Abstract
Background: We have shown previously that intra-arterial delivery of naked plasmid (pDNA) expressing human bilirubin glucuronosyl transferase (hUGT1A1) leads to bilirubin glucuronidation in muscle and significant decrease of total serum bilirubin (TBi) for at least 2 weeks in the UGT1A1 deficient Gunn rat (GR), animal model of Crigler-Najjar Syndrome Type I. This study was undertaken to evaluate the feasibility of repeat intravenous and intra-arterial pDNA delivery into skeletal muscle for long-term metabolic correction in GR.
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