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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.
Nucleic Acids Res
; 43(9): 4627-39, 2015 May 19.
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| MEDLINE | ID: mdl-25878036
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