October 13, 2023

Developing a “weed killer” to treat a type of muscular dystrophy

A WCHRI student is researching a DNA-like molecule to prevent muscle damage.

By Luis Fernando Rubio Atonal*

Saeed Anwar is currently a PhD student in the Department of Medical Genetics.

Tell us a bit about your research.

I am working on developing new treatments for muscle-related genetic diseases. My research aims to tackle a complex genetic condition called facioscapulohumeral muscular dystrophy, or FSHD, where muscles of the face, shoulder blades and upper arms are affected by weakness and atrophy. FSHD, one of the most common forms of muscular dystrophies, involves an intricate interplay between genes and proteins in muscle cells. At the heart of the disease, there is a misexpression of a toxic protein called DUX4, which you can think of as a weed. Healthy muscle cells do not produce DUX4; in FSHD, this protein is produced, resulting in the death of the muscle cells. My goal is to develop a “weed killer”. This weed killer I’m making is a type of synthetic DNA-like molecule called antisense gapmer. Gapmers specifically target and stop the production of DUX4 and prevent it from damaging the muscle.

What led you to pursue research in genetics, particularly in musculoskeletal disorders?

It’s a combination of personal experience and my early academic journey. As a kid, I experienced firsthand the challenges of living with rheumatic diseases and the regular medications and consistent hospital visits — it shaped my early life. Fast forward to my biotechnology undergraduate years at Shahjalal University in Bangladesh where my interest took a deep dive into genetic disease research. What struck me the most was the burden borne by individuals and families affected by genetic diseases. The emotional and physical struggles of those people with inherited disorders resonated deeply with me. Among those people, I saw many people with musculoskeletal disorders. I became motivated to explore potential therapeutic interventions for genetic diseases, especially musculoskeletal disorders. 

Why did you choose Canada and Toshifumi Yokota’s lab to pursue your research interests?

I set foot in Canada at the University of Alberta in the fall of 2019. It was the first time I stepped out of Bangladesh. I enrolled in a master’s program under the Maternal and Child Health Scholarship (MatCH) program. Canada’s reputation for world-class research institutions and innovative medical advancements was a major draw for me. I was eager to join an environment that not only emphasized innovative research but also had the commitment to improve the lives of individuals with genetic disorders. Toshifumi Yokota’s lab was the epitome of this to me, making it an obvious choice for my research.

How has WCHRI funding supported your research and career as a graduate student?

WCHRI is one of the partners that supports the MatCH program. Later on, I received a WCHRI Graduate Studentship Award, so WCHRI’s support has been a lifeline throughout my academic journey in Canada. Thanks to the support of the Stollery Children’s Hospital Foundation through WCHRI, I’ve been able to engage in my research without any financial stress. More than that, being a recipient of these prestigious awards has given me recognition in the academic community. I think it will undoubtedly open doors for my future endeavours in the field of medical genetics.

What are the challenges you’ve encountered as an international student at the University of Alberta?

Starting grad school, as an international student from Bangladesh, was like diving into a completely new world. Like with any other student, there was academic pressure, but then layering on adapting to Canadian culture and missing the comfort of my family and hometown was really challenging. What catches people off guard about my experience is that I found a kind of solace in Edmonton’s famous winters. I saw it as a beautiful snowy wonderland, which was a sharp contrast to the heat and monsoons of my homeland. There’s a magic to it, I would say. I am an author, so I like to find a really good place to sit and enjoy the stillness of the snow and watch the Northern Lights that often paint different kinds of beauty in front of me.

Another challenge is that I was in Edmonton for the entire pandemic. This made being away from home a bit tougher. Yet the warmth and support from the University of Alberta community, even in the coldest times, made all the difference. 

What’s one piece of advice you received from your supervisor/mentor that still sticks with you?

When I was contemplating the transition from a master’s to a doctorate program, my supervisor shared a piece of wisdom: it is not just a degree, it’s your passport to international recognition. As someone passionate about both research and writing, the idea of gaining a global voice and impact through a PhD has been incredibly motivating and feels like a great responsibility. 

Saeed Anwar is supervised by Toshifumi Yokota. His research is funded by the Stollery Children’s Hospital Foundation through WCHRI.

*Luis Fernando Rubio Atonal is a member of WCHRI’s Trainee Advisory Committee (TAC). Additional members from TAC who contributed to this story include editors Marina Giovannoni and Kaya Persad, with videography and photography by Robert Mcweeny.