With the development of molecular biology, we understood that changes in sequence and expression of genes can cause several diseases. As such, from approaches targeting “molecules” such as therapies using small molecules and antibody therapy, a new approach targeting “cells” such as gene supplementation, gene editing, and cell transplantation have been developed.
However, for such strategies to be effective, we need to address their targetability to specific cells.
In gene therapy, we must consider unexpected side-effects on healthy cells, which may diminish a treatment approach due to safety concerns; lowering efficiency and efficacy. Despite the causes of a disease being identified and viable approaches being developed, the therapeutic applicability of such methods can be limited due to low specificity.
Furthermore, low specificity also has negative effects on the cells of a high enough purity for transplantation, limiting the usefulness of transplantation in clinical settings, as it can be difficult to guarantee that transplanted cells will not evolve into tumors.
This means there is a growing need for a technology that allows us to generate, treat and target “specific cells”.
Source) Miki et al. Efficient Detection and Purification of Cell Populations Using Synthetic MicroRNA Switches. Cell Stem Cell. 2015
RNA Switches can be employed in drug development in fields such as Regenerative Medicine and Gene Therapy.
In cell transplantation approaches, RNA Switches can be utilized in the generation of cell types difficult to generate under conventional protocol. In Gene Therapy, RNA Switches can be utilized in the fine tuning of expression of therapeutic transgene in both virus-based and mRNA-based approaches. At aceRNA Technologies we are especially focused on the development of mRNA-based drugs. By exploiting RNA design technologies, we are actively researching technologies of regulating gene expression according to cell type and phenotype, with the goal of developing innovative drugs using “Smart mRNA”. Development of smart mRNA-based therapies enable us to reduce unexpected effects of the treatment on non-target cells, thus reducing the possibility of undesired side-effects. Such drugs have a potential of enabling new revolutionary approaches for treating diseases.