Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle.
basic_science · Level V
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- Record sourced from PubMed, PMID 11274433.
- Also identified by PMC identifier 31183.
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Abstract
We report here that the DNA-dependent protein kinase (DNA-PK) affects the molecular fate of the recombinant adeno-associated virus (rAAV) genome in skeletal muscle. rAAV-human alpha1-antitrypsin (rAAV-hAAT) vectors were delivered by intramuscular injection to either C57BL/6 (DNA-PKcs(+)) or C57BL/6-SCID [severe combined immunodeficient (SCID), DNA-PKcs(-)] mice. In both strains, high levels of transgene expression were sustained for up to 1 year after a single injection. Southern blot analysis showed that rAAV genomes persisted as linear episomes for more than 1 year in SCID mice, whereas only circular episomal forms were observed in the C57BL/6 strain. These results indicate that DNA-PK is involved in the formation of circular rAAV episomes.
Medical subject headings
- DNA-Binding Proteins
- Dependovirus
- Muscle, Skeletal
- Protein Serine-Threonine Kinases