Scientists at Scripps Research Institute have developed a molecular switch that can be placed into gene therapies to enable dosing control. Their research, published in the journal Nature Biotechnology, could be the first successful technique for adjusting the activity levels of their therapeutic genes. Including this safety feature will help develop gene therapy application for the future.
Lead principal investigator, Michael Farzan, and his team showed the efficacy of the switch by incorporating it into a gene therapy that produces the hormone erythropoietin. The team successfully demonstrated they could suppress expression of its gene to very low levels with a special embedded molecule and could then increase the gene’s expression using injected control molecules known as morpholinos.
According to Farzan, the simplicity of the technique, and the fact that morpholinos are already FDA-approved, could allow the new transgene switching system to be used in a wide variety of envisioned gene therapies.