NEW RESEARCH PROJECT AT THE UNIVERSITY OF ADELAIDE: “Genetic therapy – a battle against PCDH19–Clustering Epilepsy”

We are pleased to announce our new sponsored research project entitled “Genetic therapy – a battle against PCDH19–Clustering Epilepsy” under the direction of Prof PAUL THOMAS and STEFKA TASHEVA of the University of Adelaide – €99,387.00 value

Lay abstract:

“Our research project aims to develop an innovative therapeutic approach for PCDH19-Clustering Epilepsy (PCDH19-CE). This condition primarily affects girls from infancy and generally responds poorly to standard medications. We aim to test a novel treatment strategy based on the elimination of the causative gene. Using mouse models, AAVs, and CRISPR technologies, we will explore the feasibility of homogeneous elimination of the Pcdh19 gene as a potential therapy for PCDH19-CE. If successful, our genetic manipulation will rescue the PCDH19-CE symptoms. These studies will provide a platform to develop new therapeutic options for patients with PCDH19-CE. The ultimate goal of this research is to translate our findings into clinical applications through AAV-CRISPR-mediated human PCDH19 deletion.”

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– Professor of Biochemistry School of Medicine and director of SA Editing University of Adelaide, Australia
– Senior Principal Research Fellow, South Australian Health and Medical Research Institute
– Postdoctoral Research Fellow, School of Biomedicine, The University of Adelaide, Australia