Molecular BiologyNucleic Acids ResearchPeer-reviewed
Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease
Matthew P. Stratton, Jessica L. Centa, Vicki J. Swier…·Michelle L. Hastings
A splice-switching antisense oligonucleotide delivered via intravitreal injection mitigates early retinal dysfunction in a pig model of CLN3 Batten disease.
4 citations