@article{ETD, recid = {747}, author = {Wang, Zhongya}, title = {A newly designed AAV-rDNA vector for persistent therapy in genetic liver diseases}, publisher = {Oregon Health and Science University}, school = {Ph.D.}, address = {2012}, number = {ETD}, abstract = {Adeno-associated virus (AAV) is widely used in gene therapy but has limited integration, reducing long-term transgene expression. We engineered an AAV-rDNA vector to enhance integration and tested it in HT1 and hemophilia B mouse models. AAV-rDNA achieved 10–30 times higher integration and functional expression than control vectors, rescuing disease at much lower doses and restoring normal liver function. Integration was site-specific within rDNA repeats, confirmed by sequencing. These findings demonstrate that AAV-rDNA vectors enable efficient, stable gene expression and represent a promising approach for treating genetic disorders requiring persistent correction.}, url = {http://digitalcollections.ohsu.edu/record/747}, doi = {https://doi.org/10.6083/M4348HC9}, }