Research is increasingly demonstrating that genetic mutations play a significant role in the development of ALS: for example, the C9orf72 gene is one of the most prominent genetic causes of the disease.

With the scientific community abuzz about CRISPR/CAS9, a state-of-the-art technology that enables scientists to precisely manipulate and edit genes in living organisms, the application of this technique to ALS is important to explore. That’s what Dr. Gary Armstrong, a researcher at the Montreal Neurological Institute and McGill University, is doing thanks to funding from the ALS Canada Research Program. While it’s too soon for this technology to be used in humans, he is using CRISPR/CAS9 to create new zebrafish models of ALS with mutations in the C9orf72 gene. At the recent ALS Canada Research Forum, Dr. Armstrong told us more about his research as well as some clinical trials underway that hold a lot of promise.

Could you tell us a bit about your research?

Many of the people who might be watching this might have heard the technology, the CRISPR-Cas9 technology, the CRISPR mutagenic system. So what this is, is the ability to go in and edit the genome of—whether it’s a tissue in culture, so single cells, or in the animal models that we work with.

Why is this helpful for researchers?

That’s really useful for ALS researchers, because we can go in and we can make the exact same mutation, and then study it in an animal model and do things in an animal model that you just can’t, obviously, do in a patient. That’s the funding technology that ALS Canada has given me, and I’m very grateful for it.

How will this technology help people living with ALS?

If you can make that mutation in an animal model, why can’t you go to a human being with ALS and fix that mutation? And that is a wonderful goal. That would be a wonderful goal, because that has a chance to actually, possibly, provide a cure for the disease. The reality is, is that we’re not there yet. This technology needs to be refined.

What ALS research looks the most promising?

There’s a clinical trial going on that’s targeting SOD-1, and there’s another one for patients who have c9orf72 repeats. And I think that is going to be, really, the big success story in the next few years for ALS.

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