A cure???

A few weeks ago I attended a webinar for work. The topic was: gene therapy as a treatment for Duchenne’s disease.

Gene therapy can treat diseases at the genetic level by correcting the mutation within a patient’s DNA. This can either be done by gene addition or gene editing.

With gene addition functional copies of a gene are added to compensate for a faulty gene. With gene editing things get even more sophisticated, with DNA being inserted, deleted, modified or replaced in the genome of a living organism.

So this got me thinking. Could this be a possible cure for MODY? I think it actually could. There’s only one faulty gene and if scientists could find a way to either add a functional copy of this faulty gene or replace it; I would be cured, no?

I am aware of some genetic studies going on for type 1 diabetes. But to my best knowledge, those are looking for ways to predict people at higher risk for developing t1d. These are so called epidemiological studies and not yet studies looking for gene therapy as a cure.

Unfortunately, I doubt there would be much interest in curing MODY at the moment. It is such a rare illness, with so many different types and different gene defects.. I believe we are sadly not in the front row for the development of such a treatment. So even if gene therapy research moves into higher gear, it will probably still take decades before scientists would be looking at MODY.

Dreaming is allow, so some days I do think of how I would react when doctor’s would tell me they found a cure..

Would I take it?

If it means I wound overnight have a fully functioning pancreas? Yes of course! But as with all treatment, there would no doubt be risks and side effects. For me personally I would have a good look at those first before jumping on anything.

I also belief that diabetes tech will be even better in the next years to come. So it will become easier to live with. And maybe that’s enough? I don’t know. It’s one thing to think about this stuff, but a whole other story when you’re actually put in that position, I guess.