TITLE: Gene Silencing – Current Approaches and Relevance for Drug Discovery
Gene silencing is an evolving field that holds great promise for personalized drug discovery. The ability to interfere with mutated genes gives rise to the possibility of shutting down disease causing mechanisms and the advent of personalized drugs for specific diseases. The prospect of easily silencing genes in the lab for functional genomic studies has not fully crossed over into the clinical sphere. Many roadblocks are stopping this technology from being used clinically to silence mutated genes in patients. Delivery, efficiency, and effectiveness are three major problems that are being encountered when moving this technology…show more content… The idea of silencing genes has already become a major player in the study of functionalized genomics. The ability to specifically turn on or off a gene gives researchers insight into the function of specific genes with reproducibility in the lab. The field of genome sequencing has allowed disease phenotypes to be directed back to specific genes. Gene silencing research starts with determining the target gene for a specific disease phenotype. The advancement of sequencing technologies help to more easily identify specific mRNA targets associate with disease.
The two most common methods of genes silencing are ASOs and RNAi. Literature searches have led to commonalities in challenges faces when implementing gene silencing making them a useful laboratory tool but not quite an efficient therapeutic tool yet.  There are multiple disease phenotypes that have shown improvement when utilizing these methodologies, a much needed reassurance of the potential of gene silencing. There is a fear that the uncovering of more problems than solutions could to reduction of both funding and interest in this emerging field.  Overriding the current successes and the unknown of this technology is the hope that gene silencing can replace current therapies and open the door to more personalized genomic answers to disease.  The process of drug development could be simplified by starting with a specific mutated gene and developing a drug targeted to that