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
Links
Classifications
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- 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
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- 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
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- 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]
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P35/00—Antineoplastic agents
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- 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
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- 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
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- 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
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- 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)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Veterinary Medicine (AREA)
- Neurology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Neurosurgery (AREA)
- Oncology (AREA)
- Pain & Pain Management (AREA)
- Rheumatology (AREA)
- Communicable Diseases (AREA)
- Virology (AREA)
- Psychology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Enzymes And Modification 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.
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)
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 |
DE60132924T2 (en) | 2000-10-23 | 2009-03-05 | Gen-Probe Inc., San Diego | COMPOSITIONS AND METHODS FOR DETECTING HUMAN IMMUNODICITY VIRUS 2 (HIV-2) |
AU2002363997A1 (en) * | 2001-12-14 | 2003-06-30 | 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 |
WO2006037127A2 (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 | 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 |
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)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
JP2002527061A (en) * | 1998-10-09 | 2002-08-27 | インジーン・インコーポレイテッド | Enzymatic synthesis of ssDNA |
MXPA01003643A (en) * | 1998-10-09 | 2003-07-21 | Ingene Inc | PRODUCTION OF ssDNA IN VIVO. |
-
2000
- 2000-10-04 EP EP00968678A patent/EP1222259A1/en not_active Withdrawn
- 2000-10-04 IL IL14894600A patent/IL148946A0/en unknown
- 2000-10-04 JP JP2001528572A patent/JP2003511025A/en active Pending
- 2000-10-04 BR BR0014814-8A patent/BR0014814A/en not_active IP Right Cessation
- 2000-10-04 KR KR1020027004395A patent/KR20020059608A/en not_active Application Discontinuation
- 2000-10-04 WO PCT/US2000/027381 patent/WO2001025419A1/en not_active Application Discontinuation
- 2000-10-04 CA CA002386246A patent/CA2386246A1/en not_active Abandoned
- 2000-10-04 AU AU78553/00A patent/AU7855300A/en not_active Abandoned
- 2000-10-04 MX MXPA02003422A patent/MXPA02003422A/en unknown
- 2000-10-04 CN CNB008158568A patent/CN1276083C/en not_active Expired - Fee Related
-
2002
- 2002-04-03 CR CR6613A patent/CR6613A/en not_active Application Discontinuation
Also Published As
Publication number | Publication date |
---|---|
CN1276083C (en) | 2006-09-20 |
JP2003511025A (en) | 2003-03-25 |
IL148946A0 (en) | 2002-09-12 |
EP1222259A1 (en) | 2002-07-17 |
AU7855300A (en) | 2001-05-10 |
CN1399679A (en) | 2003-02-26 |
CA2386246A1 (en) | 2001-04-12 |
WO2001025419A1 (en) | 2001-04-12 |
CR6613A (en) | 2004-04-21 |
KR20020059608A (en) | 2002-07-13 |
BR0014814A (en) | 2002-09-17 |
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