및 이를 사용하는 방법
CN110998319A (zh) 用表观遗传疗法诱导合成致死性
WO2019023263A1 (en) METHODS AND COMPOSITIONS FOR TREATING TUMORS COMPRISING A BCR-ABL1 FUSION GENE
EP2101747B1 (en) Cancer chemoprevention strategy based on loss of imprinting of igf2
CN114729363A (zh) Il-34反义剂及其使用方法
JP2009516710A (ja) eIF4E−BP2の発現のモジュレート
CN112513296A (zh) 基于tp53突变状态和超突变状态的癌症治疗方法
WO2020032160A1 (ja) 炎症性腸疾患治療薬およびそのスクリーニング方法
EA045399B1 (ru) Олигонуклеотиды, препятствующие экспрессии белка, ассоциированного с синдромом ломкой х-хромосомы, и способы их применения
KR102917306B1 (ko) 비알코올 지방간염 환자에서 miR-4449 억제제를 유효성분으로 포함하는 간 섬유화의 예방 또는 치료용 약학적 조성물
KR20240009973A (ko) Il-34 안티센스 작용제 및 이의 사용 방법
JP2009501024A (ja) 癌の診断及び治療における遺伝的及びエピジェネティックな変化
HK1219489B (en) Methods of treating colorectal cancer
CN112739382A (zh) 使用了hsp47的抑制物质的化疗剂敏感性的增强
HK40031713A (en) Companion diagnostic for htra1 rna antagonists

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20210917

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1902 Submission of document of abandonment before decision of registration