2014

Innovator Award

Targeting Epigenetics To Treat Epilepsy In Tuberous Sclerosis Complex: An Epiphany From Patient Whole Genomic Expression Analysis

Avtar Roopra, PhD
University of Wisconsin-Madison
 

Epilepsy has both genetic and non-genetic roots and Tuberous Sclerosis Complex (TSC) is a leading genetic cause. Though much is understood about the mutations behind TSC, nothing is known about the drivers of gene changes in the brains of TSC patients that cause seizures and epilepsy. The work proposed here is the first to explore alterations in the way genes are controlled, a process called ‘epigenetics’ that we believe is a major driver of epilepsy in TSC. We aim to open up an entirely new arsenal of drugs – epigenetic modifiers - in the fight against epilepsy in TSC. TSC associated genes play a role in almost every model of epilepsy. Therefore we believe that our finding will aid in the quest for “no seizures, no side effects” in multiple genetic and non-genetic epilepsies.

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