Affiliation: University of Wisconsin
- Nonviral gene transfer into liver and muscle for treatment of hyperbilirubinemia in the gunn ratISTVAN DANKO
Department of Pediatrics, Waisman Center, University of Wisconsin Madison, Madison, WI 53792, USA
Hum Gene Ther 15:1279-86. 2004..A 30% decrease in serum bilirubin, if sustained, would provide meaningful clinical benefit for CN-I patients. However, to be clinically useful, this method needs further optimization and stable gene expression must be achieved...
- Long-term correction of hyperbilirubinemia in the Gunn rat by repeated intravenous delivery of naked plasmid DNA into muscleZhen Jia
Department of Pediatrics, Waisman Center, University of Wisconsin at Madison, Madison, WI 53705, USA
Mol Ther 12:860-6. 2005..The procedure is safe and simple, with great clinical potential. Further studies are needed to explain the mechanisms of loss and improve the stability of recombinant hUGT1A1 in muscle...
- Single hepatic venous injection of liver-specific naked plasmid vector expressing human UGT1A1 leads to long-term correction of hyperbilirubinemia and prevention of chronic bilirubin toxicity in Gunn ratsZhen Jia
Department of Pediatrics, Waisman Center, University of Wisconsin Madison, 53705, USA
Hum Gene Ther 16:985-95. 2005..Our results provide further evidence of the feasibility of long-term correction of hepatic enzyme deficiencies with plasmid vectors optimized for expression in the liver...
- Muscle-directed gene therapy for Crigler-Najjar SyndromeISTVAN DANKO; Fiscal Year: 2005..effects of gene therapy on chronic bilirubin toxicity (renal tubular damage) will be studied. These studies will provide proof-of-concept data for this gene therapy approach. ..