Genetic Blindness Treatments Emphasize Immune Role

Late-stage age-related macular degeneration patients suffer blindness when excess blood vessel cells proliferate in the retina, preventing it from functioning properly. Trial treatments have attempted to prevent this by injecting molecules called small interfering RNAs (siRNAs) into the eye, to bind to and specifically switch off a gene that promotes such cell proliferation.

But in studies on mice, researchers led by Jayakrishna Ambati found that the effect also seems to work with other siRNA molecules that have no complementarity to this gene. This, they suggest, means that the treatment works not by inhibiting expression of a particular gene, but by boosting the immune system to ward off the encroaching cells. This insight may change our understanding of how to tackle age-related blindness.

CONTACT

Jayakrishna Ambati (University of Kentucky, Lexington, KY, USA)
E-mail: jamba2@email.uky.edu

Abstract available online.

Message posted by: Trevor M. D'Souza