Invention Summary:
Researchers at Rutgers University have shown that fluorescent porphyrins cause protein aggregation that interferes with protein function, and consequently cellular function. They have found that uro-I accumulation leads to protein aggregation and CEP-related bone phenotype, and have developed a zebrafish model that phenocopies features of CEP. As in human patients, uro-I accumulated in the bones of zebrafish, leads to impaired bone development. In an osteoblast-like cell line, uro-I decreased mineralization, aggregated bone matrix proteins, activated endoplasmic reticulum stress and disrupted autophagy.
Using high-throughput drug screening, the researchers have identified acitretin, a second-generation retinoid, that reduces uro-I accumulation and its deleterious effects on bones. Another retinoid, tretinoin showed porphyrin clearance in zebrafish. These findings provide a new CEP experimental model and describe the use of retinoids as potential repurposed therapeutics for CEP.
Advantages:
Intellectual Property & Development Status: Provisional patent application filed, patent pending. Available for licensing and/or research collaboration. For any business development and other collaborative partnerships contact : marketingbd@research.rutgers.edu