Development of A New Biologics Product (Cell based product) for Lysosomal Storage Disorder

2014 Fiscal Year Final Research Report

• Project/Area Number: 23791208
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Pediatrics
• Research Institution: National Research Institute for Child Health and Development
• Principal Investigator: KANZAKI Seiichi 独立行政法人国立成育医療研究センター, その他部局等, 研究員 (20589741)
• Project Period (FY): 2011-04-28 – 2015-03-31
• Keywords: ライソゾーム / サンドホフ / 再生医療 / 細胞治療 / iPS

Outline of Final Research Achievements

The cell transplant treatment is regard as the only cure cure for the congenital metabolic disroder, and development of the safe and effective new cell transplant treatment are expected. In this study, I examined effectiveness and safety of the cell based product using the in vitro and in vivo models of SD. The hexosaminidase activity and storage in the fibroblast from SD mouse were improved after cocultivation with the iPS cell derived differentiated cells by the mechanism of cross-correction. Next, I transplanted iPS cells product to SD-scid mouse. In 45 days of transplantation, The serum Hex activity was restored to around 5-15% of the normal mouse. It was thought that an evaluation of the effectiveness of this new biologics product (cell processing product) was enabled by evaluating endpoints such as the improvement of the overall survival rate.

Free Research Field

再生医療