Gene Therapy | St. Louis Public Radio

Gene Therapy

(via WashU/copyright PNAS)

Researchers at Washington University have genetically-engineered cells to react to light.

By taking light-sensing receptors from the eye — called opsins — and inserting them into immune cells, the researchers were able to trick the cells into moving toward a laser beam, in the same way they would move toward a bacterial infection.

Washington University molecular biologist N. Gautam led the research.

This article first appeared in the St. Louis Beacon: Studies presented at a recent meeting of the Association for Research in Vision and Ophthalmology and published online April 27 in the New England Journal of Medicine are supporting further research into gene therapy to treat a rare genetic eye disorder.

The disorder, called Leber's congenital amaurosis (LCA), attacks the retina and can lead to severe vision loss and blindness. LCA is a rare genetic condition affecting 1 in 50,000 to 1 in 100,000 of babies born each year. Eyesight begins to fail in early childhood, progressing to total blindness by the time the patient reaches his or her late 20s or 30s.