Dr. Sergiu Pașcă

So I think some of these genetic mutations are sort of like the same.

So gene therapy is a rather actually broad term, and it covers many ways in which you can correct generally a gene or a genetic defect that we think it's causal.

So on one extreme, for instance, you can envision a gene is broken, has a mutation.

So what you want to do is you want to put it back.

So those were some of the early efforts where you would put it in a virus and deliver it to the patient.

An adult.

In an adult or in a child, depending on like the condition.

With the idea is that the gene is not there or like there's not enough of it.

So I'm just going to deliver more.

That's one extreme.

Many of the studies were done for blood disorders, of course, because it was easier, so you would inject them.

Of course, the other possibility is sometimes you don't want to put the gene, you want to put the protein already made.

And that is the case for many conditions where an enzyme, so a protein that, you know, does some interesting chemical reactions that are essential to a cell is missing.

So sometimes you just make that enzyme and then you deliver that.

It's not always working, but in some cases actually works really well.

Now, the other thing that you can do is you can try to correct that defect directly.

That means you need to operate at the DNA level.

So somehow you need to get into every single cell that is affected and correct that.

And that's where CRISPR comes into play, where presumably you could at one point deliver the guides, so the tiny pieces of nucleic acid that tell you where to go on the DNA.

And then an enzyme, they will do the cutting and then the putting back or various other versions of this that you would correct.