THE trick could have come from a thrifty plumber: why replace a leaky pipe if you can simply patch it up?

A single fault in DNA can travel a long way. It may be copied into RNA, which then produces the faulty proteins that underlie some genetic disorders.

Now biologists have laid the groundwork for creating tools designed to bind to faulty RNA and fix protein production. The tools are themselves proteins.

Ian Small at the University of Western Australia, Crawley, and his colleagues identified a special class of RNA-binding proteins 12 years ago.


“One vision of their activity is as genomic debuggers – they correct mutations at the RNA level,” he says.

Small’s team has now worked out how their RNA-binding proteins, called PPR, latch on to RNA. PPR proteins carry a pattern of amino acids that binds to the bases on RNA. The team realised that just two amino acids in each pattern determine exactly which part of an RNA molecule the PPR protein binds to (PLoS Genetics, doi.org/h7b).

The finding paves the way for building “designer PPR proteins” that target and fix specific genes, Small says.