RNA Editing

“You don’t take a permanent aspirin”

Today, Mitch Leslie's feature article in Science made me aware of the potential of mRNA editing as an exciting form to treat diseases.

The central dogma of biology states that information is stored inside our cell nucleus in the form of DNA. A part of this information is copied out via mRNA molecules, and outside of the nucleus, mRNA is translated into proteins.

With gene therapy, we edit faulty DNA inside the nucleus or introduce corrected DNA snippets. With RNA therapy, we add multiple types of RNA into cells to destroy proteins or have new proteins made.

RNA editing offers an alternative therapy form: we can take mRNA and modify it inside the cell cytosol to produce a correct protein.

This type of therapy is promising because:

• We do not need to interfere with DNA and therefore have less fear that modified DNA will end up in our reproductive system.
• It is a transient therapy form that can be stopped in case of side effects (see quote in the title).
• Reaching the cytosol is much easier than going into the nucleus.

There are still many challenges ahead. The efficiency of RNA editing will be improved in the years to come, and we will find efficient delivery forms that target specific cells.

It reminds me of the famous quote by Inder Verma: the three major challenges to gene therapy are “delivery, delivery, and delivery.”