Nucleic Acid Therapies for Genetic Modifications in Therapeutics

Abstract

Nucleic acid therapies work by modulating gene expression of either endogenous or invading genes. The replacement of defective genes with normal or suppressing the function of genes is a novel approach to treat genetic disorders. The production of therapeutic proteins in cell of an organism by transferring genes or cDNA for such protein into cells is referred to a gene therapy. Total suppression or reduction of the expression of undesirable proteins using defined antisense oligonucleotides. The inhibition of transcription at the level of DNA requires the inactivation of the transcription from only one or two active copies of a gene present in the genome; this is the rational for triplex inhibition of gene expression. The inhibition of protein activities and interference with their functions by specific, high-affinity binding of oligonucleotides to the proteins, this approach to therapeutic development is called Aptamer therapy. Ribozyme therapy means when an RNA precursor found to be self-splicing; because this RNA is enzymatically active, subsequently, itcleaves other RNA targets in a sequence-specific manner. The present review is focused on applications, processes and mechanism of nucleic acid therapies. This will help in understanding of nucleic acid therapies and also improve the knowledge about their applications in the field of medical sciences.