Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA - Worgall, Stefan; Sondhi, Dolan; Hackett, Neil R; Kosofsky, Barry; Kekatpure, Minal V; Neyzi, Nurunisa; Dyke, Jonathan P; Ballon, Douglas; Heier, Linda; Greenwald, Bruce M; Christos, Paul; Mazumdar, Madhu; et al. (Human gene therapy, 2008)
Keywords: Aminopeptidases; Antibodies, Viral - blood; Central Nervous System; Child; Child, Preschool; Dependovirus - immunology; Dipeptidyl-Peptidases and Tripeptidyl-Peptidases; Disease Progression; DNA, Complementary - genetics; Endopeptidases - genetics; Female; Follow-Up Studies; Genetic Therapy - methods; Genetic Vectors - adverse effects; Genetic Vectors - immunology; Humans; Magnetic Resonance Imaging; Male; Neuronal Ceroid-Lipofuscinoses - therapy; Serine Proteases