Our laboratory has studied gene therapy for blinding, inherited diseases of the retina for the past 20 years. We have developed animal models of several inherited human blinding diseases, including retinitis pigmentosa, Usher syndrome, and age-related macular degeneration. We have shown that we can protect photoreceptors from injury and cell death in these models by injecting adeno-associated virus or lentivirus vectors into the eye. In these experiments, we have had success in ameliorating photoreceptor cell death with a number of different neurotrophic agents, as well as ribozymes to knockdown the mutant transcripts in dominant disease. The overall goal of our research effort is to prevent or delay the course of blindness in patients. While results in the rodent models show we can protect rat photoreceptors from injury and death, these treatments are not useful for patients in later stages of retinal disease in which the photoreceptor cells have been lost.
In the News
A new genetic therapy developed by UC Berkeley scientists has not only helped blind mice regain light sensitivity sufficient to distinguish flashing from non-flashing lights, but also restored light response to the retinas of dogs, setting the stage for future clinical trials of the therapy in humans. The therapy involves inserting photoswitches into retinal cells that are normally ‘blind.’
Researchers at UC Berkeley have developed an easier and more effective method for inserting genes into eye cells that could greatly expand gene therapy to help restore sight to patients with blinding diseases ranging from inherited defects like retinitis pigmentosa to degenerative illnesses of old age, such as macular degeneration.