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Derivatives of small interfering rnas and use thereof

Other TitlesDerivados de pequeños arn de interferencia y su uso
AuthorsOcampo, Sandra M. ; Eritja Casadellà, Ramón ; Romero, Carolina; Burgueño, Joan; Fernandez, Ester; Perales, José Carlos
Issue Date10-May-2012
CitationWO2012059612 A1
Abstract[EN]The present invention relates to a small interfering RNA (siRNA) specific for tmnour necrosis factor-alpha (TNFa), being 2'-0-methyl modified and there being attached thereto by a phosphodiester bond a group R in position 3', wherein R is selected from among: a C¡-C6 alkyl substituted with at least one -OH group, in particular propanediol; a C4-C6 cycloalkyl substituted with at least one -OH group; a C4-C6 heterocycloalkyl; a heteroalkyl chain having between 2 and 7 atoms, wherein at least one thereof is an oxygen atom or a sulphur atom, the chain preferably being substituted with at least one -OH group; and a heteroaryl, the ring whereof being formed of between 3 and 6 atoms and at least one of said atoms being an oxygen atom. The present invention furthermore refers to the use of said siRNA for the treatment of inflammatory diseases, preferably inflammatory bowel disease. DRA WING: FIGs. lA and lB: s.m no medication.
[ES] La presente invención se refiere a un pequeño ARN de interferencia (pARNi) específico frente al factor de necrosis tumoral alfa (TNF-a), con modificaciones 2'-0-metilo y que lleva unido mediante un enlace fosfodiéster un grupo R a la posición 3', donde R se selecciona entre: un C¡-C6 alquilo sustituido con al menos un grupo -OH, en especial propanodiol; un C4 - C6 cicloalquilo sustituido con al menos un grupo - OH; un C4-C6 heterocicloalquilo; una cadena heteroalquílica entre 2 y 7 átomos, donde al menos uno es un átomo de oxígeno o un átomo de azufre, preferiblemente la cadena está sustituida con al menos un grupo.
Appears in Collections:(IQAC) Patentes
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