Repairing genes

A new generation of gene therapy companies is working on fixing endogenous defects, rather than simply adding a normal copy of defective genes. One such approach is being followed by Intronn LLC, a subsidiary of Proteome Sciences plc (LSE:PRM, Cobham, U.K.), which has shown in a cell model of cystic fibrosis that it can make use of a cell's RNA splicing mechanisms to repair a damaged message before it is translated into a protein.

Intronn (Durham, N.C.) used its SMaRT technology in vitro to repair the most common cause of CF, a deletion mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel gene. The technology involves introducing a "pre-therapeutic" RNA molecule (PTM) that contains the corrected RNA message into a cell and allowing the cell's own splicing mechanisms to replace the mutated portion with the corrected message...