8-1.3.3 Targeted Inhibition of Gene Expression

This is to block the expression of any diseased gene or a new gene expressing a protein which is harmful for a cell. This is particularly suitable for treating infectious diseases and some cancers.

Figure 8-1.3.3: To inhibit the target gene expression in diseased cell the antisense mRNA coding gene inserted vector or triplex-forming oligonucleotides (TFO) or antisense oligonucleotide (ODN) can be introduced which will inhibit the gene expression either by forming DNA:RNA triplex inside the nucleus or forming RNA:RNA duplex by forming complementary mRNA strand of disease protein coding mRNA. This may lead to blocking of disease causing protein expression.

8-1.3.4 Targeted Gene Mutation Correction

It is used to correct a defective gene to restore its function which can be done at genetic level by homologous recombination or at mRNA level by using therapeutic ribozymes or therapeutic RNA editing.