RT101 / Regel Therap - LARVOL DELTA

A PV-Interneuron Selective AAV9 Vector Restores SCN1A Expression and Rescues Phenotypes in a Mouse Model of Dravet Syndrome (AES 2025) - "RT101, an AAV9 vector which expresses dCas9-VP64 selectively within PV interneurons, significantly upregulates SCN1A and significantly improves survival with a reversal of hyperthermia-induced seizure phenotypes in a mouse model of Dravet syndrome. These results provide strong proof-of-concept data supporting the use of PV interneuron-selective gene modulation therapy in Dravet syndrome."

Preclinical • CNS Disorders • Epilepsy • NAV1