Batten Disease

Antisense Technology for the Treatment of CLN3 Batten Disease

CLN3 Batten disease is a fatal neurodegenerative, lysosomal storage disorder in children. Symptoms begin in early childhood and include vision loss, seizures, and loss of motor function.  This disease has no effective, disease-altering treatment and leads to death in the late teens to early thirties . The Hastings’ lab is using their expertise in modulating gene expression using antisense oligonucleotides (ASOs) to develop potential therapeutics for this devastating disease. To date, the lab has shown that disease-related symptoms in a mouse model of CLN3 Batten disease are mitigated with treatment of an ASO targeting Cln3 RNA splicing. The lab has also successfully used ASOs to target CLN3 gene expression in cells derived from patients with the disease. Further assessment of therapeutic ASOs for CLN3 Batten disease is on-going as well as using ASOs to target additional mutations in CLN3.  The lab also aims to understand the function of CLN3 and determine what is required to rescue the function in the disease context. 

Therapeutic mouse model of CLN3 Batten disease exon skipping ASO approach

Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach

ASOs for the treatment of CLN3 Batten disease

Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease