Get ANTISENSE OLIGONUCLEOTIDE MODULATION OF Raf GENE EXPRESSION - European Patent Office - EP 0863911 B1

The principle of antisense oligonucleotide (AS-OD) technologies is based on the specific inhibition of unwanted gene expression by blocking mRNA activity. It has long appeared to be an ideal strategy to leverage new genomic knowledge for drug discovery and development.

Mechanism of action of antisense oligonucleotides (ASOs): ASOs act by either causing (1) RNA cleavage or (2) RNA blockage. (1a) RNase H1 mediated cleavage, (1b) RNA interference (RNAi), (2a) Steric hindrance, and (2b) Splice modulation.

Presently, 15 oligonucleotide therapeutics have been approved in the United States of America (USA) to treat various rare diseases and 4 oligonucleotide therapeutics (eteplirsen, golodirsen, viltolarsen and casimersen) are used to treat Duchenne muscular dystrophy.

Small pieces of DNA or RNA that can bind to specific molecules of RNA. This blocks the ability of the RNA to make a protein or work in other ways. Antisense oligonucleotides may be used to block the production of proteins needed for cell growth.

Antisense RNA technology is one of the approaches that are used for the inhibition of gene expression or downregulation of a gene. This technology works on the principle that an antisense nucleic acid sequence base pairs with its complementary sense RNA strand and prevents it from being translated into protein.

Antisense oligonucleotides (AS ONs) are synthetic DNA oligomers that hybridize to a target RNA in a sequence-specific manner. They have successfully been employed to inhibit gene expression, modulate splicing of a precursor messenger RNA, or inactivate microRNAs.

These are small pieces of DNA or RNA that can bind to specific molecules of RNA. This blocks the cell's ability to use the RNA to make a protein or work in other ways. Antisense oligonucleotides are being studied in the treatment of many types of cancer.

RNA interference and antisense oligonucleotides downregulate gene expression by enzyme-dependent degradation of targeted mRNA. Steric blocking oligonucleotides block access of cellular machinery to pre-mRNA and mRNA without degrading the RNA.