AAV vector-mediated cell and gene therapy for neuronal diseases

2014 Fiscal Year Final Research Report

• Project/Area Number: 22390284
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Single-year Grants
• Section: 一般
• Research Field: Cerebral neurosurgery
• Research Institution: National Center of Neurology and Psychiatry
• Principal Investigator: OKADA Takashi 日本医科大学, 医学(系)研究科(研究院), 教授 (00326828)
• Co-Investigator(Kenkyū-buntansha): KINOH Hiromi 日本医科大学, 大学院医学研究科 (60532728) ; OKADA Hironori 日本医科大学, 大学院医学研究科 (80416271)
• Project Period (FY): 2010-04-01 – 2015-03-31
• Keywords: 神経疾患 / 遺伝子治療 / AAVベクター

Outline of Final Research Achievements

To realize novel protein supplementation therapy for neuronal diseases, we developed an efficient AAV vector production system and vector-producing tumor-targeting cells. By using effective ion-exchange adsorbers, improved GMP production protocol for highly purified vector particles was developed. In addition to the dual ion-exchange procedures, gel filtration chromatography realized ultracentrifugation-free vector purification. As an application for the AAV-mediated protein supplementation therapy, successful transduction of the various disease models was demonstrated. Furthermore, vector-producing tumor-directed stem cells were developed to amplify therapeutic genes.

Free Research Field

脳神経外科学