Gene therapy for epilepsy by intravascular administration of Adeno-associated virus

2015 Fiscal Year Final Research Report

• Project/Area Number: 25460501
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Experimental pathology
• Research Institution: Jichi Medical University
• Principal Investigator: OGURO KEIJI 自治医科大学, 医学部, 准教授 (90231232)
• Co-Investigator(Kenkyū-buntansha): SHIMAZAKI Kuniko 自治医科大学, 脳神経外科, 非常勤講師 (40142153) ; YOKOTA Hidenori 自治医科大学, 脳神経外科, 講師 (90254929)
• Co-Investigator(Renkei-kenkyūsha): MURAMATSU Shinichi 自治医科大学, 神経内科, 教授 (10239543)
• Project Period (FY): 2013-04-01 – 2016-03-31
• Keywords: てんかん / 遺伝子治療 / ELマウス

Outline of Final Research Achievements

The purpose of this project is to develop a new therapy for the intractable epilepsy patients resistant to the conventional therapies including medication and surgery. We developed a new gene therapy using Adeno Associated Virus (AAV) vector pass through the blood brain barrier (BBB) which enables us to intravascular administration. We performed intracardiac injection of AAV-Neuroligin2 on epileptic EL mice. Neuroligin2 is a synapse related molecule which is known to induce or accelerate the formation of GABAergic synapses. We found that administrated AAV-Neuroligin2 is expressed over the brain including cortex and hippocampus and successfully suppress the seizure.

Free Research Field

脳神経外科