Canadians take millions of prescription medications each day to manage chronic conditions such as high blood pressure, high cholesterol and Type 2 diabetes. A growing field of research, however, is moving beyond conventional pills toward therapies that operate at the cellular level. Known as nanomedicine, this approach aims to turn the body’s own cells into drug-producing units—delivering treatment from the inside out.
Nanotechnology, which involves manipulating matter at the scale of atoms and molecules—about one billionth of a metre—is already used in everyday products, from sunscreen to consumer electronics. In healthcare, its application is expanding rapidly, particularly in the development of RNA-based therapies.
Understanding Protein Production and Human Health
The human body depends on a precise balance of proteins to function properly. Structural proteins such as keratin and collagen support the integrity of hair, skin and connective tissue, while others regulate essential biological processes.
Factor VIII, for example, is a protein that enables blood clotting. People living with Hemophilia A—an inherited condition affecting thousands across Canada—do not produce enough of it, making even minor injuries potentially dangerous. Conversely, an excess of apolipoprotein C3 (ApoC3) can interfere with fat metabolism, leading to elevated triglyceride levels and increasing the risk of pancreatitis, cardiovascular disease and stroke.
Maintaining this balance is a complex biological task—one that nanomedicine is beginning to influence with increasing precision.
RNA: The Body’s Instruction Messenger
Cells function like microscopic factories. DNA, stored safely in the nucleus, contains the instructions required to produce proteins but does not leave this protected space. Instead, cells generate a temporary copy of these instructions in the form of messenger RNA (mRNA).
This mRNA travels to the cytoplasm, where ribosomes read the instructions and assemble amino acids into proteins. This process—often described as DNA → RNA → protein—is fundamental to human biology.
To ensure proteins are produced in the right amounts, the body uses regulatory mechanisms. Molecules such as small interfering RNA (siRNA) and antisense oligonucleotides (ASO) can interrupt protein production by degrading mRNA before it is translated. This acts as a built-in control system to prevent overproduction or deficiency.
RNA-Based Drugs: A New Approach to Treatment
Unlike traditional drugs that target symptoms or proteins after they are produced, RNA-based therapies act earlier—at the level of genetic instructions.
Researchers can design treatments that either increase or decrease the production of specific proteins. If a beneficial protein is lacking, synthetic mRNA can be delivered to boost production. If a harmful protein is being overproduced, siRNA or ASO molecules can silence the gene responsible.
This approach is gaining traction within Canada’s life sciences sector, with major research activity in centres such as Vancouver, Toronto and Montréal, where scientists are advancing precision medicine technologies.
Teaching Cells to Produce Missing Proteins
In conditions like Hemophilia A, the issue originates in faulty genetic instructions. A mutation in the gene responsible for factor VIII leads to the production of a defective protein—or none at all.
Scientists can now create a correct version of the mRNA blueprint in the lab. This synthetic mRNA is packaged into lipid nanoparticles—tiny fat-based carriers that protect it as it travels through the bloodstream.
Once delivered, typically through intravenous infusion, these nanoparticles enter liver cells. The cells then use the new instructions to produce functional factor VIII, effectively turning the body into its own source of treatment.
Canadian researchers, including teams at the University of British Columbia, have played a key role in developing these delivery systems, building on expertise that also contributed to mRNA vaccine technologies during the COVID-19 pandemic.
Silencing Harmful Proteins
Nanomedicine can also reduce the production of harmful proteins. In rare conditions such as familial chylomicronemia syndrome, excessive ApoC3 prevents the body from properly breaking down fats, leading to dangerously high triglyceride levels.
A recently approved injectable therapy, authorized by both the U.S. Food and Drug Administration and Health Canada, uses siRNA technology to target and destroy the mRNA responsible for producing ApoC3. The siRNA molecule binds to the matching mRNA sequence, prompting the cell to degrade it before protein production occurs.
This targeted intervention helps restore metabolic balance and lowers the risk of serious complications, including acute pancreatitis.
A New Era of Precision Medicine
Nanomedicine represents a shift in how diseases are treated—moving from broad, symptom-based approaches to highly targeted interventions at the molecular level. By directly controlling protein production, scientists can effectively adjust biological processes with precision.
For Canada’s healthcare system, which continues to manage rising rates of chronic illness and an aging population, these innovations could lead to more personalized and effective treatments.
As research advances, the concept of cells acting as their own pharmacies is becoming increasingly practical—pointing toward a future where medicine works from within the body itself.
