How gene editing tools could one day cure rare and debilitating diseases

Meet Dr. Soh Ishiguro

How gene editing tools could one day cure rare and debilitating diseases.

Dr. Soh Ishiguro is a postdoctoral fellow at UBC’s School of Biomedical Engineering.

Most genetic diseases are caused by tiny mutations in a person’s DNA. Dr. Soh Ishiguro, a postdoctoral fellow at the UBC School of Biomedical Engineering, is working on new genome editing tools that could one day cure rare and debilitating diseases from blood disorders to heart disease.

What is the focus of your research?

We study how single cells form complex organisms as they develop into organs and tissue structures. This helps us understand the cell’s behaviours, which in turn helps us better understand animal development and human diseases. I am also developing new genome editing tools using the CRISPR–Cas9 system.

This precise CRISPR–Cas9 DNA base editing tool allows us to emulate diseased tissue structures in pre-clinical models to study what’s happening in severe genetic diseases. We know that most human diseases occur due to point mutations, which are errors in single DNA nucleotides. By using this technology, we can install specific substitutions into the target gene. Then we can potentially cure the disease associated with that mutation.

Why is your research important?

The big goal is to make a universal DNA base editing technology. This will allow us to precisely install any type of DNA substitution so we can study human gene function. That could help us cure specific human genetic diseases associated with single DNA changes such as sickle-cell anemia, a disorder of the red blood cells, and Duchenne muscular dystrophy. Several critical trials of the CRISPR base editors have already begun.

Dr. Soh Ishiguro

If your research is successful what might the result look like?

More than 60 per cent of variations associated with human genetic disorders are in a single DNA change. So, if we develop a universal base editing technology tool, we could cure over 60 per cent of genetic diseases. This is a promising approach, especially because some genetic diseases are very rare, and both difficult and expensive to treat with more conventional therapies.

We hope through iterations in development and testing — using a synthetic biology approach, including protein engineering and screening — we can create these tools and use them to find potential drugs for gene therapy.

“If we develop a universal DNA base editing technology tool, we could cure over 60 per cent of genetic diseases.”

– Dr. Soh Ishiguro

How do you hope your work could accelerate translational medicine for the future?

I believe that the power of technology will develop our understanding of protein and gene functions in the future.

The first step is to translate these basic research technologies — working in collaboration with researchers in clinical settings — into practical settings like hospitals. This will allow us to receive important feedback that will ultimately optimize new technologies. Down the road, we hope this will directly benefit patients in the community.

Why did you choose UBC?

When I was working on my PhD in Japan my supervisor was Dr. Nozomu Yachie. After I finished my PhD, my supervisor moved his lab to UBC when he was awarded the Canada Research Chair in Synthetic Biology. I thought moving to UBC was an incredible opportunity for me because it provided the opportunity to expand my research in collaboration with many students and researchers. I was looking to push my science forward and coming to UBC was a great opportunity to do that.