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. 2017 Jul 27;24(10):661–667. doi: 10.1038/gt.2017.53

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AAV2/9-CMV-hNDUFS4. (a) Schematic representation of AAV2/9-CMV-hNDUFS4 vector. BGH pA, bovine growth hormone polyadenilation signal; CMV, cytomegalovirus promoter; hNDUFS4, human NDUFS4 coding sequence; ITR, inverted terminal repeats; WPRE, woodchuck hepatitis virus posttranscriptional regulatory element. (b) Sequence alignment of Human vs Mouse NDUFS4 proteins. The grey line indicates the predicted mitochondrial targeting sequence. Letters on black background: identities; letters on grey background: similarities; letters on white background: mismatches. Gray arrows show the position of missense mutations found in patients.