Splice-shifting oligonucleotide (SSO) mediated blocking of an exonic splicing enhancer (ESE) created by the prevalent c.903+469T>C MTRR mutation corrects splicing and restores enzyme activity in patient cells

Bruno Palhais, Veronica Præstegaard, Rugivan Sabaratnam, Thomas Koed Doktor, Seraina Lutz, Patricie Burda, Terttu Suormala, Matthias Baumgartner, Brian Fowler, Gitte Hoffmann Bruun, Henriette Skovgaard Andersen, Viktor Kozich, Brage Storstein Andresen

Publikation: Bidrag til tidsskrift › Tidsskriftartikel › Forskning › peer review

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Biochemistry, Genetics and Molecular Biology