ATE31536T1 - Verbindungen,welche eine oligonucleotide sequenz enthalten, die an einen interkalationsstoff gebunden ist, verfahren zu deren synthese und deren verwendung. - Google Patents

Verbindungen,welche eine oligonucleotide sequenz enthalten, die an einen interkalationsstoff gebunden ist, verfahren zu deren synthese und deren verwendung.

Info

Publication number
ATE31536T1
ATE31536T1 AT84400143T AT84400143T ATE31536T1 AT E31536 T1 ATE31536 T1 AT E31536T1 AT 84400143 T AT84400143 T AT 84400143T AT 84400143 T AT84400143 T AT 84400143T AT E31536 T1 ATE31536 T1 AT E31536T1
Authority
AT
Austria
Prior art keywords
intercalative
synthesis
methods
compounds containing
oligonucleotide sequence
Prior art date
Application number
AT84400143T
Other languages
English (en)
Inventor
Thanh Thuong Nguyen
Claude Helene
Ulysse Asseline
Original Assignee
Centre Nat Rech Scient
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 Centre Nat Rech Scient filed Critical Centre Nat Rech Scient
Application granted granted Critical
Publication of ATE31536T1 publication Critical patent/ATE31536T1/de

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Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Saccharide Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AT84400143T 1983-01-27 1984-01-23 Verbindungen,welche eine oligonucleotide sequenz enthalten, die an einen interkalationsstoff gebunden ist, verfahren zu deren synthese und deren verwendung. ATE31536T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
FR8301223A FR2540122B1 (fr) 1983-01-27 1983-01-27 Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
EP84400143A EP0117777B1 (de) 1983-01-27 1984-01-23 Verbindungen,welche eine oligonucleotide Sequenz enthalten, die an einen Interkalationsstoff gebunden ist, Verfahren zu deren Synthese und deren Verwendung

Publications (1)

Publication Number Publication Date
ATE31536T1 true ATE31536T1 (de) 1988-01-15

Family

ID=9285325

Family Applications (1)

Application Number Title Priority Date Filing Date
AT84400143T ATE31536T1 (de) 1983-01-27 1984-01-23 Verbindungen,welche eine oligonucleotide sequenz enthalten, die an einen interkalationsstoff gebunden ist, verfahren zu deren synthese und deren verwendung.

Country Status (6)

Country Link
US (1) US4835263A (de)
EP (1) EP0117777B1 (de)
JP (1) JP2519406B2 (de)
AT (1) ATE31536T1 (de)
DE (1) DE3468220D1 (de)
FR (1) FR2540122B1 (de)

Families Citing this family (710)

* Cited by examiner, † Cited by third party
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JPS59141598A (ja) 1984-08-14
DE3468220D1 (en) 1988-02-04
EP0117777B1 (de) 1987-12-23
JP2519406B2 (ja) 1996-07-31
US4835263A (en) 1989-05-30

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