adenovirus-SCN1A

adenovirus-SCN1A (also known as CAV-SCN1A) is an investigational gene therapy being developed for the treatment of Dravet syndrome. It utilizes a helper-dependent canine adenovirus type 2 (CAV-2) vector to deliver a codon-modified SCN1A open reading frame (ORF) encoding the alpha subunit of the voltage-gated sodium channel Nav1.1. Because the SCN1A gene is too large (~6 kb) to fit within standard adeno-associated virus (AAV) vectors, the larger packaging capacity of CAV-2 is leveraged. Developed by researchers at Tel Aviv University, the CNRS, the Université de Montpellier, and the Fundacion para la Investigacion Medica Aplicada, the therapy is designed to restore Nav1.1 expression in both inhibitory and excitatory neurons, thereby correcting the excitation-inhibition imbalance, reducing spontaneous seizures, preventing sudden unexpected death in epilepsy (SUDEP), and improving cognitive and behavioral comorbidities.

Other names

CAV-2-SCN1ACAV2-SCN1ACAV 2-SCN1Acanine adenovirus type 2-mediated SCN1A