Article

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

2025-05-31

Abstract excerpt

<h4>ABSTRACT</h4> 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,...

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Literature Corpus work: a3b9f6f7-e42d-5f36-b806-9cd680766615
DOI: 10.1101/2025.05.30.656864

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