The use of synthetic oligonucleotides in gene therapy
is to inactivate the genes
involved in the disease process. There are several methods by which this is achieved. One strategy uses antisense specific to the target gene to disrupt the transcription
of the faulty gene
. Another uses small molecules
called siRNA to signal the cell
to cleave specific unique sequences in the mRNA transcript of the faulty gene, disrupting translation
of the faulty mRNA, and therefore expression
of the gene.
A further strategy uses double stranded oligodeoxynucleotides as a decoy for the transcription factors that are required to activate the transcription of the target gene. The transcription factors bind to the decoys instead of the promoter of the faulty gene, which reduces the transcription of the target gene, lowering expression..