Nature Biotechnology unveils a more efficient RNA editing technique

In a new study published in the journal Nature Biotechnology, researchers at the University of California, San Diego, made it easier to repair disease-causing mutations in RNA without compromising accuracy or efficiency

This new RNA-editing technique holds promise as a gene therapy for the treatment of inherited diseases

The technology is unique in that it effectively utilizes RNA-editing enzymes that are naturally present in human cells

Researchers have been exploring ways to use ADARs for RNA editing to correct G-to-A mutations underlying genetic disorders such as cystic fibrosis, Rett syndrome and Hurler syndrome

To use ADAR for targeted A-to-I (essentially A-to-G) editing on RNA, they need a small piece of helper RNA, also known as guide RNA, to guide the ADAR to the target and Make the required changes there

A big challenge with this approach, explained Prashant Mali, a professor of bioengineering at UC San Diego, is that traditional guide RNAs are not efficient when using native ADARs inside cells, so they need to introduce external ADARs into cells

To overcome these problems, Mali and colleagues designed a new guide RNA that recruits the cell's own ADARs very efficiently to edit precise target RNA regions

The research team also designed guide RNAs for a single G-to-A mutation that causes Hurler syndrome

One thing that makes the new guide RNAs more efficient is that they are longer than traditional guide RNAs

These guide RNAs can persist for days and remain in the target RNA region for longer periods of time

Mali believes that the research is still in the early stages, and "it remains to be seen how this RNA editing technology will work in primates

Shape Therapeutics, a Seattle-based biotech startup founded by Mali et al.

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Katrekar, D.