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

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OriginalsprogEngelsk
TidsskriftNucleic Acids Research
Vol/bind43
Udgave nummer9
Sider (fra-til)4627-4639
ISSN0305-1048
DOI
StatusUdgivet - 2015

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