   3.4.14.9 | more |
neonatally treated mice display high levels of TPP-I activity in the CNS 1 year after administration of AAVrh.10hCLN2 vector directly to the neonatal brain of CLN2 knockout mice at 2 days, 3 weeks, and 7 weeks of age, the AAVrh.10hCLN2 vector contains an expression cassette consisting of the human CLN2 cDNA driven by a CMV/beta-actin hybrid promoter consisting of the enhancer from the cytomegalovirus immediate early gene, the promoter, splice donor and intron from the chicken beta-actin gene, the splice acceptor from the rabbit beta-globin gene followed by a CLN2 cDNA with an optimized Kozak translation initiation sequence, the cDNA is followed by the polyadenylation sequence from rabbit beta-globin, the expression cassette is surrounded by the inverted terminal repeats of adeno-associated virus 2 |
697808 |
