The search for COVID-19 vaccines have recently put the concept of mRNA technology as central for the treatment of diseases. It has provided evidence based proof that this emerging branch of medicine offers a unique viable way to save lives by preventing emerging infectious diseases. With this discovery, the gateway to mRNA research and development has seen a tremendous surge and also has accelerated in the process.

Now, scientists and researchers globally are looking to leverage and harness mRNA for a greater purpose: treating chronic illnesses hitherto unknown before.

Over the past decade, mRNA –messenger ribonucleic acid has emerged as a promising next-generation technology for creating a new class of medications to treat cystic fibrosis, heart disease, rare genetic conditions, and even some cancers.

Cells in our body use mRNA to translate the genes of our DNA into dynamic proteins which are involved in virtually every bodily function and health condition. Biotech companies make some of these proteins as drugs, using genetically engineered cells in factories.

In theory, mRNA therapies may be used to prompt proteins to be made in your body — in effect putting the drug factory inside you, says Moderna President Stephen Hoge, MD.

mRNA and how it works

What is mRNA, and How Does it Work?

Essentially, mRNA is a single-stranded molecule, naturally present in all of our cells. Identified in 1961, mRNA carries instructions to cells for making proteins from our genes that are used as building blocks of their fundamental structures and functions.

A segment of DNA gets copied into a piece of mRNA that gives our cells commands for making specific proteins, some of which can cause or prevent disease.

The COVID-19 vaccines, for example, contain the coronavirus’s mRNA (which cannot cause infections). When injected into muscle, our cells “read” it and synthesize the non-infectious “spike protein” found on the surface of the virus.

That prompts our immune systems to create these harmless proteins and develop the tools needed to target and kill the virus if and when we become infected .

MRNA technology is not new. Researchers have been delving into the mysteries of mRNA — and unravelling the role it might play in diseases — for decades.

The reason mRNA holds such promise is that it combats disease in an entirely different way than most drugs on the market which has been discovered only now.

Hundreds of clinical trials are in various stages of testing mRNA as a way to prevent or treat infections and chronic diseases according to clinicaltrials.gov which is the federal clearinghouse for ongoing studies worldwide.

What does mRNA do? mRNA produces instructions to make proteins that may treat or prevent disease.

Unlike traditional pharmaceuticals, mRNA medicines aren’t small molecules. Neither are they recombinant proteins and monoclonal antibodies like traditional biologics. Instead, mRNA medicines are sets of instructions that direct the cells in out body to activate gene expression and make proteins to prevent or fight disease.

It’s actually basic human biology. 

DNA (deoxyribonucleic acid) is a double-stranded molecule that stores the genetic instructions your body’s cells need to make proteins. Proteins, on the other hand, are the ‘workhorses’ of the body. Nearly every function in the human body – both normal and disease-related – is carried out by one or many proteins.

mRNA is just as critical as DNA.

Without mRNA, your genetic code will never get used by your body. Proteins will never get made. And your body will not be able to perform its functions. Messenger ribonucleic acid, or mRNA for short, plays a vital role in human biology, specifically in a process known as protein synthesis. mRNA is a single-stranded molecule that carries the genetic code from your DNA in a cell’s nucleus to ribosomes, the cell’s protein-making machinery.

mRNA’s role in protein synthesis

mRNA’s role in protein synthesis
Image Credit: https://www.modernatx.com
  1. Through a process known as transcription, an RNA copy of a DNA sequence for creating a given protein is made.
  2. This copy – mRNA – travels from the nucleus of the cell to the part of the cell known as the cytoplasm, which houses ribosomes. Ribosomes are complex machinery in the cells that are responsible for making proteins.
  3. Then, through another process known as translation, ribosomes ‘read’ the mRNA, and follow the instructions, creating the protein step by step. 
  4. The cell then expresses the protein and it, in turn, carries out its designated function in the cell or the body. 

Overcoming Key Challenges

Using mRNA to create medicines is a complex undertaking and requires overcoming novel scientific and technical challenges.  Not only does the mRNA need to get into the targeted tissues and cells, it also needs to do so by evading the immune system. If the immune system is triggered, the resultant response may limit protein production and, thus, limit the therapeutic benefits of mRNA medicines. Ribosomes are also required to be somehow programmed to think that the mRNA was produced naturally, so they can accurately read the instructions to produce the right protein. Finally, in order to achieve the desired therapeutic effect, the stimulated cells need to express enough of the protein.