MA28029A1 - Double helice d'arn d'interference possedant des extremites franches et des modifications en 3' - Google Patents

Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'

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Publication number
MA28029A1
MA28029A1 MA28861A MA28861A MA28029A1 MA 28029 A1 MA28029 A1 MA 28029A1 MA 28861 A MA28861 A MA 28861A MA 28861 A MA28861 A MA 28861A MA 28029 A1 MA28029 A1 MA 28029A1
Authority
MA
Morocco
Prior art keywords
aryl
alkyl
propeller
changes
double
Prior art date
Application number
MA28861A
Other languages
English (en)
Inventor
Jan Weiler
Jonathan Hall
Jean-Charles Bologna
Francois Jean-Charles Natt
Robert Haener
Original Assignee
Novartis Ag
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Publication date
Application filed by Novartis Ag filed Critical Novartis Ag
Publication of MA28029A1 publication Critical patent/MA28029A1/fr

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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04—Antibacterial agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/311—Phosphotriesters
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

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  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Apparatus For Disinfection Or Sterilisation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)

Abstract

La présente invention porte sur des composés d'ARN double brin pourvus d'au moins une extrémité franche comprenant au moins une extrémité 3' de formule (I). dans laquelle X représente O ou S; R1 et R2 représentent, indépendamment, OH, NH2, SH, alkyle, aryle, alkyl-aryle, aryl-alkyle, où alkyle, aryle, alkyl-aryle, aryl-alkyle peuvent être substitués par des hétéro-atomes additionnels et des groupes fonctionnels; de préférence, un hétéro-atome est sélectionné dans le groupe de N, O ou S ou un groupe fonctionnel est sélectionné dans le groupe OH, NH2, SH, l'acide carboxylique ou un ester; ou R| et R2 peuvent être de la formule Y-Z dans laquelle Y représente O. N, S et Z représente H, alkyle, aryle, alkyl-aryle, aryl-alkyle, où alkyle, aryle, alkyl-aryle, aryl-alkyle peuvent être substitués par des hétéro-atomes additionnels, de préférence un hétéro-atome sélectionné dans le groupe de N, O ou S. Dans la formule de cette invention, l'ARN double brin induit une interférence ARN. Les modifications préférées à l'extrémité 3' sont: phosphate, phosphorothioate, ribonucléoside abasique. hydroxypropyle, phosphodiester.
MA28861A 2003-08-28 2006-03-08 Double helice d'arn d'interference possedant des extremites franches et des modifications en 3' MA28029A1 (fr)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US49851403P 2003-08-28 2003-08-28

Publications (1)

Publication Number Publication Date
MA28029A1 true MA28029A1 (fr) 2006-07-03

Family

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Family Applications (1)

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MA28861A MA28029A1 (fr) 2003-08-28 2006-03-08 Double helice d'arn d'interference possedant des extremites franches et des modifications en 3'

Country Status (21)

Country Link
US (2) US20070203084A1 (fr)
EP (3) EP2338995A3 (fr)
JP (2) JP2007503803A (fr)
KR (3) KR101059315B1 (fr)
CN (2) CN1845993B (fr)
AU (1) AU2004269150C1 (fr)
BR (1) BRPI0413146A (fr)
CA (1) CA2536333C (fr)
CO (1) CO5680494A2 (fr)
EC (1) ECSP066388A (fr)
IL (1) IL173565A (fr)
IS (1) IS8358A (fr)
MA (1) MA28029A1 (fr)
MX (1) MXPA06002216A (fr)
NO (1) NO20061345L (fr)
NZ (1) NZ545360A (fr)
RU (2) RU2399671C2 (fr)
SG (1) SG152279A1 (fr)
TN (1) TNSN06065A1 (fr)
WO (1) WO2005021749A1 (fr)
ZA (1) ZA200601003B (fr)

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NO20061345L (no) 2006-05-29
ZA200601003B (en) 2007-04-25
NZ545360A (en) 2009-06-26
BRPI0413146A (pt) 2006-10-03
CA2536333C (fr) 2013-01-08
RU2010119292A (ru) 2011-12-10
CO5680494A2 (es) 2006-09-29
US20090192113A1 (en) 2009-07-30
CN1845993A (zh) 2006-10-11
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JP2007503803A (ja) 2007-03-01
KR101059315B1 (ko) 2011-08-24
AU2004269150C1 (en) 2010-11-18
RU2006109547A (ru) 2007-10-10
EP2338995A3 (fr) 2012-01-11
TNSN06065A1 (en) 2007-10-03
KR20080034046A (ko) 2008-04-17
US20070203084A1 (en) 2007-08-30
EP1660657A1 (fr) 2006-05-31
IL173565A (en) 2012-07-31
IL173565A0 (en) 2006-07-05
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US8097716B2 (en) 2012-01-17
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AU2004269150A1 (en) 2005-03-10
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SG152279A1 (en) 2009-05-29
RU2399671C2 (ru) 2010-09-20
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WO2005021749A1 (fr) 2005-03-10
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