Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease
Matthew P. Stratton, Jessica L. Centa, Vicki J. Swier, Wanda L. Pfeifer, Clarissa D. Booth, Karlee Albert, John L. Hunyara, Mitchell J. Rechtzigel, Fox J. Duelli, Hannah G. Leppert, Frank Rigo, Trisha Smit, Paymaan Jafar‐Nejad, Jill M. Weimer, Arlene V. Drack, Michelle L. Hastings
A splice-switching antisense oligonucleotide delivered via intravitreal injection mitigates early retinal dysfunction in a pig model of CLN3 Batten disease.
Nucleic Acids Research · Oct 16, 2025Peer-reviewedMolecular Biology