The eyes have it for non-viral gene therapy

Nanoparticles for gene therapy may work better than viruses and produce fewer side-effects, according to research from Tufts University. MedGadget reports that, in the latest Nature Molecular Therapy, researchers used nanoparticles, rather than viruses, to deliver a gene that codes for a photoreceptor protecting protein produced in the eye.

According to Tufts, mice were treated with nanoparticles carrying a gene for GDNF (Glial Cell Line-Derived Neurotrophic Factor), a protein that protects the photoreceptor cells in the eye. Retinas treated with the GDNF-carrying nanoparticles showed significantly less photoreceptor cell death than controls. Preservation of these cells resulted in significantly better eyesight in the treatment group seven days after treatment, compared to controls.

"The next step in this research is to prolong this protection by adding elements to the DNA that permit its retention in the cell. Bringing forth a more potent and enduring result will move us closer to clinical application of non-viral gene therapy," said senior author Rajendra Kumar-Singh in a prepared statement.

- read the Medgadget report
- look at the Tufts news release
- and see the abstract