JP2024524974A - ナンセンス変異依存ｒｎａ分解機構に基づく病態及び疾患の処置のためのアンチセンスオリゴマー - Google Patents

ナンセンス変異依存ｒｎａ分解機構に基づく病態及び疾患の処置のためのアンチセンスオリゴマー Download PDF

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JP2024524974A
JP2024524974A JP2023578922A JP2023578922A JP2024524974A JP 2024524974 A JP2024524974 A JP 2024524974A JP 2023578922 A JP2023578922 A JP 2023578922A JP 2023578922 A JP2023578922 A JP 2023578922A JP 2024524974 A JP2024524974 A JP 2024524974A
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nucleotides
mrna
phip
exon
nmd
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JP2024524974A5 (enExample
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アズナレズ，イザベル
サイズ，アナ・コリオネロ
カハ，ヤコブ・アルバート
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ストーク セラピューティクス，インク．
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/314—Phosphoramidates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/315—Phosphorothioates
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
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    • 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
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    • 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
    • C12N2310/3233—Morpholino-type ring
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
JP2023578922A 2021-06-21 2022-06-21 ナンセンス変異依存ｒｎａ分解機構に基づく病態及び疾患の処置のためのアンチセンスオリゴマー Pending JP2024524974A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163212981P 2021-06-21 2021-06-21
US63/212,981 2021-06-21
PCT/US2022/034344 WO2022271699A2 (en) 2021-06-21 2022-06-21 Antisense oligomers for treatment of non-sense mediated rna decay based conditions and diseases

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JP2024524974A true JP2024524974A (ja) 2024-07-09
JP2024524974A5 JP2024524974A5 (enExample) 2025-07-01

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US (1) US20240254488A1 (enExample)
EP (1) EP4359538A4 (enExample)
JP (1) JP2024524974A (enExample)
CN (1) CN117980479A (enExample)
WO (1) WO2022271699A2 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12338437B2 (en) 2020-05-11 2025-06-24 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
TW202504646A (zh) * 2023-06-16 2025-02-01 北京大學 利用環狀ｒｎａ抑制無義介導的降解的系統和方法

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1280903A2 (en) * 2000-05-11 2003-02-05 Maria Rozakis-Adcock Ph domain-interacting protein
US20130288967A1 (en) * 2012-04-25 2013-10-31 Euclid Diagnostics Llc Materials and methods for cancer diagnosis by evaluation of the methylation status of cpg islands on chromosomes 6 and 8
US20150376612A1 (en) * 2014-06-10 2015-12-31 The General Hospital Corporation CCCTC-Binding Factor (CTCF) RNA Interactome
LT3700570T (lt) * 2017-10-23 2025-04-10 Stoke Therapeutics, Inc. Priešprasmiai oligomerai, skirti būklių ir ligų, pagrįstų nonsens kodono nulemta mrnr degradacija, gydymui

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021034985A1 (en) * 2019-08-19 2021-02-25 Stoke Therapeutics, Inc. Compositions and methods for modulating splicing and protein expression

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
GENOME BIOLOGY, vol. 22, JPN6026025833, 2021, pages 132, ISSN: 0005889701 *

Also Published As

Publication number Publication date
EP4359538A4 (en) 2025-09-17
EP4359538A2 (en) 2024-05-01
WO2022271699A3 (en) 2023-02-09
CN117980479A (zh) 2024-05-03
WO2022271699A2 (en) 2022-12-29
US20240254488A1 (en) 2024-08-01

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