mRNA has been used to deliver functioning genetic code into cells that produces specific proteins. Researchers have found new ways to deliver such molecules and induce gene expression that could help or even treat diseases such as cancer.
Messenger RNA or mRNA is a version of RNA produced by your DNA genes. It is not the first one though, hnRNA or pre-mRNA comes first and later is modified to produce mRNA. This is a way more stable and functional version of RNA. pre-mRNA molecules contain introns and exons. Introns have to be removed, and then several sections at the start and the end of the molecule are also modified to make it more usable by the molecular machines of the cell. This becomes and mRNA that the cell uses to make proteins. On one end it has some regulatory regions, while on the other end it has some additional A (adenine) ribonucleotides. This makes a poly-A tail that is useful for the molecules stability and can even control the rate of protein production.
Creating mRNAs in the lab is hard, but is even harder to introduce them in cells, in a way that isn’t toxic and that simultaneously allows for protein production. This technology is very different to gene editing that is explained in more detail here, and has the potential to be a lot safer too. Due to the lack of gene editing tools used, there is no risk for unwanted mutagenesis. Unfortunately, technologies like this one haven’t had much time to improve, and results remain poor. This new study though, shows that with the right technique it is possible to transfect cells and make them express exogenous mRNA.
Some key components of this study, that contribute to the promising results are the associated proteins, and transfering material used. The carriers previously used for mRNA delivery into the cells have been shown to block mRNA from producing any proteins. To overcome those issues the researchers did a few very interesting things. First they used a protein called eIF4E, an initiation factor, preloaded on the mRNA, so that the translation can start more easily. They also used a cationic nanoplex polypeptide specifically designed to hold the mRNA with a long poly-A tail and poly A binding proteins (PABPs). This according to previous studies that have validated the effectiveness of such methods.
The results are promising for this technology. But it is only one step towards developing something usable in clinical trials. It appears that this methodology is in relatively early stages. Although the logic behind it, is simple, there are serious issues. Genetics is lately a field that promises a lot of cures but also raises a lot of concerns. As any scientific field should. We see a lot of promising techniques being developed and reaching maturity like CRISPR. I think that once we find which technique works best, we will end up changing medicine completely, making gene therapy, either by CRISPR or mRNA techniques, a very powerful tool that treats many diseases.
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