Stable transduction of the neonatal mouse liver using a hybrid rAAV/sleeping beauty transposon gene delivery system.

Sharon C CunninghamPhilip M ZakasNatsuki SasakiEva B van DijkErhua ZhuYanfang FuWilliam E SalomonRobert J CitorikJacob R RubensCecilia Cotta-RamusinoWilliam QuerbesIan E Alexander

Published in: The journal of gene medicine (2024)

The present study demonstrates the first in vivo utility of a hybrid rAAV/SB100X transposon system to achieve stable long-term therapeutic gene expression following delivery to the highly proliferative newborn mouse liver. These results have relevance to the treatment of genetic metabolic liver diseases with neonatal onset.

Keyphrases

gene expression
genome wide
dna methylation
copy number
combination therapy