AAV vector-mediated cell and gene therapy for neuronal diseases

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To realize novel protein supplementation therapy for neuronal diseases, we have developed systems for AAV vector production and vector-producing cells. By using effective ion-exchange adsorbers, improved purification of vector particles was investigated. In addition to the step-gradient ultracentrifugation procedure, dual ion-exchange procedures enhanced removal of the empty capsids to realize highly-purified vector production at hand. As an application for the AAV-mediated protein supplementation therapy, therapeutic effects of anti-inflammatory treatment on the mice stroke model were successfully demonstrated. Furthermore, vector-producing mesenchymal stem cells to target glioma were developed to target seat of disease with improved anti-tumor effects in vivo.