Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease
Sign Up to like & getrecommendations! Published in 2025 at "Nucleic Acids Research"
DOI: 10.1093/nar/gkaf1141
Abstract: Abstract CLN3 Batten disease is a lethal pediatric neurodegenerative disease caused by mutations in the CLN3 gene. Typically, the disease manifests as vision loss in early childhood and progresses to neurological dysfunction and death in… read more here.
Keywords: retinal dysfunction; cln3 batten; disease; batten disease ... See more keywords
Searching for novel biomarkers using a mouse model of CLN3-Batten disease
Sign Up to like & getrecommendations! Published in 2018 at "PLoS ONE"
DOI: 10.1371/journal.pone.0201470
Abstract: CLN3-Batten disease is a rare, autosomal recessive disorder involving seizures, visual, motor and cognitive decline, and premature death. The Cln3Δex7/8 mouse model recapitulates several phenotypic characteristics of the most common 1.02kb disease-associated deletion. Identification of… read more here.
Keywords: cln3 batten; disease; batten disease; cln3 ... See more keywords