Researchers in the United States have discovered an exceptional antibody, that could lead to a treatment for retinitis pigmentosa (RP).
RP is a group of inherited eye diseases that affect the retina in the back of the eye. It is caused by the death of cells that detect light signals, known as photoreceptor cells. There is no known cure for RP, and the development of new treatments for this condition relies on cell and gene therapies.
The paper, by a team from the University of California and published in Nature Communications,1 concentrates on their investigation of the molecule rhodopsin, which stands as a pivotal photosensitive molecule in the human retina.
More than 150 mutations in rhodopsin can cause RP, making it challenging to develop targeted gene therapies,” said Professor Krzysztof Palczewski, from the UCI School of Medicine.2
For this study, researchers used a special type of antibody, known as a nanobody, derived from llamas.
“However due to the high prevalence of RP, there has been significant investment in research and development efforts to find novel treatments.”
Although rhodopsin has been studied for over a century, key details of its mechanism for converting light into a cellular signal have been difficult to experimentally address. For this study, researchers used a special type of antibody, known as a nanobody, derived from llamas. This nanobody can halt the process of Rhodopsin photoactivation, allowing it to be investigated at high resolution.
“Our team has developed nanobodies that work through a novel mechanism of action. These nanobodies have high specificity and can recognize the target rhodopsin extracellularly,” said Dr David Salom, also from the UCI School of Medicine. “
“In the future, we hope to involve the in vitro evolution of these initial set of nanobodies,” said Dr Arum Wu, researcher and project scientist, UCI School of Medicine. “We will also evaluate the safety and effectiveness of a future nanobody gene therapy for RP.”
References
- Wu, A., Salom, D., Hong, J.D.et al. Structural basis for the allosteric modulation of rhodopsin by nanobody binding to its extracellular domain. Nat Commun 14, 5209 (2023). org/10.1038/s41467-023-40911-9.
- UCI School of Medicine, UC Irvine researchers discover a nanobody which may lead to treatment for Retinitis Pigmentosa (media release), 29 Aug 2023, available at: medschool.uci.edu/news/uc-irvine-researchers-discover-nanobody-which-may-lead-treatment-retinitis-pigmentosa [accessed 8 Sept 2023].
