Development of regenerative medicine and cell therapy for inherited metabolic diseases using hypophosphatasia mice

2015 Fiscal Year Final Research Report

• Project/Area Number: 25461564
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Nippon Medical School
• Principal Investigator: Iijima Osamu 日本医科大学, 医学部, 助教 (40466206)
• Co-Investigator(Kenkyū-buntansha): MIYAKE Koichi 日本医科大学, 医学部, 准教授 (90267211) ; SHIMADA Takashi 日本医科大学, 医学部, 教授 (20125074)
• Co-Investigator(Renkei-kenkyūsha): WATANABE Atsushi 日本医科大学, 医学部, 准教授 (10307952)
• Project Period (FY): 2013-04-01 – 2016-03-31
• Keywords: 再生・細胞医療 / 遺伝子治療 / 低ホスファターゼ症 / 骨髄幹細胞 / 造血幹細胞 / 骨格筋芽細胞 / レンチウイルスベクター / アルカリホスファターゼ

Outline of Final Research Achievements

We evaluated the feasibility of bone marrow stem cell-based enzyme replacement therapy (ERT) for hypophosphatasia model mice (Akp2-/- mice). Bone marrow stem cells transduced with bone-targeted form of tissue-nonspecific alkaline phosphatase (TNALP-D10) gene was intravenously transplanted into neonatal Akp2-/- mice on day 2 after birth. The treated Akp2-/- mice showed significantly prolonged survival with improved bone mineralization. These results provided us important insight regarding development of novel regenerative and cell therapy for inherited metabolic diseases including hypophosphatasia.

Free Research Field

医歯薬学