CN119013399A - 靶向假外显子的等位基因特异性剪接转换寡核苷酸 - Google Patents

靶向假外显子的等位基因特异性剪接转换寡核苷酸 Download PDF

Info

Publication number
CN119013399A
CN119013399A CN202380030631.5A CN202380030631A CN119013399A CN 119013399 A CN119013399 A CN 119013399A CN 202380030631 A CN202380030631 A CN 202380030631A CN 119013399 A CN119013399 A CN 119013399A
Authority
CN
China
Prior art keywords
sso
gene
pseudoexon
mrna
splice site
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.)
Pending
Application number
CN202380030631.5A
Other languages
English (en)
Chinese (zh)
Inventor
布拉吉·斯托尔斯泰因·安德烈森
托马斯·科艾德·多克托
利兹·洛莱·霍尔姆
乌尔丽卡·西蒙妮·斯潘斯伯格·彼得森
吉特·霍夫曼·布鲁恩
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Syddansk Universitet
Original Assignee
Syddansk Universitet
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
Priority claimed from PCT/EP2022/051790 external-priority patent/WO2022162020A2/en
Application filed by Syddansk Universitet filed Critical Syddansk Universitet
Publication of CN119013399A publication Critical patent/CN119013399A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN202380030631.5A 2022-01-26 2023-01-26 靶向假外显子的等位基因特异性剪接转换寡核苷酸 Pending CN119013399A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EPPCT/EP2022/051790 2022-01-26
PCT/EP2022/051790 WO2022162020A2 (en) 2021-01-26 2022-01-26 Splice switching oligonucleotides targeting pseudoexons
DKPA202270346 2022-06-29
DKPA202270346 2022-06-29
PCT/EP2023/051916 WO2023144263A1 (en) 2022-01-26 2023-01-26 Allele specific splice switching oligonucleotides targeting pseudoexons

Publications (1)

Publication Number Publication Date
CN119013399A true CN119013399A (zh) 2024-11-22

Family

ID=85108839

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380030631.5A Pending CN119013399A (zh) 2022-01-26 2023-01-26 靶向假外显子的等位基因特异性剪接转换寡核苷酸

Country Status (7)

Country Link
US (1) US20250101419A1 (https=)
EP (1) EP4469572A1 (https=)
JP (1) JP2025504661A (https=)
CN (1) CN119013399A (https=)
AU (1) AU2023210747A1 (https=)
CA (1) CA3247528A1 (https=)
WO (1) WO2023144263A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024243087A2 (en) * 2023-05-19 2024-11-28 Leverna Therapeutics Inc. Compositions and methods for regulating expression of atxn2

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108603230A (zh) * 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
WO2017087282A1 (en) * 2015-11-18 2017-05-26 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (lrrk2) for the treatment of parkinsons disease
KR102318434B1 (ko) * 2017-08-25 2021-11-01 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
TWI833770B (zh) * 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
US12595479B2 (en) * 2019-03-20 2026-04-07 President And Fellows Of Harvard College Antisense oligonucleotide-based progranulin augmentation therapy in neurodegenerative diseases
WO2022119881A1 (en) * 2020-12-01 2022-06-09 Emendobio Inc. Differential knockout of a heterozygous allele of lrrk2
CA3204779A1 (en) * 2021-01-26 2022-08-04 Brage Storstein Andresen Splice switching oligonucleotides targeting pseudoexons

Also Published As

Publication number Publication date
WO2023144263A1 (en) 2023-08-03
AU2023210747A1 (en) 2024-08-01
CA3247528A1 (en) 2023-08-03
EP4469572A1 (en) 2024-12-04
JP2025504661A (ja) 2025-02-14
US20250101419A1 (en) 2025-03-27

Similar Documents

Publication Publication Date Title
Haddad et al. Biogenesis and function of circular RNAs in health and in disease
Zhao et al. P53-R273H mutation enhances colorectal cancer stemness through regulating specific lncRNAs
Wang et al. Circular RNAs as potential biomarkers for cancer diagnosis and therapy
Song et al. Long non-coding RNA PVT1 promotes glycolysis and tumor progression by regulating miR-497/HK2 axis in osteosarcoma
Lee et al. MicroRNA-296-5p (miR-296-5p) functions as a tumor suppressor in prostate cancer by directly targeting Pin1
Hu et al. miR-501-3p mediates the activity-dependent regulation of the expression of AMPA receptor subunit GluA1
Cheng et al. Foxo3a-mediated overexpression of microRNA-622 suppresses tumor metastasis by repressing hypoxia-inducible factor-1α in ERK-responsive lung cancer
Xiong et al. Role of miR‑34 in gastric cancer: From bench to bedside
JP2015518485A (ja) 熱発生のｍｉＲＮＡ調節剤
Vetere et al. Selective inhibition of miR‐92 in hippocampal neurons alters contextual fear memory
Kong et al. Recent advances in understanding FOXN3 in breast cancer, and other malignancies
Nielsen et al. Directing HER4 mRNA expression towards the CYT2 isoform by antisense oligonucleotide decreases growth of breast cancer cells in vitro and in vivo
Saramago et al. The implication of mRNA degradation disorders on human DISease: focus on DIS3 and DIS3-like enzymes
Bruxel et al. Multi‐omic strategies applied to the study of pharmacoresistance in mesial temporal lobe epilepsy
Chen et al. Tumor-associated intronic editing of HNRPLL generates a novel splicing variant linked to cell proliferation
Boguslawska et al. Regulatory feedback loop between T3 and microRNAs in renal cancer
WO2009149318A2 (en) Mirnas as therapeutic targets in cancer
Krauss et al. The role of microRNAs in spinocerebellar ataxia type 3
Yang et al. DNMT3B regulates proliferation of A549 cells through the microRNA-152-3p/NCAM1 pathway
Sheng et al. Decoding the role of aberrant RNA alternative splicing in hepatocellular carcinoma: a comprehensive review
CN119013399A (zh) 靶向假外显子的等位基因特异性剪接转换寡核苷酸
Chen et al. Mutant huntingtin induces neuronal apoptosis via derepressing the non-canonical poly (A) polymerase PAPD5
Jia et al. miR-29b upregulates miR-195 by targeting DNMT3B in tongue squamous cell carcinoma
EP4482958A1 (en) Therapeutics for syngap haploinsufficiency
WO2023049800A1 (en) Therapeutics for haploinsufficiency conditions

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination