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ArticleMolecular therapy. Nucleic acids2026

From N-of-1 to versatility in propionic acidemia: Antisense oligonucleotide-mediated skipping of a constitutive

Eriko Totsune et al.PubMed ↗Full text ↗Publisher ↗

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Full record →Abstract, authors, funding and every citing paper · PMID 42028575