Transformative Research Priorities for the Epilepsies

Advancing genetic therapies for monogenic epilepsies

Why would this research priority transform epilepsy research, our understanding of the epilepsies, and/or treatment of the epilepsies? :

This idea would accelerate promising treatments for the many stakeholders interested in advancing genetic therapies. Many of the patient advocates for monogenic epilepsies are viewing genetic therapies and a potential path towards a cure, with many working independently to achieve their goals. There have been innumerable advances in genetic diagnoses, availability of genetic testing (although there is more to do) and development of generic tools and technologies that have been essential in enabling the promise of genetic therapies; however, many obstacles remain. Some include, development and availability of appropriate viral vectors, a lack of understanding of topics that range from dosing paradigms for the different approaches through safety issues and translatability to people. In addition, there are ethical considerations that will require thoughtfulness and input from the broad community. This topics could be advanced and accelerated by a deep and focused investment in topics that cut across the needs of the different groups working in this space to accelerate the progress for many.

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Idea No. 264