A single intravenous rAAV injection as late as P20 achieves efficacious and sustained CNS Gene therapy in Canavan mice.
Ahmed SS, Li H, Cao C, Sikoglu EM, Denninger AR, Su Q, Eaton S, Liso Navarro AA, Xie J, Szucs S, Zhang H, Moore C, Kirschner DA, Seyfried TN, Flotte TR, Matalon R, Gao G.
Ahmed SS, et al. Among authors: matalon r.
Mol Ther. 2013 Dec;21(12):2136-47. doi: 10.1038/mt.2013.138. Epub 2013 Jul 2.
Mol Ther. 2013.
PMID: 23817205
Free PMC article.