Gene therapy can be defined as a broad biomedical and biotechnological technique by which diseases resulting due to the inheritance of mutated or inaccurate gene sequence from the parents or the generation of a sudden mutation in the genome of a person during the course of his life can be prevented.
RNAi or RNA interference technology is one of the many techniques used to correct various hereditary or gene related disorders.
In this technique, small oligonucleotides of RNA (Ribonucleic acid) of 22 to 24 nucleotides in length are used to bind to the complementary sequence present in the mRNA (messenger RNA) molecules.This results in the generation of double stranded RNA molecules which functions in two ways:
They are recognised by RISC or RNA-induced silencing complexes, having endonuclease activity. The RISC complex cleaves and degrades the target mRNA and hence prevents the formation of the target protein.
The double-stranded RNA molecules stalls the ribosomes and prevent their progression along the mRNA molecules, hence protein synthesis in inhibited.
This technique is useful in the following ways:
Cancer often results due to the unregulated activation of the oncogenes which are responsible for promoting uncontrolled growth and division in cells. The use of RNAi can prevent the expression of the onco-proteins which are majorly responsible for causing cancer.
Alzheimer's disease is often caused due to the accumulation of misfolded amyloid beta and tau proteins in the neurons. The RNAi technology can be used to prevent the formation of the improperly folded proteins.
