Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease.

ABSTRACT: CLN3 Batten disease is a lethal pediatric autosomal recessive neurodegenerative disease caused by mutations in the CLN3 gene. Typically, the disease manifests as vision loss early in life and progresses to neurological dysfunction and death in young adulthood. Therapeutic development has focused on treating the central nervous system. However, such therapies may not protect against vision loss, which has a significant impact on quality of life. We have shown that a splice-switching antisense oligonucleotide (ASO) delivered to the central nervous system can reduce neurological disease burden in mouse models of CLN3 disease. Here, we report on a similar ASO approach for treating CLN3 Batten disease retinal dysfunction in a pig model of the disease, which is more representative of huma

SUBMITTER: Stratton MP 

PROVIDER: S-EPMC12154811 | biostudies-literature | 2025 May

REPOSITORIES: biostudies-literature