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UC Irvine researchers discover a nanobody which may lead to treatment for retinitis pigmentosa

August 31, 2023
UCI scientists

Study authors and UC Irvine investigators Arum Wu, David Salom, and Krzysztof Palczewski. 

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-bound nanobodies

Crystal structure of two nanobodies binding to a rhodopsin dimer. The rhodopsin molecules are shown in green and blue, with 11-cis-retinal displayed in red. The figure emphasizes the significant interactions between the nanobodies (represented in a semi-transparent surface cartoon) and the extracellular surface of rhodopsin, including its N-terminal glycans highlighted in orange. Credit: University of California Irvine.

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.