Dr. Sergiu Pașcă

So for instance, for Timothy syndrome, there is a very dramatic effect

in the size of the neurons.

They're almost twice as smaller than a control neuron.

In the patient.

Well, in the patient.

Only when you transplant the cells, we can see that defect.

In a dish, you look at them and they're identical.

And then you transplant them and some of them grow really large to control and the patients fail.

And that phenotype can only really be seen properly in vivo.

So that has been actually essential also as we've been developing a therapeutic for this condition.

And you start thinking, like, how do you test a therapeutic?

You know, if there's no animal model of the disease, you test everything in a dish.

You do want to have some safety check, first of all, for making sure that there are no adverse effects, but also you want to make sure that it works in an in vivo environment.

And actually, it turns out that this model that we've built was essential because now we could take actually the animal.

and inject the therapeutic into the nervous system of the animal, but look at the effect on human neurons in an in vivo context.

And, you know, so I think that's one application for this.

But if you do the transplantation at a later stage, like for instance in an adult, that integration would probably not happen.

I see.

So it's quite dependent on the species.

And there's another thing.