Dr. Sergiu Pașcă

Of course, there are challenges with that.

Yeah, well, presumably you could inject into the brain as well, right?

There are ways in which you can inject through either surgery or through an injection in the spinal canal, like intrathecally.

So that's certainly one way in which you can do it.

It is very challenging though, because of course the brain has a lot of cell types.

And, you know, you very often, the way you deliver this, like through a virus or through other modalities, you know, there's only so much

of that virus that you can actually put inside the nervous system.

And the efficiency is not yet like very high.

So another way is to go like one level down.

So that gene will produce an RNA that will produce a protein.

So perhaps we don't have to correct the DNA everywhere, but perhaps we can correct something that happens downstream.

And that's so like being the strategy that we've been using primarily.

Just mostly because at this point, and probably in the future it will be possible, who knows, like in 10 years or maybe even earlier, we'll be able to deliver very effectively some of the genetic therapies using CRISPR.

And they're very often modified to make sure that they don't cause disease.

Of course, another limitation of that is that if the gene is really large,

It simply won't fit in a virus.

So, for instance, that would be the case if you think about a calcium channel.

Calcium channel is a gigantic gene.

It would be very difficult to fit inside a virus.

And then, of course, the other thing is like with these viruses very often, especially with the adenoviruses or AAVs, is that you have one shot.