Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice.
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Nucleic Acids Res. 2022 Nov 11;50(20):11401-11414. doi: 10.1093/nar/gkac641.
Nucleic Acids Res. 2022.
PMID: 35944903
Free PMC article.