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

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense spanning the whole gene, or a large part of it
    • CCHEMISTRY; METALLURGY
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed

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  • 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)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Psychology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Oncology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Saccharide Compounds (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 (10)

* 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
US6870045B2 (en) 2000-10-23 2005-03-22 Gen-Probe Incorporated Kits for detecting HIV-2
MXPA04005747A (en) * 2001-12-14 2005-07-05 Univ Yale Intracellular generation of single-stranded dna.
US20050020526A1 (en) * 2003-06-03 2005-01-27 Cytogenix, Inc. Oligodeoxynucleotide intervention for prevention and treatment of sepsis
EP1802754A2 (en) * 2004-09-28 2007-07-04 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 Polymerase chain reaction (PCR)-based quick construction method for tandem repetitive sequence and special primer pair and use
US11136597B2 (en) 2016-02-16 2021-10-05 Yale University 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
CN113195001A (en) * 2018-11-02 2021-07-30 耐克基因有限责任公司 Recombinant parvovirus vector and preparation method and application thereof

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2000022113A1 (en) * 1998-10-09 2000-04-20 Ingene, Inc. ENZYMATIC SYNTHESIS OF ssDNA
AU6298899A (en) * 1998-10-09 2000-05-01 Ingene, Inc. Production of ssdna (in vivo)

Also Published As

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

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