Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases.
Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases.
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DOI：
10.1089/hum.2016.088
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发表时间：
2016-10
影响因子：
4.2
通讯作者：
SiddiqiFaez;H. WolfeJohn
中科院分区：
文献类型：
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作者：
SiddiqiFaez;H. WolfeJohn
具有遗传病因的神经系统疾病导致整个CNS的神经细胞的损失或功能障碍。目前，大多数神经遗传性疾病的治疗选择很少。干细胞移植（SCT）到中枢神经系统有可能成为一种有效的治疗方式，因为祖细胞可以取代病变脑中丢失的细胞，提供多种营养因子，或提供缺失的蛋白质。本文综述了SCT在溶酶体贮积病（LSDs）中的应用，LSDs是一组具有突出CNS疾病的单基因疾病。在大多数患者中，CNS疾病导致智力残疾，这是目前标准治疗难治的。在啮齿动物LSD模型中进行了大量的脑定向SCT临床前工作。已经用于直接递送到CNS中的细胞类型包括神经干细胞、胚胎和诱导多能干细胞以及间充质干细胞。造血干细胞是一种有效的治疗方法...
Neurological diseases with genetic etiologies result in the loss or dysfunction of neural cells throughout the CNS. At present, few treatment options exist for the majority of neurogenetic diseases. Stem cell transplantation (SCT) into the CNS has the potential to be an effective treatment modality because progenitor cells may replace lost cells in the diseased brain, provide multiple trophic factors, or deliver missing proteins. This review focuses on the use of SCT in lysosomal storage diseases (LSDs), a large group of monogenic disorders with prominent CNS disease. In most patients the CNS disease results in intellectual disability that is refractory to current standard-of-care treatment. A large amount of preclinical work on brain-directed SCT has been performed in rodent LSD models. Cell types that have been used for direct delivery into the CNS include neural stem cells, embryonic and induced pluripotent stem cells, and mesenchymal stem cells. Hematopoietic stem cells have been an effective therapy ...