WO2003052071A3 - Intracellular generation of single-stranded dna - Google Patents

Intracellular generation of single-stranded dna Download PDF

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Publication number
WO2003052071A3
WO2003052071A3 PCT/US2002/040024 US0240024W WO03052071A3 WO 2003052071 A3 WO2003052071 A3 WO 2003052071A3 US 0240024 W US0240024 W US 0240024W WO 03052071 A3 WO03052071 A3 WO 03052071A3
Authority
WO
WIPO (PCT)
Prior art keywords
single stranded
stranded dna
dna
chromosomal
triplex
Prior art date
Application number
PCT/US2002/040024
Other languages
French (fr)
Other versions
WO2003052071A2 (en
Inventor
Peter M Glazer
Original Assignee
Univ Yale
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 Univ Yale filed Critical Univ Yale
Priority to IL16246202A priority Critical patent/IL162462A0/en
Priority to MXPA04005747A priority patent/MXPA04005747A/en
Priority to CA002470033A priority patent/CA2470033A1/en
Priority to EP02798516A priority patent/EP1468086A2/en
Priority to AU2002363997A priority patent/AU2002363997A1/en
Publication of WO2003052071A2 publication Critical patent/WO2003052071A2/en
Publication of WO2003052071A3 publication Critical patent/WO2003052071A3/en

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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
    • C12N15/11—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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing 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
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid

Abstract

Methods for intracellularly generating single stranded DNA molecules that are active in mediating triplex-dependent or independent chromosomal events are provided herein. The method is based on the discovery that one can introduce viral vectors or plasmids into the cells, where they generate within the cells to be engineered the single stranded DNA molecules that bind to the target chromosomal DNA to form a triplex, which may induce a desired mutation, and/or be recombinagenic and induce a change to the chromosomal DNA by incorporation of a donor DNA molecule. The vectors or plasmid not only encode the TFO and optionally the donor DNA, but also a reverse transcriptase, and optionally, a restriction enzyme, which is present in the preferred embodiment as a fusion protein which reverse transcribes the RNA encoded by the vector or plasmid, then cleaves it at a restriction enzyme site to yield a single stranded DNA. The single stranded DNA may be produced directly, or initially as a double stranded stem-single stranded loop structure, which is then cleaved to yield the single stranded DNA.
PCT/US2002/040024 2001-12-14 2002-12-13 Intracellular generation of single-stranded dna WO2003052071A2 (en)

Priority Applications (5)

Application Number Priority Date Filing Date Title
IL16246202A IL162462A0 (en) 2001-12-14 2002-12-13 Intracellular generation of single-stranded dna
MXPA04005747A MXPA04005747A (en) 2001-12-14 2002-12-13 Intracellular generation of single-stranded dna.
CA002470033A CA2470033A1 (en) 2001-12-14 2002-12-13 Intracellular generation of single-stranded dna
EP02798516A EP1468086A2 (en) 2001-12-14 2002-12-13 INTRACELLULAR GENERATION OF SINGLE−STRANDED DNA
AU2002363997A AU2002363997A1 (en) 2001-12-14 2002-12-13 Intracellular generation of single-stranded dna

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US34080301P 2001-12-14 2001-12-14
US60/340,803 2001-12-14

Publications (2)

Publication Number Publication Date
WO2003052071A2 WO2003052071A2 (en) 2003-06-26
WO2003052071A3 true WO2003052071A3 (en) 2003-11-20

Family

ID=23335000

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2002/040024 WO2003052071A2 (en) 2001-12-14 2002-12-13 Intracellular generation of single-stranded dna

Country Status (8)

Country Link
US (1) US20030148352A1 (en)
EP (1) EP1468086A2 (en)
CN (1) CN1620503A (en)
AU (1) AU2002363997A1 (en)
CA (1) CA2470033A1 (en)
IL (1) IL162462A0 (en)
MX (1) MXPA04005747A (en)
WO (1) WO2003052071A2 (en)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050020526A1 (en) * 2003-06-03 2005-01-27 Cytogenix, Inc. Oligodeoxynucleotide intervention for prevention and treatment of sepsis
US20040248101A1 (en) * 2003-06-03 2004-12-09 Cytogenix, Inc. Identification of novel antibacteria agents by screening the single-stranded DNA expression library
EP1773995B1 (en) * 2004-08-03 2009-10-07 The Regents Of The University Of California Site specific system for generating diversity protein sequences
WO2015172149A1 (en) 2014-05-09 2015-11-12 Yale University Hyperbranched polyglycerol-coated particles and methods of making and using thereof
US11918695B2 (en) 2014-05-09 2024-03-05 Yale University Topical formulation of hyperbranched polymer-coated particles
CN109476706A (en) * 2016-02-16 2019-03-15 耶鲁大学 For promoting the composition and its application method of target gene editor
WO2017143061A1 (en) 2016-02-16 2017-08-24 Yale University Compositions and methods for treatment of cystic fibrosis
WO2018187493A1 (en) 2017-04-04 2018-10-11 Yale University Compositions and methods for in utero delivery
US20210189431A1 (en) 2018-08-10 2021-06-24 Yale University Compositions and methods for embryonic gene editing in vitro
CN112912502A (en) 2018-08-31 2021-06-04 耶鲁大学 Compositions and methods for enhancing triplex and nuclease-based gene editing
WO2020112195A1 (en) 2018-11-30 2020-06-04 Yale University Compositions, technologies and methods of using plerixafor to enhance gene editing
US20220243211A1 (en) 2019-06-21 2022-08-04 Yale University Peptide nucleic acid compositions with modified hoogsteen binding segments and methods of use thereof
WO2020257779A1 (en) 2019-06-21 2020-12-24 Yale University Hydroxymethyl-modified gamma-pna compositions and methods of use thereof
US20220339294A1 (en) 2019-09-09 2022-10-27 Yale University Nanoparticles for selective tissue or cellular uptake

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000022114A1 (en) * 1998-10-09 2000-04-20 Ingene, Inc. PRODUCTION OF ssDNA $i(IN VIVO)
WO2000022113A1 (en) * 1998-10-09 2000-04-20 Ingene, Inc. ENZYMATIC SYNTHESIS OF ssDNA
WO2001025419A1 (en) * 1999-10-04 2001-04-12 Cytogenix, Inc. ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5792632A (en) * 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5962426A (en) * 1993-06-25 1999-10-05 Yale University Triple-helix forming oligonucleotides for targeted mutagenesis

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000022114A1 (en) * 1998-10-09 2000-04-20 Ingene, Inc. PRODUCTION OF ssDNA $i(IN VIVO)
WO2000022113A1 (en) * 1998-10-09 2000-04-20 Ingene, Inc. ENZYMATIC SYNTHESIS OF ssDNA
WO2001025419A1 (en) * 1999-10-04 2001-04-12 Cytogenix, Inc. ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO

Also Published As

Publication number Publication date
EP1468086A2 (en) 2004-10-20
CA2470033A1 (en) 2003-06-26
AU2002363997A1 (en) 2003-06-30
IL162462A0 (en) 2005-11-20
WO2003052071A2 (en) 2003-06-26
US20030148352A1 (en) 2003-08-07
MXPA04005747A (en) 2005-07-05
CN1620503A (en) 2005-05-25

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