2018 ISPD Conference

Set up an amniotic fluid stem cell-derived induced pluripotent stem cells disease model and in utero therapy for spina bifida
S.W. Steven Shaw
Taipei Chang Gung Memorial Hospital, Taipei Chang Gung Memorial Hospital, Taipei, Taiwan
Objectives
The amniotic fluid stem cells (AFS) is a potential source of induced pluripotent stem (iPS) cells. The advantages of AFS-iPS cells when a genetic disorder is detected and the use of cells differentiated from AFS-iPS cells as a source for autologous cell therapy pre- or postnatally. Spina bifida (SB) is the most common neural tube defect disease with huge burden for society health insurance. We have set up the animal model of spina bifida in pregnant rodent. 
Methods
Human SB and normal control AF will be collected from amniocentesis. AFS cells were underwent selection for C-kit+ population and then were reprogrammed into iPS cells using lentiviral vector encoding Yamanaka factors. AFS-iPS cells derived from different diseases were studied for potential treatment, drug investigation and disease modeling. The animal model was induced via retinoid acid to achieve 60-80% of SB fetal mice. 
Results
Hundred thousand human AFS or AFS-iPS cells were injected into the amniotic cavity of each pup of pregnant mouse at E14 locally around the SB defect. Engraftment and long-term motoneural function studies showed the evidence in major organs including liver, muscles, and spinal cord with improvement of ability of exercise. The differentiation into neural stem cells was successfully induced from AFS / AFS-iPS cells and demonstrated the local effect in SB mice. 
Conclusions
The development of AFS/ AFS-iPS cell lines from SB could accelerate the development of existing targets for different diseases after prenatal diagnosis. Prenatal transplantation of stem cells could be the possible way to improve the clinical outcome of spina bifida.