Nobody took issue with describing mRNA as a form of "gene therapy" prior to 2020 as the article below clearly illustrates.
It was only when mRNA vaccines burst onto the scene with the potential for untold riches for many that "experts" suddenly became pedantic over the use of the term.
mRNA therapy: A new form of gene medicine
https://medium.com/@hareth.wassiti?source=post_page-----5d859dadd1e--------------------------------
Harry Al-Wassiti
Dec 11, 2019 · 5 min read
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It has been several weeks since I came back from Berlin. I was visiting the international mRNA health conference, where I presented our latest development in the mRNA therapy field. Unlike the USA and Europe, mRNA research in Australia is still a young concept. As far as we know, we are probably one of the only groups in Australia who are actively working on mRNA therapy. On my way back to Australia thinking of all the great discoveries I just witnessed in the conference- I had a thought. I must write about mRNA therapy, so more people can know about it. Because when mRNA therapy reaches its age, it will touch most of us.
So what are mRNA and gene therapies and how do they work?
To understand this new class of therapies, we must first understand what makes up mRNA and gene therapies. These molecules are known as nucleic acids. In our cells, there are two major types of nucleic acid molecules: Genomic DNA and mRNA. These carry the manual for all the instructions your cells and body need. They are the code for life. The basic principle of traditional gene therapy is to insert or add a
DNA molecule, with specific “designed” instructions and is “translated” by your cell as a protein. In the case of gene therapy, it treats people who lack that protein. mRNA, also known as
messenger RNA, is naturally found in all our cells. mRNA is responsible for carrying out a message from the DNA that lies inside the nucleus to the cytoplasm. In there, the proteins are made from the mRNA sequence in a process known as translation. So
mRNA therapy uses mRNA molecules instead of DNA molecules, thereby bypassing the need for DNA and simplifying the process (See the figure below).
Nucleic acid therapy, whether it is derived from mRNA or DNA, is a transformational concept in medicine. In contrast to conventional drugs- small molecules acting on a protein or a “target” inside your body- nucleic acid therapy instead
instruct your body to make or break the proteins inside your cells at a more fundamental level. You could imagine your DNA and mRNA as the operating program system (OS) for life. Much like computer OS, mRNA therapy can reprogram your body to produce its own therapies. An intriguing concept, indeed.
And what are the uses of mRNA therapy?
Back to Berlin’s conference, many academic and biotech groups presented a wide range of medical applications using mRNA. We and others have envisaged, many times, the range of applications mRNA therapy can have.
Given the mRNA inherent “programmability”, it’s relatively easy to adjust the sequence of mRNA to make, theoretically, any therapeutic protein. This means it can cover a wide range of diseases. Examples of applications that have gone into clinical trials (i.e. trialled in a small number of humans) are cancer immunotherapy, viral vaccines and enzyme replacement therapy for the liver. But the list, as we all expect, won’t stop here. In the context of the human application, many groups and biotech companies presented impressive findings, but these results are still early.
Why is Biotech building mRNA tech?
Effectiveness: Conventional DNA gene therapy needs to overcome many challenges before it becomes a therapeutically viable option. For DNA gene therapy to be active, it requires to reach not only the cell but the very nucleus inside that cell. This is an incredibly difficult task given that our nuclei have evolved to prevent any foreign DNA from entering (Think viruses!). While mRNA therapy shares some of the difficulties of traditional gene therapy, it requires to reach only the cytoplasm of the cells, not the nucleus. Arguably, this is a simpler technical challenge compared to DNA therapy.