mRNA vaccines are developing very quickly. There are already 25 ongoing clinical trials against cancers, as well as clinical trials against infectious diseases such as HIV. Recombinant monoclonal antibody (mAB) based treatments that have lead to the most successful therapies could even switch to mRNA coding heavy and light chains for direct antibody production into the patient (as of yet still at the level of mouse models).

Indeed, messenger RNAs (mRNA) are now new biological entities to take in consideration for the development of efficient and personalized therapies. They have several advantages over conventional drugs:

This summer, Moffett et al. published in Nature Communications (Nature Communications – doi:10.1038/s41467-017-00505-8) a new approach for cancer therapeutic research and development based on mRNA delivery. They called it ‘Hit-and-run programming’ because of the simplicity of the method. Interestingly, the optimization of the delivery was performed with eGFP mRNA.  This post describes how TriLink’s mRNAs contribute to the simple and efficient delivery of mRNA into cells.

Messenger RNAs – a promising class of new therapeutic biologics

As messenger RNAs (mRNAs) are easier to deliver into cells than plasmids or viral vectors, they are useful for non-dividing cells. Inversely to the vectors, they ensure genome integrity that is recommended for cell therapeutics. mRNA are also well adapted to transient expression as required for cellular reprogramming, gene editing, and vaccines.

You might like to take a look at this poster, presented by Trilink (renowned modified nucleic acid experts) at the Keystone symposia last March (Pattern Recognition Signaling: From Innate Immunity to Inflammatory Disease). They share results on how to optimize messenger RNA for therapeutic activity.

“Innate Immune focused approaches to maximize messenger RNA therapeutic activity”
Download your copy of the poster here.

CRISPR/Cas9 is relatively simple to implement, as the researcher fully controls the experimental design of the tools, from the sgRNA sequence to the Cas9 protein.