Why are some patients literally immune to gene therapy?

Scientists are searching for a way to help gene therapies sneak past the body's defence system
Jovana Drinjakovic
Scientist and DNA

Over the past decade, scientists have been talking about another new dawn of another new era where it will become possible to reverse genetic disorders using gene therapies.

The excitement rose a little in 2012 when the European Union approved its first therapy targeted at a rare, lipid-processing disease, lipoprotein lipase deficiency.

A few years ago, the US Food and Drug Administration granted approval for a genetic treatment for patients with a rare gene mutation resulting in retinal dystrophy.

“Gene therapy has the potential to be transformational,” says Professor David Schaffer, whose lab at the University of California, Berkeley, researches gene therapeutics.