Splice-Switching Antisense Oligonucleotide Drug Discovery for CLN3 Batten Disease

Authors:

Jessica Centa, Matthew Stratton, Vicki Swier, Wanda Pfeifer, Clarissa Booth, Karlee Albert, Mitchell Rechtzigel, Fox Duelli, Hannah Leppert, Trisha Smit, Jill Weimer, Arlene Drack, Michelle Hastings

Journal:

MOLECULAR THERAPY

Publication Date:

Apr 28 2025