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Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease.
Nat Med. 2020 Sep;26(9):1444-1451. doi: 10.1038/s41591-020-0986-1. Epub 2020 Jul 27.
Nat Med. 2020.
PMID: 32719489
Free PMC article.
Targeting Alternative Splicing for Therapeutic Interventions.
Centa JL, Hastings ML.
Centa JL, et al.
Methods Mol Biol. 2022;2537:21-36. doi: 10.1007/978-1-0716-2521-7_2.
Methods Mol Biol. 2022.
PMID: 35895256
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Protracted CLN3 Batten disease in mice that genetically model an exon-skipping therapeutic approach.
Centa JL, Stratton MP, Pratt MA, Osterlund Oltmanns JR, Wallace DG, Miller SA, Weimer JM, Hastings ML.
Centa JL, et al.
Mol Ther Nucleic Acids. 2023 Jun 3;33:15-27. doi: 10.1016/j.omtn.2023.05.025. eCollection 2023 Sep 12.
Mol Ther Nucleic Acids. 2023.
PMID: 37359347
Free PMC article.
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