AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease

• DOI: 10.1126/scitranslmed.aac6191
• Publication Date: 2015-11-11T19:32:59Z
• Authors: Martin L. Katz; Luis Tecedor; Yonghong Chen; Baye G. Williamson; Elena Lysenko; Fred A. Wininger; Whitney M. Young; Gayle C. Johnson; Rebecca E. H. Whi...; Joan R. Coates; Beverly L. Davidson
• Keywords: Tripeptidyl-Peptidase 1; Genetic Vectors; Dependovirus; Aminopeptidases; Cerebral Ventricles; 3. Good health; Disease Models, Animal; Dogs; Neuronal Ceroid-Lipofuscinoses; Transduction, Genetic; Animals; Enzyme Replacement Therapy; Serine Proteases; Dipeptidyl-Peptidases and Tripeptidyl-Peptidases

AAV-mediated gene transfer to ependymal cells in a dog model of Batten disease provides sustained enzyme replacement and delays disease onset.