LVV-WT SLC6A1

**LVV-WT SLC6A1** is an experimental lentiviral gene therapy vector designed to deliver a wild-type copy of the *SLC6A1* gene, which encodes the sodium- and chloride-dependent GABA transporter 1 (GAT-1). Developed by UCB, this vector is utilized in preclinical research to address SLC6A1-related neurodevelopmental disorders, including childhood-onset epilepsy, myoclonic-atonic epilepsy, and neurodevelopmental delay. In vitro studies using human iPSC-derived GABAergic neurons harboring pathogenic *SLC6A1* mutations have demonstrated that transduction with LVV-WT SLC6A1 successfully increases GAT-1 expression and restores GABA uptake function, highlighting its potential as a therapeutic approach for SLC6A1 haploinsufficiency.

Other names

LV-WT SLC6A1lentiviral vector wild-type SLC6A1