Similarly, there were dose-dependent increases in hABCD1 protein expression in Abcd1 −/y mice administered SBT101, but these did not reach statistical significance compared with control treatment ( Figures 3 B and 3F).
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An <i>in vitro</i> and <i>in vivo</i> efficacy evaluation of gene therapy candidate SBT101 in mouse models of adrenomyeloneuropathy and in NHPs.
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