JP2025512501A - シャルコー・マリー・トゥース病に対するポリヌクレオチド治療 - Google Patents

シャルコー・マリー・トゥース病に対するポリヌクレオチド治療 Download PDF

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JP2025512501A
JP2025512501A JP2024560756A JP2024560756A JP2025512501A JP 2025512501 A JP2025512501 A JP 2025512501A JP 2024560756 A JP2024560756 A JP 2024560756A JP 2024560756 A JP2024560756 A JP 2024560756A JP 2025512501 A JP2025512501 A JP 2025512501A
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exon
pmp22
seq
mrna
aso
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JP2025512501A5 (https=
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ディー． オコナー スティーヴン
ローソン クリスチャン
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Shift Pharmaceuticals Holding Inc
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Shift Pharmaceuticals Holding Inc
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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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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    • A61K31/00—Medicinal preparations containing organic active ingredients
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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
    • C12N15/1138—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 receptors or cell surface proteins
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • C12N2310/11—Antisense
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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
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/158—Expression markers

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  • Wood Science & Technology (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
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  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
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  • Endocrinology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2024560756A 2022-04-14 2023-04-13 シャルコー・マリー・トゥース病に対するポリヌクレオチド治療 Pending JP2025512501A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202263331045P 2022-04-14 2022-04-14
US202263331044P 2022-04-14 2022-04-14
US63/331,045 2022-04-14
US63/331,044 2022-04-14
PCT/US2023/065728 WO2023201300A2 (en) 2022-04-14 2023-04-13 Polynucleotide treatments for charcot-marie-tooth disease

Publications (2)

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JP2025512501A true JP2025512501A (ja) 2025-04-17
JP2025512501A5 JP2025512501A5 (https=) 2026-04-03

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JP2024560756A Pending JP2025512501A (ja) 2022-04-14 2023-04-13 シャルコー・マリー・トゥース病に対するポリヌクレオチド治療

Country Status (7)

Country Link
US (2) US20250333744A1 (https=)
EP (1) EP4508219A2 (https=)
JP (1) JP2025512501A (https=)
KR (1) KR20250005287A (https=)
AU (1) AU2023254106A1 (https=)
CA (1) CA3248302A1 (https=)
WO (2) WO2023201300A2 (https=)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2009538603A (ja) * 2006-06-02 2009-11-12 ヒューマン ジェネティック シグネチャーズ ピーティーワイ リミテッド 微生物の検出および分析に使用するための微生物改変核酸
US20150086982A1 (en) * 2011-08-19 2015-03-26 Roka Bioscience, Inc. Compositions and methods for detecting and discriminating between yeast or mold
EP3174981B1 (en) * 2014-07-31 2020-03-25 Association Institut de Myologie Treatment of amyotrophic lateral sclerosis
WO2017156242A1 (en) * 2016-03-09 2017-09-14 Ionis Pharmaceuticals, Inc. Methods and compositions for inhibiting pmp22 expression
WO2021113390A1 (en) * 2019-12-02 2021-06-10 Shape Therapeutics Inc. Compositions for treatment of diseases
AU2021244774A1 (en) * 2020-03-23 2022-09-29 Monash University Antisense oligomers for treatment of disease

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Publication number Publication date
US20250333744A1 (en) 2025-10-30
AU2023254106A1 (en) 2024-10-24
WO2023201300A2 (en) 2023-10-19
WO2023201300A3 (en) 2023-11-23
CA3248302A1 (en) 2023-10-19
WO2023201322A1 (en) 2023-10-19
EP4508219A2 (en) 2025-02-19
US20250327078A1 (en) 2025-10-23
KR20250005287A (ko) 2025-01-09

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