Cellular engineering for treatment of animal model for inherited brain disorder

1)Krabbe病的发病机制及动物模型的建立：培养Krabbe病的动物模型Twitcher‘s Brain，并在培养的少突胶质细胞上观察其形态和生化变化。抽动肌组织中聚合胞苷的积累量是对照的100倍。神经素的蓄积改变了膜信号转导系统。2)Niemann-Pick小鼠模型：小鼠体内的Niemann-Pick小鼠相当于人类的Niemann-Pick C型小鼠，因为该突变小鼠体内积累了游离胆固醇和各种糖脂。这些脂质的积累与高尔基体-溶酶体膜复合体密切相关。因此，我们推测该突变小鼠的异常可能是由溶酶体膜转运系统引起的。3)将神经生长因子基因导入神经母细胞瘤细胞。成功地将NGF受体基因导入神经母细胞瘤细胞，发现神经细胞呈良性突起。

1) Pathogenesis and treatment using animal model of Krabbe's disease : Animal inodel of Krabbe disease, named twitcher's brain was cultured and chased morphological and biochemical changes in oligodendroglial cell cultures. Pyschosine was accumulated 100 times more than control in twitcher tissues. the accumulation of psychosine alters membrane signal transduction system. 2) Niemann-Pick mouse model : Niemann-Pick mouse in mouse is equivalent to human Niemann-Pick type C, since free cholesterol and various glycolipids were accumulated in this mutant mice. The accumulation of these lipids are closely related to Golgi-lysosomal membrane complexes. Therefore, we speculate that abnormality of this mutant mice may be caused by lysosomal membrane transport system. 3) Introduction of nerve growth factor gene into neuroblastma cells. We succeeded transfection of NGF receptor gene intoneuroblastma cells and found benign process of neural cells.