A class of miniature proteins is making big waves in medicine and science. These microproteins are revolutionising our understanding of a wide variety of fields including cell biology, ageing, DNA repair, developmental biology, immunology and cancer biology. And it looks like it won’t be long before groundbreaking research on microproteins leads to a new wave of therapeutic developments.
“Microproteins are a rapidly expanding class of small proteins that have reshaped our understanding of proteome composition and revealed new biological pathways through their characterization,” said Dr Annie Rathore, the lead author on a new paper devoted to human microproteins. Defined as proteins that contain less than 100 amino acids, microproteins were only discovered in 2011 when they were found in the humble house fly. A lack of appropriate scientific methods of detection coupled with their small size kept these proteins hidden but thankfully cutting-edge genomic and proteomic technologies have now allowed researchers to discover an entire class of these tiny proteins across a variety of organisms, including humans.
There may have been some doubt about their biological importance in the beginning. But it didn’t take long for scientists to begin to appreciate just how vital these proteins can be to a range of biological process and that they might be just as important to our health as their larger cousins. For instance, research published in 2017 showed that a microprotein called CYREN has a fundamental role in DNA repair – a process with close ties to cancer. The same paper shows evidence that microproteins can bind to larger proteins in order to regulate biological processes. This opens the door for drug development based on chemicals that inhibit the action of the microproteins rather than the larger ‘main’ proteins.
“Overall, microprotein research has so far proven valuable in revealing new genes and biological mechanisms for the regulation of significant processes, while also expanding the limits of what was considered a typical gene or protein. Just as the discovery of DNA, RNA and proteins revolutionized the pharmaceutical industry in the past decades, uncovering the role of microproteins in human health and disease has the potential to lead a new wave of therapeutics developments in the foreseeable future,” said Dr Rathore.
So little is currently known about these tiny proteins as researchers have mostly confined their efforts to discovering them. No doubt, the focus will soon shift to developing methods of identifying microproteins that contribute to biological processes in some way and in particular, to discovering the role of specific microproteins in human disease.
If you want to learn more about microproteins, Dr Rathore’s paper Small, but mighty? Searching for human microproteins and their potential for understanding health and disease is a great place to start.
Dr Annie Rathore is a research scientist conducting groundbreaking research into microproteins. She was one of the youngest scientists to earn a PhD from the Salk Institute of Biological Studies. You can connect with her or follow her work through LinkedIn.