神经退行性疾病干细胞移植治疗PPT课件.ppt

Clinical Trials using ESCs and iPSCs There is also a report of one Japanese patient who received a transplant of a sheet of iPSC-derived RPE Summary on Molecular Mechanism of Stem Cell Transplantation for Neurological Diseases Transplanted cells survive，differentiate to neurons, astrocytes,oligodendrocyte precursors (hESC, hiPSC, NSC ) and release neurological transmittors such as dopamine,Ach. Release of neurotrophic factors (GDNF, GDNE,IGF,) to increase the functions of the endogenous neural stem cells Release of immuno-regulatory factors such as IL-2, 6,8,10 to play immuno-modulation and attenuation of the inflammatory process, such as MSC. The transplanted cells formed synapse with host cells. Others such as delaying the onset and prolonging survival of SOD1 rats Increasing host neurogenesis 今后干细胞治疗神经退行性疾病的临床研究需要考虑的问题 1. Cell Sources： Neural projenitors， MSC， hES cells， iPS cells 2. SC grafting should be conducted to ensure 100,000 dopaminergic neurons （PD）survive per transplantation site. 3. SC grafts should exhibit regulated release of dopamine in line with that of endogenous dopaminergic neurons. 4. By reestablishing the striatal dopaminergic system, grafts should show the capacity to restore functional connectivity within the basal ganglia and at extra-striatal loci. 5. Long-lasting and significant symptom- relief must be achieved （over 2-3 years）. 6. Evaluation System （Symptoms, Dopamine release, Levodopa-response), Unified Parkinson ’s Disea se Ra ting Scal e (UPDRS)，Biomarkers 7. Adverse effects must be minimal. This include s the absence of tumor formation and GIDs （graf t-induced dyskinesia） throughout long-term follow-up periods. 8. Sample Size： over 50-100 patients。 致谢/Acknowledgements 北京大学生命科学研究中心康新江博士 干细胞与再生医学实验室全体科研人员： 王伟，张男，陈超，李森，卢现杰，段婧，吴士超，宋昊，宋娜，陈清法，刘延明 干细胞与再生医学实验室研究团队 Neural Differentiation of hUCB-MSC in Vitro hUCB-MSC 细胞移植改善6-OHDA诱导PD大鼠不对称运动行为 Differentiation of UC-MSC cells into Neurons and Dopamine Neurons 神经退行性疾