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ARISTIDE MEROLA: My name is Aristide Merola. I'm a movement disorder expert here at the Ohio State University. We are lucky to have here today Krystof Bankiewicz, who has devoted his entire life in developing gene therapy. Krys, do you want to give us an introduction about how gene therapy was born and the what are the basic principles behind that?

KRYSTOF BANKIEWICZ: Be happy to. Gene therapy is based on utilization of properties that are being carried by the viral vectors, which are based on viruses that are modified to express a single gene. In some cases, depending on the type of vector that is being utilized, one can put more than one, but typically it's just one gene that is being expressed. And then it's meant to be delivered just once into the patient's brain.

The principle behind this is really utilization of a normal function of the viruses in which these particular particles are being created, by modifying the virus and not allowing it to be replicated. So it can only infect once. It will never replicate, and in case of viral vectors that we're using right now, in patients the expression is expected to last a lifetime in those patients.