- Identification of novel miRNAs in epilepsy. Our research will generate the most complete characterisation of functioning miRNA responses in experimental and human epilepsy. This is a major step toward a comprehensive knowledge of the molecular pathways controlling gene expression in epilepsy. The data will also drive novel directions in pre-clinical anti-epileptogenesis strategies.
- Deep mechanistic understanding of how miRNA changes de-stabilize neuronal networks in brain. Our studies will identify novel miRNAs that control genes regulating brain excitability. By locating these miRNA within causal pathways our results will explain how miRNA changes influence the epileptogenic process.
- Identification of epilepsy biomarkers. We expect to identify miRNA biomarkers of epilepsy. This would help predict disease risk and course/prognosis, direct treatment and avoid adverse side-effects, thereby improving care of epilepsy patients.
- Pre-clinical evaluation of targeting novel miRNAs in epilepsy models. We will obtain pre-clinical data on viral and small molecule delivery for miRNA manipulations and establish effects of miRNA-targeting on epileptogenesis and established epilepsy in rodents. These results will drive interest within (e.g. InteRNA, Bicoll) and outside the consortium (e.g. Biotechnology companies working in RNA therapeutics), as well as academic teams, and stimulate future therapeutic approaches to prevent the development of epilepsy.
- Identification of miRNA genetic variants in patients with epilepsy. DNA sequencing of miRNA will define a completely new area of human epilepsy genetics that improves our understanding of epilepsy, can lead to revised standards that potentially reduce time to diagnosis and treatment. This provides potential healthcare savings and refines genetic counselling so patients have a better understanding of the cause and prognosis of their condition.
- Technological advances. Technological advances and training will be delivered as well as generating a systems biology framework for miRNA which does not yet exist. EpimiRNA will deliver advances in gene therapy, oligo/DNA-based and traditional small chemical scaffolds. EpimiRNA will have a strong impact on bioinformatics, generating in a new data integration platform for RNA-seq, genetics and proteomics.
- SME impact: EpimiRNA will afford SME partners several opportunities to develop and protect intellectual property rights (IPR) in the areas of therapeutics, medical devices for epilepsy, miRNA tools and data platforms.
- Stakeholder impacts: The programme satisfies demands for more research into epilepsy from patient groups and research agendas (e.g. White paper on epilepsy). In its first year EpimiRNA hosted a researchers forum on miRNAs at the 11th ECE in Stockholm. Our programme will ensure continued engagement and agenda sharing with our stakeholders to ensure an engaged and scientifically informed public.
Other impacts: The programme creates new inter-sectorial collaboration that increases the quality of scientific research, drives innovation and ensures translation. The high-impact science of EpimiRNA will support researchers becoming competitive for funding under other European programmes (e.g. ERC).