Scientists have discovered a novel way to use a radical new type of gene therapy to prevent blindness caused by retinitis pigmentosa.
The research, led by Muna Naash, at the University of Oklahoma Health Sciences Center, with collaborators in Cleveland and Buffalo, discovered a way to deliver known gene therapies directly to the light-sensitive cells affected by this disease.
The discovery already is being used to develop new treatments for another disease – macular degeneration, the leading cause of blindness in the United States.
Utilizing nanoparticle technology, scientists created a microscopic capsule capable of carrying genetic therapies to their destination inside cells of the retina.
The tiny delivery vehicle is being tested with a variety of gene therapies in animal models with the potential of treating several diseases from bladder cancer to diabetes.
The capsules have proven very effective, carrying therapies to the designated location in the eye within 15 minutes of delivery and spreading the genetic repair message quickly to nearby cells.