Composición farmacéutica que comprende oligonucleótidos antisentido anti-ARNmi

• Main Authors: Kearney, Phil, Elmén, Joacim, Kauppinen, Sakari
• Format: Patent
• Language: Spanish
• Published: 11.05.2016
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; DERIVATIVES THEREOF; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NUCLEIC ACIDS; NUCLEOSIDES; NUCLEOTIDES; ORGANIC CHEMISTRY; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS; SPIRITS; SUGARS; VINEGAR; WINE

Oligonucleótido de fórmula: 5'-CcAttGTcaCaCtCC-3' (SEC ID nº 99) en la que una letra minúscula identifica la base nitrogenada de una unidad de ADN; una letra mayúscula identifica la base nitrogenada de una unidad de ANB y en la que las citosinas de ANB se encuentran opcionalmente metiladas. The invention provides oligonucleotides 8 - 22 nucleotides in length, for inhibition of a microRNA target in a cell, which comprise a high proportion of high affinity nucleotide analogue units as well as LNA units. The oligonucleotides are particularly effective at alleviating miRNA repression in vivo. It Is found that the incorporation of high affinity nucleotide analogues Into the oligonucleotides results in highly effective anti-mlcroRNA molecules which appear to function via the formation of almost irreversible duplexes with the miRNA target, rather than RNA cleavage based mechanisms, such as mechanisms associated with RNaseH or RISC,