Psd3-AAV

Psd3-AAV is an experimental gene therapy that utilizes an adeno-associated virus (AAV) vector to deliver a CRISPR activation (CRISPRa) system targeting the Pleckstrin and Sec7 domain containing 3 (PSD3) gene. It is being investigated as a genetic modifier for Dravet syndrome, a severe form of epilepsy typically caused by SCN1A mutations. By upregulating PSD3 expression, the therapy aims to mitigate the disease phenotype. In preclinical studies using the Scn1a+/- mouse model, intracerebroventricular administration of Psd3-AAV significantly increased seizure thresholds in hyperthermia-induced seizure assays, suggesting its potential as a therapeutic approach to improve survival and reduce seizure susceptibility.