Potential clinical applications of siRNA technique: benefits and limitations

BACKGROUND: RNA interference (RNAi) has become the method of choice for researchers wishing to target specific genes for silencing and has provided immense potential as therapeutic tools. This narrative review article aimed to understand potential benefits and limitations of RNAi technique for clinical application and in vivo studies through reading the articles published during the recent 3 years. MATERIALS AND METHODS: Medline database was searched by using 'siRNA' or 'RNAi' and 'in vivo' with limits of dates 'published in the last 3 years', language 'English' and article type 'clinical trial' for obtaining articles on in vivo studies on the use of RNAi technique. Characteristics of clinical trials on siRNA registered at the http://www.ClinicalTrials.gov were analysed. RESULTS: The only three clinical studies published so far and many in vivo studies in animals showed that the RNAi technique is safe and effective in treatment of cancers of many organ/systems and various other diseases including viral infection, arterial restenosis and some hereditary diseases with considerable benefits such as high specificity, many possible routes of administration and possibility of silencing multiple genes at the same time. Limitations and uncertainty include efficiency of cellular uptake, specific guidance to the target tissue or cell, long-term safety, sustained efficacy and rapid clearance from the body. CONCLUSIONS: RNAi technique will become an important and potent weapon for fighting against various diseases. RNAi technique has benefits and limitations in its potential clinical applications. Overcoming the obstacles is still a formidable task.