Retinitis Pigmentosa Animal Models

Retinitis pigmentosa is a human genetic disorder that causes blindness due to death of photoreceptors. Many cases of retinitis pigmentosa are caused by a mutation in the rhodopsin gene, rhodopsin P23H. This disorder has been modeled in transgenic Xenopus laevis by expressing the bovine rhodopsin cDNA containing the P23H mutation. An assay has been developed which allows quantification of photoreceptor death using an antibody that detects total rhodopsin. The expression of the bovine P23H rhodopsin can be independently assessed using a different specific antibody. This X. laevis P23H rhodopsin model system can be used to investigate possible mechanisms for the toxicity of P23H mutant rhodopsin – such mechanisms may have broad applicability to other mammalian forms of RP (ie. human). The model could possibly be used to screen for drugs which mimic the rescuing effects of darkness. Alternatively, the model and assay could be used to identify specific wavelengths of light that are most detrimental to photoreceptor survival, and this information could in turn be used to develop protective glasses for patients with retinitis pigmentosa. Advantages:

The X. laevis transgenics are easy to generate in large numbers, are genetically non-chimeric, have rapid development, and produce large numbers of offspring. In addition, the rod cells are large and easily resolved by light microscopy

The quantitative assay adds simplicity and utility to the already advantageous model system – this could add the ability to use the system as a high throughput screen for RP drugs.

Type of Offer: Licensing

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