RNAi has the potential to have direct therapeutic benefits by silencing deleterious genes in cancer and other diseases. It provides a means to investigate the role of an individual gene in a cell and allows researchers to examine the mechanisms of disease, which can lead to the development of new treatment strategies. Research into the endogenous miRNA mechanism and RNAi continue as biologists believe there is still much to be learned about both processes. Both miRNA and RNAi prevent gene expression and provide control over cell proliferation, cell death, and fat metabolism in flies, neuronal patterning in nematodes, modulation of hematopoietic lineage differentiation in mammals, and control of leaf and flower development in plants. In fact, a 2005 Nature article suggested that analyzing expression of 217 miRNAs, as opposed to 16,000 mRNAs, can better define human cancers.

The expected benefits of a technology like RNAi need to be weighed against the possible harm its misuse could cause. This presents a significant challenge to the scientific community to balance the openness of research with the vigilance required to be responsible for technology and knowledge that could be misused. Scientists need to be aware of the possible dual-use concerns posed by their work and learn to address them effectively so that important research can still move forward.