Innovator Award, Funded by the Julie's Hope Award

Scalable Transcriptional-Translational Therapy Of Epileptogenic Gene Haploinsufficiencies

Antonello Mallamaci, PhD
Scuola Internazionale Superiore di Studi Avanzati, Trieste, Italy

More than one hundred distinct gene losses may lead to epilepsy. As these pathologies are individually rare and the defective genes are implicated in a variety of cellular processes, the development of effective cures is hard, due to the complexity of the problem and the huge economic investments needed. We will try to fix this issue via multilevel stimulation of the spared gene copy, by three novel enabling technologies, which comply with endogenous gene regulation needed for neuronal function. If successful, this approach will be exploitable for scalable, personalized cures of epileptogenic gene loss.


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