CN116034161A - 用于恢复stmn2水平的方法和组合物 - Google Patents

用于恢复stmn2水平的方法和组合物 Download PDF

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Publication number
CN116034161A
CN116034161A CN202180040347.7A CN202180040347A CN116034161A CN 116034161 A CN116034161 A CN 116034161A CN 202180040347 A CN202180040347 A CN 202180040347A CN 116034161 A CN116034161 A CN 116034161A
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seq
stmn2
tdp
antisense oligonucleotide
site
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Chinese (zh)
Inventor
凯文·C·埃根
约瑟夫·罗伯特·克里姆
小罗伯特·H·布朗
乔纳森·K·瓦特斯
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Harvard University
University of Massachusetts Amherst
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Harvard University
University of Massachusetts Amherst
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Publication of CN116034161A publication Critical patent/CN116034161A/zh
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/316—Phosphonothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
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  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
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  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (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)
CN202180040347.7A 2020-03-25 2021-03-25 用于恢复stmn2水平的方法和组合物 Pending CN116034161A (zh)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202062994797P 2020-03-25 2020-03-25
US62/994,797 2020-03-25
US202063063174P 2020-08-07 2020-08-07
US63/063,174 2020-08-07
US202163133749P 2021-01-04 2021-01-04
US63/133,749 2021-01-04
PCT/US2021/024254 WO2021195446A2 (en) 2020-03-25 2021-03-25 Methods and compositions for restoring stmn2 levels

Publications (1)

Publication Number Publication Date
CN116034161A true CN116034161A (zh) 2023-04-28

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CN202180040347.7A Pending CN116034161A (zh) 2020-03-25 2021-03-25 用于恢复stmn2水平的方法和组合物

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US (1) US20240011027A1 (https=)
EP (1) EP4127172A4 (https=)
JP (2) JP2023519871A (https=)
CN (1) CN116034161A (https=)
CA (1) CA3176884A1 (https=)
WO (1) WO2021195446A2 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118737269A (zh) * 2024-08-30 2024-10-01 墨卓生物科技(浙江)有限公司 在单细胞微生物基因组测序结果中区分菌株的方法

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2020150290A2 (en) 2019-01-14 2020-07-23 President And Fellows Of Harvard College Methods and compositions for restoring stmn2 levels
JP7795914B2 (ja) * 2019-06-03 2026-01-08 クラリス コーポレーション 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法
EP4162051A4 (en) * 2020-06-03 2025-07-09 Quralis Corp TREATMENT OF NEUROLOGICAL DISEASES USING GENE TRANSCRIPT MODULATORS
WO2022018187A1 (en) * 2020-07-23 2022-01-27 F. Hoffmann-La Roche Ag Oligonucleotides targeting rna binding protein sites
GB2617565A (en) * 2022-04-11 2023-10-18 Ucl Business Ltd A construct, vector and system and uses thereof
GB202208387D0 (en) * 2022-06-08 2022-07-20 Ucl Business Ltd Modified U7 snRNA construct
WO2024249458A2 (en) * 2023-05-31 2024-12-05 The Trustees Of Columbia University In The City Of New York Fine-tuning inflammation by altering 3'utr length
WO2025076126A1 (en) * 2023-10-02 2025-04-10 Emory University Agents that bind nucleic acids encoding elavl3 cryptic exons, pharmaceutical compositions, and uses in managing neurological diseases

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3103429A1 (en) * 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
JP7795914B2 (ja) * 2019-06-03 2026-01-08 クラリス コーポレーション 神経学的疾患を処置するためのオリゴヌクレオチドおよびその使用方法

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118737269A (zh) * 2024-08-30 2024-10-01 墨卓生物科技(浙江)有限公司 在单细胞微生物基因组测序结果中区分菌株的方法

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Publication number Publication date
EP4127172A4 (en) 2025-06-04
JP2026027295A (ja) 2026-02-18
WO2021195446A3 (en) 2021-11-11
WO2021195446A2 (en) 2021-09-30
CA3176884A1 (en) 2021-09-30
EP4127172A2 (en) 2023-02-08
JP2023519871A (ja) 2023-05-15
US20240011027A1 (en) 2024-01-11

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