EP4705453A2 - Système modulaire pour convertir des cassettes d'expression de microarn thérapeutique de promoteurs de polymérase iii à des promoteurs de polymérase ii - Google Patents

Système modulaire pour convertir des cassettes d'expression de microarn thérapeutique de promoteurs de polymérase iii à des promoteurs de polymérase ii

Info

Publication number
EP4705453A2
EP4705453A2 EP24729494.5A EP24729494A EP4705453A2 EP 4705453 A2 EP4705453 A2 EP 4705453A2 EP 24729494 A EP24729494 A EP 24729494A EP 4705453 A2 EP4705453 A2 EP 4705453A2
Authority
EP
European Patent Office
Prior art keywords
promoter
nucleotide sequence
nucleic acid
enhancer
seq
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
EP24729494.5A
Other languages
German (de)
English (en)
Inventor
Scott Quenton HARPER
Matthew GUGGENBILLER
Noah Taylor
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.)
Nationwide Childrens Hospital Inc
Original Assignee
Nationwide Childrens Hospital Inc
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
Application filed by Nationwide Childrens Hospital Inc filed Critical Nationwide Childrens Hospital Inc
Publication of EP4705453A2 publication Critical patent/EP4705453A2/fr
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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (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)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

L'invention concerne un système modulaire, c'est-à-dire une cassette d'expression d'ADN, spécifiquement conçu pour convertir des cassettes d'expression de miARN thérapeutiques de l'utilisation de promoteurs d'ARN polymérase III ubiquiste à l'utilisation de promoteurs d'ARN polymérase Il pour permettre l'expression spécifique de tissu des miARN tout en maintenant la fidélité et l'efficacité du traitement. L'invention concerne également des compositions, des vecteurs, des nanoparticules, des vésicules extracellulaires et des exosomes comprenant la cassette d'expression d'acide nucléique, ainsi que des procédés d'utilisation et des procédés de traitement faisant appel à la cassette d'expression d'ADN.
EP24729494.5A 2023-05-02 2024-05-02 Système modulaire pour convertir des cassettes d'expression de microarn thérapeutique de promoteurs de polymérase iii à des promoteurs de polymérase ii Pending EP4705453A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363499697P 2023-05-02 2023-05-02
PCT/US2024/027396 WO2024229211A2 (fr) 2023-05-02 2024-05-02 Système modulaire pour convertir des cassettes d'expression de microarn thérapeutique de promoteurs de polymérase iii à des promoteurs de polymérase ii

Publications (1)

Publication Number Publication Date
EP4705453A2 true EP4705453A2 (fr) 2026-03-11

Family

ID=91302426

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24729494.5A Pending EP4705453A2 (fr) 2023-05-02 2024-05-02 Système modulaire pour convertir des cassettes d'expression de microarn thérapeutique de promoteurs de polymérase iii à des promoteurs de polymérase ii

Country Status (3)

Country Link
EP (1) EP4705453A2 (fr)
AU (1) AU2024265701A1 (fr)
WO (1) WO2024229211A2 (fr)

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
ATE272123T1 (de) 1993-11-09 2004-08-15 Ohio Med College Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (fr) 1994-12-06 1996-06-13 Targeted Genetics Corporation Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
AU722196B2 (en) 1995-08-30 2000-07-27 Genzyme Corporation Chromatographic purification of adenovirus and AAV
EP1983057A3 (fr) 1995-09-08 2009-01-07 Genzyme Corporation Vecteurs AAV améliorés pour la thérapie génique
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
AU723497C (en) 1996-09-06 2001-10-11 Trustees Of The University Of Pennsylvania, The Method for recombinant adeno-associated virus-directed gene therapy
DK1944362T3 (en) 1997-09-05 2016-01-25 Genzyme Corp Fremgangsmåder til fremstilling af hjælpevirusfri præparater med høj titer af rekombinante AAV-vektorer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
WO2002053703A2 (fr) 2001-01-05 2002-07-11 Children's Hospital, Inc. Vecteurs aav2 et procedes
ES2975413T3 (es) 2001-12-17 2024-07-05 Univ Pennsylvania Secuencias de serotipo 8 de virus adenoasociado (AAV), vectores que las contienen y usos de las mismas
AU2012286994B2 (en) 2011-07-25 2017-08-10 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of DUX4
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (fr) 2014-03-21 2015-09-24 株式会社日立国際電気 Appareil de traitement de substrat, procédé de fabrication de dispositif semi-conducteur et support d'enregistrement
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
EP3997225A1 (fr) * 2019-07-10 2022-05-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Méthodes pour le traitement de l'épilepsie
WO2022119826A1 (fr) * 2020-12-01 2022-06-09 Research Institute At Nationwide Children's Hospital Compositions et procédés pour l'inhibition de l'expression de la protéine 22 de la myéline périphérique

Also Published As

Publication number Publication date
WO2024229211A3 (fr) 2024-12-12
AU2024265701A9 (en) 2026-01-08
AU2024265701A1 (en) 2025-12-04
WO2024229211A2 (fr) 2024-11-07

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