Blood to brain to the rescue.
Blood to brain to the rescue.
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血液注入大脑来救援。
DOI：
10.1172/jci21476
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发表时间：
2004
期刊：
影响因子：
--
通讯作者：
Wu,Yun-Ping
中科院分区：
文献类型：
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作者：
Proia,RichardL;Wu,Yun-Ping
神经退行性变发生在40多种已知的溶酶体贮积病中。由于这些疾病的神经系统可在全球范围内受到影响，因此有效的治疗需要持续广泛的矫正。Biffi等人表明，在异色性白质营养不良小鼠模型中，通过移植经过基因修饰的过表达缺失溶酶体酶的造血细胞，这种纠正是可能的。研究结果揭示了一种神经系统损伤反应途径，可以用来治疗这些严重疾病的神经系统。
Neurodegeneration occurs in the majority of the more than 40 known lysosomal storage diseases. Since the nervous system in these disorders can be globally affected, effective treatment would require persistent widespread correction. Biffi et al. show such correction is possible in a mouse model of metachromatic leukodystrophy by the transplantation of hematopoietic cells genetically modified to overexpress the missing lysosomal enzyme. The results reveal a nervous system damage-response pathway that can be harnessed to provide therapy to the nervous system in these serious disorders.