Interview

● Interviewer: Can you tell us about the problem your research is addressing?

Certainly. The focus of my research is on neuromuscular diseases, which encompass over 140 different incurable forms, including more than 30 types of muscular dystrophy. While individually rare, these diseases are relatively common when considered as a group. The aim of our research is to discover, optimize, and translate innovative therapies for neuromuscular diseases. We are specifically focused on developing and applying new methods such as precision health and personalized genetic medicine to address genetic mutations in a precise manner. Our research combines experimental and computational approaches, such as antisense oligonucleotides, CRISPR-mediated genome editing, and machine learning, to develop next-generation therapeutics for neuromuscular and musculoskeletal diseases.

Muscular Dystrophy Association of New Zealand, 2016

● Interviewer: What are some of the significant achievements you've made so far?

One of our most significant achievements to date has been demonstrating the effectiveness of a cocktail of antisense oligonucleotides called phosphorodiamidate morpholino oligomers (PMOs) or morpholinos in restoring gene function and improving muscle strength in a severe animal model of Duchenne muscular dystrophy (DMD). We achieved this through a technique called exon skipping, which involves skipping one or more protein-coding parts of the gene called exons to mitigate the impact of mutations. This breakthrough led to a collaboration with a pharmaceutical company and the development of viltolarsen, an antisense oligonucleotide drug for treating DMD. Viltolarsen received FDA approval in 2020 after successful clinical trials conducted in multiple countries. It is the first and only FDA-approved drug that clearly restored dystrophin, a critical muscle-supporting protein lacking in DMD patients, and improved muscle function in clinical trials.

SCIENCE TRANSLATIONAL MEDICINE 2014.  6, 230fs14

● Interviewer: What initially sparked your interest in medical research?

My interest in medical research began in 9th grade when my mother experienced a subarachnoid hemorrhage. Witnessing the use of state-of-the-art technologies, such as MRI, by the doctors to accurately locate the hemorrhage and the subsequent successful surgery to save her life fascinated me. Despite not having any scientists in my family, this experience ignited my passion for medical research and the potential to make a positive impact in healthcare.

● Interviewer: What changes do you envision when your research is completed?

The ultimate goal of our research is to improve the lives of individuals living with devastating genetic disorders. By utilizing next-generation therapeutics, such as CRISPR-mediated genome editing and antisense oligonucleotides, we aim to develop effective treatments and potentially cures for these conditions. Our ongoing testing of these methods in various cell and animal models of incurable genetic diseases is paving the way for future clinical trials. When our research is completed, we anticipate the development and implementation of new therapies that could significantly enhance the quality of life for individuals affected by these disorders.

● Interviewer: Lastly, do you have any messages for students?

Absolutely. My message to students is to embrace the multitude of opportunities available at the university. There are exceptional professors, researchers, and world-class facilities that can support your growth and exploration. Seek out and seize these opportunities, and make the most of every moment. Remember that opportunities exist not only locally but also globally, so don't limit yourself. Personally, I had never traveled abroad until I was 24 years old, and I never imagined becoming a professor in Canada. Take inspiration from the words of Japanese entrepreneur Hiromasa Ezoe: "Create opportunities by yourself and change yourself through opportunities." The world is becoming increasingly interconnected, and there are limitless possibilities awaiting you. So, don't be afraid to explore, take chances, and push beyond your comfort zone. Embrace the interconnectedness of the world and the potential for growth and learning. Keep an open mind, be curious, and be proactive in seeking out opportunities that align with your interests and goals. Remember that you have the power to create your own path and make a difference in the world through your passion and dedication.

Additionally, it's important to enjoy the journey. Pursuing research and academia can be challenging at times, but remember to find joy in the process. Celebrate your achievements, both big and small, and surround yourself with a supportive community of peers and mentors who can inspire and uplift you.

In conclusion, seize the opportunities that come your way, explore the world of research with passion and determination, and embrace the transformative power of education. The possibilities are endless, and you have the potential to make a significant impact on the lives of others through your contributions to the field of medical research.

Adapted with modification from The Friends of Garrett Cumming Research & Muscular Dystrophy Canada Endowed Research Chair Annual Report 2022 and The Gateway April 2022.

Last updated: 30 June 2023