- 2021 - 2026 BBB通過型GALC発現AAVベクターはクラッベ病マウスの寿命延長効果を持つか
- 2019 - 2022 細胞送達能を高めた改変型酵素を用いるムコ多糖症II型の造血幹細胞遺伝子治療法開発
- 2018 - 2021 Development of Hematopoietic Stem Targeted Gene Therapy for GM1-gangliosidosis
- 2015 - 2019 Preparation of device for mucopolysaccharidosis treatment using iPS cells and gene modification system
- 2015 - 2018 Development of efficient enzyme replacement therapy for lysosomal storage disease with immune modulation
- 2015 - 2018 Improvement of peripheral neuropathy by Adeno-associated virus vector expressing a-galactosidase A in murine Fabry model
- 2014 - 2017 Analysis of the Pathophysiological Mechanism of Bone Lesion in Mucopolysaccharidosis Mouse Model ; Its Natural History and Therapeutic Effect
- 2012 - 2016 Development of effective treatment by immune tolerance induction in enzyme replacement therapy for lysosomal storage diseases
- 2011 - 2012 Generation of iPSCells from human fibroblast with Pompe Disease and Pathological Analysis of the iPS Cells
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