A team at the University of Iowa has developed an artificial tRNA that could restore the production of the protein that's affected in people with cystic fibrosis, New Scientist reports.
Iowa's Christopher Ahern and his colleagues report in a preprint at BioRxiv that they engineered tRNAs to convert an in-frame nonsense stop mutation to the wild-type version. In particular, the researchers focused on that CFTR W1282X nonsense mutation that's found in cystic fibrosis patients, and they report that their engineered tRNAs could rescue that mutation.
The New Scientist notes that these artificial tRNAs work by competing with unaltered ones to translate RNAs into proteins. "This means that artificial tRNAs will not fix every protein made by the faulty gene, but they may be able to fix just enough to make a difference — for many genetic disorders, even low levels of protein can make a huge difference," it says.
However, Newcastle University's Malcolm Brodlie tells the New Scientist that any artificial tRNA-based treatment is a ways off as ways to deliver such a therapy to cells are still under development. He adds, though, that the work is "really exciting."