Cannabidiol attenuates epileptic phenotype and increases survival in a mouse model of developmental and epileptic encephalopathy type 1.

ABSTRACT:

Objective

Developmental and epileptic encephalopathy type 1 (DEE1) is a rare drug-resistant pediatric epilepsy caused by trinucleotide repeat expansions in the X-linked ARX gene, leading to elongation of the first polyalanine tract. It presents with early onset tonic seizures or spasms, developmental and cognition delay, and high risk of premature mortality. We evaluated the therapeutic potential of highly purified cannabidiol (CBD) in Arx^(GCG)7/Y mice, a genetic DEE1 model that replicates key features of the human condition.

Methods

Arx^(GCG)7/Y mice received daily intraperitoneal CBD (100 mg/kg) for 7 days. The epileptic phenotype was evaluated via video monitoring and a scoring matrix. In Arx-DEE1 male cortex, real-time polymerase chain reaction and W

SUBMITTER: Verrillo L 

PROVIDER: S-EPMC12605674 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature