Stable transduction of the neonatal mouse liver using a hybrid rAAV/<i>sleeping beauty</i> transposon gene delivery system
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.
Authors
- Eva B. van Dijk (ORCID: https://orcid.org/0009-0000-7241-2280)
- Jacob R. Rubens
- Erhua Zhu
- William E. Salomon (ORCID: https://orcid.org/0000-0002-5388-0203)
- Robert J. Citorik (ORCID: https://orcid.org/0000-0002-6397-5417)
- Ian E. Alexander (ORCID: https://orcid.org/0000-0002-6213-5627)
- Sharon C. Cunningham (ORCID: https://orcid.org/0000-0002-6335-9104)
- Philip M. Zakas (ORCID: https://orcid.org/0000-0002-4919-5182)
- Cecilia Cotta‐Ramusino
- William Querbes
- Yanfang Fu
- Natsuki Sasaki (ORCID: https://orcid.org/0009-0000-2624-6568)
Institutions
- The University of Sydney (AU)
- Children's Medical Research Institute (AU)
- Xperi (United States) (US)
- Sydney Children’s Hospitals Network (AU)
Publication Details
- Journal
- The Journal of Gene Medicine
- Published
- 2024-08-01
- DOI
- https://doi.org/10.1002/jgm.3726
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- University of Pennsylvania