Development of alternative gene transfer techniques for

Gene therapy have recently attracted much attention as a curative therapeutic option for inherited single gene disorders such as hemophilia. Hemophilia is a hereditary bleeding disorder caused by the deficiency of clotting activity of factor VIII (FVIII) or factor IX (FIX), and gene therapy for hemophilia using viral vector have been vigorously investigated worldwide. Toward further advancement of gene therapy for hemophilia, we have previously developed and validated the efficacy of novel two types of gene transfer technologies using a mouse model of hemophilia A. Here we investigated the efficacy and safety of the technologies in canine model. Especially, validations of technical procedures of the gene transfers for dogs were focused. Green fluorescence protein (GFP) gene were transduced into normal beagle dogs by No major adverse events were observed during the gene transfers in both gene transfer systems. As for Two types of gene transfer techniques were successfully applied to a canine model, and the transduced gene expressions persisted for a long term. Toward clinical application for hemophilia patients, practical assessments of therapeutic efficacy of these techniques will need to be performed using a dog model of hemophilia and FVIII (or FIX) gene.

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Déclaration de conflit d'intérêts

Teruo Okano, Ph.D. is a stockholder of CellSeed Inc. which has licenses for certain cell sheet-related technologies and patents from Tokyo Women's Medical University. Other authors have no interests to declare.