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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 DA, Giannaris EL, Sanders CT, Mendez BS, De B, Rostkowski AB, Blanchard B, Bjugstad K, Sladek JR, Redmond DE, Leopold PL, Kaminsky SM, Hackett NR, Crystal RG. AAV2-mediated CLN2 gene transfer to rodent and non-human primate brain results in long-term TPP-I expression compatible with therapy for LINCL. Gene Ther. 2005 Nov; 12(22):1618-32.

View in: PubMed