Scientists Show Message From Gene to Cell Can Be Changed

In a step that suggests a new approach to gene therapy, scientists have been able to edit the message that a gene sends to a cell.

The strategy might allow treatment of genetic diseases that appear when faulty genes send improper messages to cells, causing them to produce harmful or malfunctioning proteins, researchers said.

The traditional gene therapy approach to such diseases is to try to implant a working version of the gene instead.

Genes tell cells to produce certain proteins by sending a message in a substance called RNA. This RNA message is usually modified by other substances before it reaches the cell’s protein-making machinery.

In the June issue of the journal Nature Medicine, scientists from the Duke University Medical Center in Durham, N.C., report that they were able to intervene in this modification process to edit an RNA message.

The work was done with cells in laboratory dishes. The message came from a gene that researchers had inserted into the cells, and the editing was done by inserting an enzyme designed to target the RNA from the gene.

Results showed that the enzyme altered not only the RNA it was aimed at, but also RNA from other genes in the cell. So it will take more work to make the enzyme work only on its target, researchers said.