The main focus of my lab is finding novel gene therapy approaches for intractable neurological diseases, with rapid potential for translation. We start by investigating the pathophysiological mechanisms of disease and then use novel insights to develop and improve therapeutic tools to cure them.
We use cutting-edge technologies such as CRISPR and synthetic promoters to overcome current limitations to gene therapy and develop new approaches for curing currently untreatable genetic diseases such as Dravet Syndrome and Episodic Ataxia-1, and acquired pharmaco-resistant temporal lobe epilepsies.
We use techniques ranging from molecular biology to in vitro and in vivo electrophysiology, in both cell lines and animal models, and iPSC-derived neurons from patients. We collaborate with several groups worldwide to complement our techniques and to accelerate our goal of finding innovative cures for devastating pathologies.