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WO2024263991A3 - Méthodes et compositions pour vecteurs viraux déficients pour l'intégration - Google Patents

Méthodes et compositions pour vecteurs viraux déficients pour l'intégration Download PDF

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Publication number
WO2024263991A3
WO2024263991A3 PCT/US2024/035123 US2024035123W WO2024263991A3 WO 2024263991 A3 WO2024263991 A3 WO 2024263991A3 US 2024035123 W US2024035123 W US 2024035123W WO 2024263991 A3 WO2024263991 A3 WO 2024263991A3
Authority
WO
WIPO (PCT)
Prior art keywords
compositions
methods
viral vectors
integration deficient
deficient viral
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
PCT/US2024/035123
Other languages
English (en)
Other versions
WO2024263991A2 (fr
Inventor
Luke Gilbert
Gary Wang
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.)
Arc Research Institute
University of California Berkeley
University of California San Diego UCSD
Original Assignee
Arc Research Institute
University of California Berkeley
University of California San Diego UCSD
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 Arc Research Institute, University of California Berkeley, University of California San Diego UCSD filed Critical Arc Research Institute
Publication of WO2024263991A2 publication Critical patent/WO2024263991A2/fr
Publication of WO2024263991A3 publication Critical patent/WO2024263991A3/fr
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203—Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente divulgation concerne de manière générale des compositions et des méthodes comprenant des vecteurs rétroviraux déficients pour l'intégration recombinante, l'expression de cargo(s) d'intérêt dépendant d'un PRE en l'absence d'un promoteur exogène.
PCT/US2024/035123 2023-06-22 2024-06-21 Méthodes et compositions pour vecteurs viraux déficients pour l'intégration Pending WO2024263991A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363509673P 2023-06-22 2023-06-22
US63/509,673 2023-06-22

Publications (2)

Publication Number Publication Date
WO2024263991A2 WO2024263991A2 (fr) 2024-12-26
WO2024263991A3 true WO2024263991A3 (fr) 2025-04-17

Family

ID=93936324

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/035123 Pending WO2024263991A2 (fr) 2023-06-22 2024-06-21 Méthodes et compositions pour vecteurs viraux déficients pour l'intégration

Country Status (1)

Country Link
WO (1) WO2024263991A2 (fr)

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018058002A1 (fr) * 2016-09-23 2018-03-29 Fred Hutchinson Cancer Research Center Tcr spécifiques de l'antigène ha-1 d'histocompatibilité (h) mineure et ses utilisations
US20180119170A1 (en) * 2007-12-11 2018-05-03 The University Of North Carolina At Chapel Hill Polypurine tract modified retroviral vectors
WO2021013867A1 (fr) * 2019-07-22 2021-01-28 Genethon Intégration précise à l'aide d'un idlv ciblant une nucléase

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180119170A1 (en) * 2007-12-11 2018-05-03 The University Of North Carolina At Chapel Hill Polypurine tract modified retroviral vectors
WO2018058002A1 (fr) * 2016-09-23 2018-03-29 Fred Hutchinson Cancer Research Center Tcr spécifiques de l'antigène ha-1 d'histocompatibilité (h) mineure et ses utilisations
WO2021013867A1 (fr) * 2019-07-22 2021-01-28 Genethon Intégration précise à l'aide d'un idlv ciblant une nucléase

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
HAGINO-YAMAGISHI K, DONEHOWER L A, VARMUS H E: "Retroviral DNA integrated during infection by an integration-deficient mutant of murine leukemia virus is oligomeric", JOURNAL OF VIROLOGY, vol. 61, no. 6, 1 June 1987 (1987-06-01), US , pages 1964 - 1971, XP093306122, ISSN: 0022-538X, DOI: 10.1128/jvi.61.6.1964-1971.1987 *
MATJUSAITIS MANTAS, TEDESCO DONATO, CIUKAS AZUOLAS: "Adaptation of STARR-seq method to be used with 3 rd generation integrase-deficient promoterless lentiviral vectors", BIORXIV, 26 June 2020 (2020-06-26), pages 1 - 7, XP093306121, Retrieved from the Internet <URL:https://www.biorxiv.org/content/10.1101/2020.06.24.169714v1.full.pdf> DOI: 10.1101/2020.06.24.169714 *

Also Published As

Publication number Publication date
WO2024263991A2 (fr) 2024-12-26

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