ATE167680T1 - Pteridin-nukleotidderivate als fluoreszierende sonden - Google Patents

Pteridin-nukleotidderivate als fluoreszierende sonden

Info

Publication number
ATE167680T1
ATE167680T1 AT95917197T AT95917197T ATE167680T1 AT E167680 T1 ATE167680 T1 AT E167680T1 AT 95917197 T AT95917197 T AT 95917197T AT 95917197 T AT95917197 T AT 95917197T AT E167680 T1 ATE167680 T1 AT E167680T1
Authority
AT
Austria
Prior art keywords
pteridine
fluorescent probe
fluorescent
nucleotide derivatives
pteridine nucleotide
Prior art date
Application number
AT95917197T
Other languages
English (en)
Inventor
Mary E Hawkins
Wolfgang Pfleiderer
Michael Dean Davis
Frank Balis
Original Assignee
Us Gov Health & Human Serv
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 Us Gov Health & Human Serv filed Critical Us Gov Health & Human Serv
Application granted granted Critical
Publication of ATE167680T1 publication Critical patent/ATE167680T1/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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
    • C07H19/02Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
    • C07H19/04Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
    • C07H19/22Pteridine radicals

Landscapes

  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Saccharide Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AT95917197T 1994-05-18 1995-04-25 Pteridin-nukleotidderivate als fluoreszierende sonden ATE167680T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US08/245,923 US5525711A (en) 1994-05-18 1994-05-18 Pteridine nucleotide analogs as fluorescent DNA probes

Publications (1)

Publication Number Publication Date
ATE167680T1 true ATE167680T1 (de) 1998-07-15

Family

ID=22928654

Family Applications (1)

Application Number Title Priority Date Filing Date
AT95917197T ATE167680T1 (de) 1994-05-18 1995-04-25 Pteridin-nukleotidderivate als fluoreszierende sonden

Country Status (10)

Country Link
US (2) US5525711A (de)
EP (1) EP0759927B1 (de)
JP (2) JPH10500949A (de)
AT (1) ATE167680T1 (de)
AU (1) AU688036B2 (de)
CA (1) CA2190588C (de)
DE (1) DE69503129T2 (de)
DK (1) DK0759927T3 (de)
ES (1) ES2118593T3 (de)
WO (1) WO1995031469A1 (de)

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US5525711A (en) 1996-06-11
EP0759927B1 (de) 1998-06-24
EP0759927A1 (de) 1997-03-05
JP2009091358A (ja) 2009-04-30
WO1995031469A1 (en) 1995-11-23
DE69503129D1 (de) 1998-07-30
CA2190588A1 (en) 1995-11-23
US5612468A (en) 1997-03-18
JPH10500949A (ja) 1998-01-27
CA2190588C (en) 2003-03-18
AU2399195A (en) 1995-12-05
DE69503129T2 (de) 1999-02-18
DK0759927T3 (da) 1999-04-06
AU688036B2 (en) 1998-03-05

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