2012 Fiscal Year Final Research Report

Gene Therapy for Fabry disease: development of a miniature swine model

Research Project

Project/Area Number	22590303
Research Category	Grant-in-Aid for Scientific Research (C)
Allocation Type	Single-year Grants
Section	一般
Research Field	Human genetics
Research Institution	Kagoshima University
Principal Investigator	TAKENAKA Toshihiro 鹿児島大学, 大学院・医歯学総合研究科, 特任准教授 (70363645)
Co-Investigator(Kenkyū-buntansha)	HIGUCHI Koji 鹿児島大学, 大学院・医歯学総合研究科, 特任助教 (90448580) YOSHIMITSU Makoto 鹿児島大学, 大学院・医歯学総合研究科, 特任助教 (70404530)
Project Period (FY)	2010 – 2012
Keywords	遺伝子治療 / 心 Fabry 病 / Fabry 病 / ミニブタ / 造血幹細胞
Research Abstract	The aim of this study is to develop a miniature swine model forgene therapy for Fabry disease. We tried to collect hematopoietic stem cells from bonemarrow or G-CSF mobilized peripheral blood mononuclear cells, however G-CSF mobilized apherisis method only allowed us to collect enough number of peripheral blood mononuclear cells. We also tried to establish mouse anti-miniature swine CD34 peptide antibody, however the cells recognized by the antibody did not grow in cytokine containing methlycellulose plate. Further characterization of miniature swine hematopoietic stem cell need to be clarified for utilizing this model in gene therapy preclinical mode.

(4 results)