Excited to share our paper! #GRIN2D-DEE 🐭model mirrors patient symptoms, from behavioral deficits and pathological brain activity to synaptic changes🔗 shorturl.at/JL6pyA An incredible collaboration with a GRIN2D family, @KarenAvraham, researchers & clinicians.

Happy to share our new collaborative paper exploring the molecular basis of DEE-related #SCN8A mutation 🍾 | Work led by @QuinnShir with Yuanyuan Liu, @roybensh, @HaitinLab 🥰, Haim Bassan #epilepsy surl.li/rvzbw

And corrected spatial working memory. 🧬🧬

Protected from heat-induced seizures.

Stopped spontaneous seizures.

Protected from premature death.

When injected at P21, following the onset of seizures in Dravet mice, CAV2-SCN1A corrected hippocampal inhibition.

CAV-2 vectors driving the expression of Nav1.1 under the control of the NSE promoter led to expression in excitatory and inhibitory neurons at the injection site, and with CAV-2's retrograde transport properties, in additional brain regions that project to the injection site.

📢 A novel gene therapy for #Dravet. jci.org/articles/view/159316…. Using Canine adenovirus-2 (CAV-2) we delivered the full SCN1A coding sequence into the hippocampus and thalamus of juvenile Dravet mice.

Thank you @banet_g and Eden for an amazing dinner! Wonderful food and atmosphere❤️🥰🙏🏼