ARTICLE | Translation in Brief

Survival of the modified

How gene therapies can give themselves a leg up in the liver

July 21, 2016 7:00 AM UTC

A selection strategy commonly used to clone cells in vitro has been co-opted by a group at Oregon Health & Science University to create a gene therapy platform that gives a proliferation advantage to cells that receive and properly integrate the gene.

The platform aims to overcome a common limitation of gene therapy - low numbers of successfully modified cells. "The whole idea here is that you take a rare event and amplify it," said Markus Grompe, professor of molecular and medical genetics and pediatrics at OHSU. In a Science Translational Medicine study published last month, the team used the platform to deliver a human Factor IX transgene in mice...