AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL

SONDHI, D ; PETERSON, D. A ; REDMOND, D. E ; LEOPOLD, P. L ; KAMINSKY, S. M ; HACKETT, N. R ; CRYSTAL, R. G ; GIANNARISI, E. L ; SANDERS, C. T ; MENDEZ, B. S ; DE, B ; ROSTKOWSKI, A. B ; BLANCHARD, B ; BJUGSTAD, K ; SLADEK, J. R

Gene therapy, 2005-11, Vol.12 (22), p.1618-1632 [Periódico revisado por pares]

Basingstoke: Nature Publishing Group

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