Small RNA

RNA editing cargo is small enough to get into cells without the need of viral or non-viral approaches. It can be formulated in solution and chemically modified to protect it from degradation. Further chemical modifications enhance its stability and extend the half-life of the cargo. This approach is not a one and done and we expect dosing would need to happen several times a year. Alternatively, small RNAs can also be delivered with a medical device for continuous delivery. Micro pumps exist for intracranial or intrathecal delivery which can manage a sustained concentration in spinal fluid.