Treatment of muscular dystrophy mouse model
Human dystrophin gene is a large gene which is composed of 2.4Mb and 79 exons. We have inserted the gene into the HAC vector, and introduced it to mouse cells. The cell transplantation into a dystrophy mouse model improved the dystrophy symptoms. (PubID:21849666)
Construction of HAC vector carrying human dystrophin
Transplantation of cells carrying dystrophin-HAC
- Extraction of mdxMABs from mdx
- Introduction of DYS-HAC into MABs by MMCT
- Selection of cells containing DYS-HAC, followed by infection of the cells with lenti virus expressing MyoD and nLacZ
- in vitro evaluation (selection of clones, evaluation of proliferation and differentiation)
- After the selection process (as in 4), transplant the cells into SCID/mdx mouse
- Dystrophin gene expression, morphology analysis, and evaluation of functional recovery
Gene expression analysis of cells carrying DYS-HAC
- control mouse expressed dystrohin protein (Dys) (right：Untreated SCID)
- muscular dystrophy mouse model did not show Dys protein expression(center: Untreated SCID/mdx)