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POLYALA — Result In Brief

Project ID: 18675
Country: Portugal

Tracking the molecular processes of neurodegeneration

A cellular target has been identified as a possible suppressor of the development of a rare neuromuscular disease. Further research into its molecular effects could be used to develop relevant therapeutic approaches.
Tracking the molecular processes of neurodegeneration
The 'Insights into novel therapeutic strategies for a nuclear inclusion disease caused by polyalanine expansion' (Polyala) project made headway in uncovering new information related to the development of oculopharyngeal muscular dystrophy (OPMD). The EU-funded project built on earlier designed cellular and animal models for a better understanding of the molecular mechanisms of OPMD.

Intending to also propose new strategies for treatment of the disease, Polyala studies involved basic research on the structure and function of normal versus expanded nuclear poly(A) binding protein (PABPN1). The approach aimed to learn more about its interacting partners within the cell.

Project partners developed an assay for screening molecules that could alleviate OPMD-like phenotypes in Drosophila, a genus of small flies commonly referred to as 'fruit flies'. A particular species of Drosophila is widely used in research in genetics and is the basis for a model organism commonly employed in developmental biology studies. Results identified the anti-aggregation molecule 6AP, which has been previously identified in models of another family of neurodegenerative disorders, prion disease.

Further investigation identified the 6AP molecule as a good suppressor of OPMD in Drosophila. Boasting a similar molecular effect as other less toxic molecules, these findings have the potential for more beneficial development of future OPMD therapeutic strategies.

Other Polyala project activities included the establishment of a competitive European network on OPMD. The communication structure that was created continues to facilitate the exchange of information, resources and reagents among field researchers. This initiative served to boost interactions with patients and patient organisations, and will also ensure that the community is maintained beyond the scope of the project.

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