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MXPA02003422A - ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO. - Google Patents

ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO.

Info

Publication number
MXPA02003422A
MXPA02003422A MXPA02003422A MXPA02003422A MXPA02003422A MX PA02003422 A MXPA02003422 A MX PA02003422A MX PA02003422 A MXPA02003422 A MX PA02003422A MX PA02003422 A MXPA02003422 A MX PA02003422A MX PA02003422 A MXPA02003422 A MX PA02003422A
Authority
MX
Mexico
Prior art keywords
sequence
cdna
stem
target cell
double stranded
Prior art date
Application number
MXPA02003422A
Other languages
Spanish (es)
Inventor
A Conrad Charles
Original Assignee
Cytogenix 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 Cytogenix Inc filed Critical Cytogenix Inc
Publication of MXPA02003422A publication Critical patent/MXPA02003422A/en

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
    • C12N15/113—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Neurology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Neurosurgery (AREA)
  • Communicable Diseases (AREA)
  • Rheumatology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Pain & Pain Management (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

A methods for altering expression of a target nucleic acid sequence in a target cell by production of single stranded cDNA (ss cDNA) in the target cell in vivo. The target cell is transfected with a cassette comprising a sequence of interest, an inverted tandem repeat, and a primer binding site 3 to the inverted tandem repeat. Transcription of the cassette by the target cell produces an RNA template which is reverse transcribed to produce ss cDNA of a specified sequence. A reverse transcriptase RNAse H coding gene may also be transfected into the target cell. The ss cDNA is modified to remove all flanking vector sequences by taking advantage of the stem loop structure of the ss cDNA, which forms as a result of the inverted tandem repeat that allows the ss cDNA to fold back on itself, forming a double stranded DNA stem. The double stranded stem contains one or more restriction endonuclease recognition sites and the loop, which remains as ssDNA, is comprised of the sequence of interest, which can be any desired nucleotide sequence. This design allows the double stranded stem of the stem loop intermediate to be cleaved by the desired corresponding restriction endonuclease(s) and the loop portion, or sequence of interest, is then released as a linearized, single stranded piece of DNA. This released (or cleaved) ssDNA piece contains minimal, if any, sequence information either upstream 5 or downstream 3 from the double stranded stem. The resulting ssDNA binds to an endogenous target nucleic acid sequence to alter the expression of that sequence for such therapeutic purposes as gene inactivation using duplex or triplex binding of nucleic acids, site directed mutagenesis, interruption of cellular function by binding to specific cellular proteins, and interfering with RNA splicing functions.
MXPA02003422A 1999-10-04 2000-10-04 ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO. MXPA02003422A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US41156899A 1999-10-04 1999-10-04
US51470700A 2000-02-28 2000-02-28
PCT/US2000/027381 WO2001025419A1 (en) 1999-10-04 2000-10-04 ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO

Publications (1)

Publication Number Publication Date
MXPA02003422A true MXPA02003422A (en) 2004-09-10

Family

ID=27021437

Family Applications (1)

Application Number Title Priority Date Filing Date
MXPA02003422A MXPA02003422A (en) 1999-10-04 2000-10-04 ALTERING GENE EXPRESSION WITH ssDNA PRODUCED IN VIVO.

Country Status (11)

Country Link
EP (1) EP1222259A1 (en)
JP (1) JP2003511025A (en)
KR (1) KR20020059608A (en)
CN (1) CN1276083C (en)
AU (1) AU7855300A (en)
BR (1) BR0014814A (en)
CA (1) CA2386246A1 (en)
CR (1) CR6613A (en)
IL (1) IL148946A0 (en)
MX (1) MXPA02003422A (en)
WO (1) WO2001025419A1 (en)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7419964B2 (en) 1999-09-16 2008-09-02 Cytogenix, Inc. Treatment of HSV-related pathologies using ssDNA
WO2002034951A2 (en) 2000-10-23 2002-05-02 Gen-Probe Incorporated Compositions and methods for detecting human immunodeficiency virus 2 (hiv-2)
CA2470033A1 (en) * 2001-12-14 2003-06-26 Yale University Intracellular generation of single-stranded dna
US20050020526A1 (en) * 2003-06-03 2005-01-27 Cytogenix, Inc. Oligodeoxynucleotide intervention for prevention and treatment of sepsis
CA2582814A1 (en) * 2004-09-28 2006-04-06 Cytogenix, Inc. Single-stranded antimicrobial oligonucleotides and uses thereof
US8658608B2 (en) 2005-11-23 2014-02-25 Yale University Modified triple-helix forming oligonucleotides for targeted mutagenesis
CN102154269B (en) * 2011-01-06 2012-05-23 湖南农业大学 PCR-based rapid construction method of tandem repeat sequence, special primer pair and application
JP2019508037A (en) 2016-02-16 2019-03-28 イェール ユニバーシティーＹａｌｅ Ｕｎｉｖｅｒｓｉｔ Compositions for enhancing targeted gene editing and methods of use thereof
CN108396043A (en) * 2017-02-06 2018-08-14 中国科学院上海应用物理研究所 A kind of preparation method and applications of the ends 5` phosphorylation single stranded DNA
EP3873537A4 (en) * 2018-11-02 2022-10-12 Nikegen Limited Recombinant parvoviral vectors and method of making and use thereof
GB201913898D0 (en) * 2019-09-26 2019-11-13 Lightbio Ltd Nucleic acid construct

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU6298899A (en) * 1998-10-09 2000-05-01 Ingene, Inc. Production of ssdna (in vivo)
BR9914772A (en) * 1998-10-09 2001-12-11 Ingene Inc Set of genetic elements, vector, host cell, set for the production of a nucleic acid sequence, method for in vivo or in vitro production of a nucleic acid sequence, cdna transcription, inhibitor nucleic acid molecule, mrna transcription, heteroduplex molecule and pharmaceutical composition

Also Published As

Publication number Publication date
AU7855300A (en) 2001-05-10
KR20020059608A (en) 2002-07-13
CA2386246A1 (en) 2001-04-12
EP1222259A1 (en) 2002-07-17
CR6613A (en) 2004-04-21
IL148946A0 (en) 2002-09-12
BR0014814A (en) 2002-09-17
WO2001025419A1 (en) 2001-04-12
JP2003511025A (en) 2003-03-25
CN1276083C (en) 2006-09-20
CN1399679A (en) 2003-02-26

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