WO2026006151A3 - Fonctionnalisation d'ace-arnt codant pour des picovecteurs linéaires synthétiques - Google Patents

Fonctionnalisation d'ace-arnt codant pour des picovecteurs linéaires synthétiques

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Publication number
WO2026006151A3
WO2026006151A3 PCT/US2025/034736 US2025034736W WO2026006151A3 WO 2026006151 A3 WO2026006151 A3 WO 2026006151A3 US 2025034736 W US2025034736 W US 2025034736W WO 2026006151 A3 WO2026006151 A3 WO 2026006151A3
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WIPO (PCT)
Prior art keywords
ace
trna
constructs
picovectors
nuclear
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Pending
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PCT/US2025/034736
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English (en)
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WO2026006151A2 (fr
Inventor
John LUECK
Joseph Porter
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University of Rochester
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University of Rochester
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Publication of WO2026006151A2 publication Critical patent/WO2026006151A2/fr
Publication of WO2026006151A3 publication Critical patent/WO2026006151A3/fr
Pending legal-status Critical Current
Anticipated expiration legal-status Critical

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    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/532—Closed or circular
    • 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
    • C12N2320/32—Special delivery means, e.g. tissue-specific

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  • 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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

La présente divulgation concerne des compositions et des méthodes de traitement de troubles génétiques causés par des mutations non-sens à l'aide de constituants d'ARN de transfert à modification d'anticodon (ACE-ARNt). Ces constituants d'ACE-ARNt à base d'ADN sont conçues pour supprimer des codons de terminaison prématurés (PTC) et pour restaurer l'expression de protéines fonctionnelles pleine longueur. La divulgation concerne en outre des formulations de constituants d'ACE-ARNt comportant des nanoparticules polymères de poly(amine-co-ester) (PACE) pour améliorer la stabilité, pour protéger des acides nucléiques et pour améliorer l'administration aux cellules épithéliales des voies respiratoires. La divulgation concerne également des picovecteurs d'ACE-ARNt fonctionnalisés (sLPV) incorporant des éléments de ciblage tels que des signaux de localisation nucléaire (NLS), des séquences de localisation nucléolaire (NoLS), et des séquences de ciblage nucléaire d'ADN (DTS) pour améliorer l'importation et la localisation nucléaires.
PCT/US2025/034736 2024-06-24 2025-06-23 Fonctionnalisation d'ace-arnt codant pour des picovecteurs linéaires synthétiques Pending WO2026006151A2 (fr)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US202463663222P 2024-06-24 2024-06-24
US63/663,222 2024-06-24
US202463719360P 2024-11-12 2024-11-12
US63/719,360 2024-11-12

Publications (2)

Publication Number Publication Date
WO2026006151A2 WO2026006151A2 (fr) 2026-01-02
WO2026006151A3 true WO2026006151A3 (fr) 2026-02-12

Family

ID=96658436

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2025/034736 Pending WO2026006151A2 (fr) 2024-06-24 2025-06-23 Fonctionnalisation d'ace-arnt codant pour des picovecteurs linéaires synthétiques

Country Status (1)

Country Link
WO (1) WO2026006151A2 (fr)

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WO2024178172A1 (fr) * 2023-02-23 2024-08-29 University Of Rochester Agents et méthodes de production de molécules de fil d'adn à extrémités fermées

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US20080187999A1 (en) * 2007-02-06 2008-08-07 Uthayashanker Ezekiel Methods and systems for high homologous recombination ("HR") targeting efficiency
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WO2021252354A1 (fr) * 2020-06-12 2021-12-16 University Of Rochester Codage et expression d'arnt ace
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