University of California Irvine (UCI) researchers believe they have discovered a special antibody that may lead to a treatment for an inherited eye disease called retinitis pigmentosa.
Rhodopsin is a light-sensing molecule in the retina's rod photoreceptor cells.
“More than 150 mutations in rhodopsin can cause retinitis pigmentosa, making it challenging to develop targeted gene therapies,” said Krzysztof Palczewski, Ph.D., UCI School of Medicine and senior author of a report on the research, published in Nature Communications.
The researchers investigated rhodopsin at high-resolution using a llama-derived antibody, called a nanobody that can halt rhodopsin photoactivation.
The nanobodies targeted an unexpected site on the rhodopsin molecule. They also found that the stabilizing effect of these nanobodies can also be applied to rhodopsin mutants that are associated with retinal disease, suggesting their use as therapeutics.