MicroRNA as diagnostics and therapeutics for retinal degenerations

MicroRNA (miRNA) are small, endogenous, non-coding molecules that are powerful regulators of genetic information. Despite only being discovered as recently as the turn of this century, miRNAs are already used in clinical trials as therapeutic candidates for complex diseases such as cancer. This rapid bench-to-bedside transition demonstrates the therapeutic potential of miRNAs, particularly for multi-faceted diseases. In fact, miRNAs have already been implicated in the pathogenesis of complex neurodegenerative disorders such as Parkinson’s, Alzheimer’s, and Age-related Macular Degeneration (AMD). At the Clear Vision Research Lab, we believe that we can use miRNAs for two key areas currently lacking in the clinical landscape for AMD:

Diagnostic biomarkers

The Clear Vision Research Lab is currently undertaking a project in which we are investigating the use of miRNA as biomarkers for retinal degenerations. MiRNA demonstrate relatively high stability and abundance in biofluids such as tears, saliva, urine and blood, making them a promising target for prognostic research. Current investigations exploiting biofluid miRNAs for AMD diagnosis has yielded inconsistent results due to the multifaceted nature of disease progression and existing co-morbidities. Our research aims to identify specific miRNAs indicative for different stages in retinal disease and develop a method of disease grading based on their expression in biofluids. We further aim to determine if this panel of miRNAs can also be used as an indicator of therapeutic efficacy.

Therapeutic candidates

We currently have a number of ongoing projects where we are attempting to harness the regulatory capabilities of miRNAs to use as therapeutics for AMD. A single miRNA has the ability to control multiple different mRNA, often within the same molecular pathway (e.g. inflammation). This ability makes miRNAs promising therapeutic molecules to target multiple players in a single pathway. Due to the complex nature of AMD, we believe that this approach may prove fruitful in ameliorating key pathways known to lead to retinal degeneration, such as inflammation and oxidative stress. Ourresearch aims to characterise key miRNAs in the retina and, by understanding their dynamic activity under retinal stress, exploit those as therapeutic molecules in the retina.