Population
SCID mice (defective in DNA-dependent protein kinase catalytic subunit) and wild-type mice
Comparison
Recombinant adeno-associated virus vector… vs Wild-type mice
Design
Preclinical
Authors
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DNA-PKcs deficiency reduces circular AAV genomes without impairing dual-vector expression in muscle; leaves open its role in optimizing human gene therapy.
The lack of DNA-PKcs activity alters the equilibrium between circular and linear rAAV concatemer genomes but does not significantly affect the efficiency of dual-vector gene expression in muscle tissue.
Duan et al. (2003) studied this question.
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