Evaluation of Transduction Properties of an Adenovirus Vector in Neonatal XXXX .

@entity1 @entity4670 @entity19 @entity2694 @entity74 @entity2834
In gene therapy for @entity4670 , treatments during neonate and @entity1 stages are promising. Replication-incompetent adenovirus (Ad) vectors have been used in gene therapy studies of @entity74 ; however, the transduction properties of Ad vectors in neonates and @entity1 have not been fully examined. Accordingly, this study examined the properties of Ad vector-mediated transduction in neonatal @entity19 . A first-generation Ad vector containing a cytomegalovirus ( @entity2834 ) promoter-driven luciferase expression cassette was administered to neonatal @entity19 on the second day of life via @entity2694 . The highest Ad vector genome copy numbers and transgene expression were found in the neonatal liver. The neonatal heart exhibited the second highest levels of transgene expression among the organs examined. There was an approximately 1500-fold difference in the transgene expression levels between the adult liver and heart, while the neonatal liver exhibited only an approximately 30-fold higher level of transgene expression than the neonatal heart. A liver-specific promoter for firefly luciferase expression conferred a more than 100-fold higher luciferase expression in the liver relative to the other organs. No apparent hepatotoxicity was observed in neonatal @entity19 following Ad vector administration. These findings should provide valuable information for gene therapy using Ad vectors in neonates and @entity1 .
