TY  - GEN
AB  - 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.
AD  - Oregon Health and Science University
AU  - Wang, Zhongya
DA  - 2012
DO  - 10.6083/M4348HC9
DO  - DOI
ID  - 747
KW  - Acetoacetates
KW  - Virus Integration
KW  - Liver
KW  - Tyrosinemias
KW  - Dependovirus
KW  - Genetics
KW  - Factor IX
KW  - Liver Diseases
KW  - Hemophilia B
KW  - DNA, Ribosomal
KW  - adenoviruses
KW  - recombinant dna
KW  - blood coagulation factor ix
L1  - https://digitalcollections.ohsu.edu/record/747/files/750_etd.pdf
L2  - https://digitalcollections.ohsu.edu/record/747/files/750_etd.pdf
L4  - https://digitalcollections.ohsu.edu/record/747/files/750_etd.pdf
LK  - https://digitalcollections.ohsu.edu/record/747/files/750_etd.pdf
N2  - 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.
PB  - Oregon Health and Science University
PY  - 2012
T1  - A newly designed AAV-rDNA vector for persistent therapy in genetic liver diseases
TI  - A newly designed AAV-rDNA vector for persistent therapy in genetic liver diseases
UR  - https://digitalcollections.ohsu.edu/record/747/files/750_etd.pdf
Y1  - 2012
ER  -