The Genome Book

Gene therapy — the use or manipulation of genes to address disease — doesn’t get much attention in general health news. There were notable failures and problems early on, and since then, efforts have been more low-key.

But every now and then, a new study makes it clear that gene therapy researchers keep trying, and sometimes succeed.

A new study in The Lancet found that in patients with an inherited vision disorder called LCA, gene therapy improved eyesight, especially in children. (Tip for all non-nerds: If you find the study in The Lancet a bit too heavy on medical and genetic jargon, you can find a more readable overview here.)

The usual cautions apply here. LCA is a rare condition. This study was small and early-stage, involving only 12 patients. More research is required.

But it’s also an indication of how gene therapy is likely to proceed. Innovations will emerge gradually, one condition at a time, but they will come. As The Lancet notes, one upcoming challenge will be getting patients genotyped (having their genetic makeup analyzed) so that it’s easier to know who might benefit. In our book, we have more background on gene therapy in Chapter 9. In our next edition, we’d like to include more information on vision disorders, as many common ones have a strong genetic component.

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