ECSP066388A - Interferencia de arn doble que tiene extremos romos y modificaciones 3' - Google Patents

Interferencia de arn doble que tiene extremos romos y modificaciones 3'

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Publication number
ECSP066388A
ECSP066388A EC2006006388A ECSP066388A ECSP066388A EC SP066388 A ECSP066388 A EC SP066388A EC 2006006388 A EC2006006388 A EC 2006006388A EC SP066388 A ECSP066388 A EC SP066388A EC SP066388 A ECSP066388 A EC SP066388A
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EC
Ecuador
Prior art keywords
aryl
alkyl
modifications
rna interference
romes
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Application number
EC2006006388A
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English (en)
Inventor
Jan Weiler
Jonathan Hall
Jean-Charles Bologna
Francois Jean-Charles Natt
Robert Haener
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Novartis Ag
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Publication date
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Publication of ECSP066388A publication Critical patent/ECSP066388A/es

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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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
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  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
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Abstract

La presente invención se refiere a compuestos de ARN de estructura de cadena doble con por lo menos un extremo romo que comprende por lo menos un extremo 3' de la Fórmula (I): en donde X es O o S, R1 y R2 son independientemente OH, NH2, SH, alquilo, arilo, alquil-arilo, aril-alquilo, en donde el alquilo, arilo, alquil-arilo, aril-alquilo pueden estar substituidos por átomos heterogéneos adicionales y grupos funcionales, preferiblemente un átomo heterogéneo seleccionado del grupo de N, O, o S o un grupo funcional seleccionado de OH, NH2, SH, ácido o éster carboxílico; o, R1 y R2 pueden ser de la fórmula Y-Z en donde Y es O, N, S y Z es H, alquilo, arilo, alquil-arilo, aril-alquilo, en donde el alquilo, arilo, alquil-arilo, aril-alquilo pueden estar substituidos por átomos heterogéneos adicionales, preferiblemente un átomo heterogéneo seleccionado del grupo de N, O, o S; y en donde dicho ARN de estructura de cadena doble media la interferencia de ARN. Las modificaciones del extremo 3' preferidas son: fosfato, fósforotioato, ribonucleósido abásico, fósforodiester de hidroxipropilo.
EC2006006388A 2003-08-28 2006-02-22 Interferencia de arn doble que tiene extremos romos y modificaciones 3' ECSP066388A (es)

Applications Claiming Priority (1)

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

Publications (1)

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ECSP066388A true ECSP066388A (es) 2006-08-30

Family

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Family Applications (1)

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EC2006006388A ECSP066388A (es) 2003-08-28 2006-02-22 Interferencia de arn doble que tiene extremos romos y modificaciones 3'

Country Status (21)

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

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AU2004269150B2 (en) 2009-06-04
US8097716B2 (en) 2012-01-17
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MXPA06002216A (es) 2006-04-27
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RU2399671C2 (ru) 2010-09-20
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NO20061345L (no) 2006-05-29
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IL173565A (en) 2012-07-31
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