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首页> 外文期刊>The European Journal of Neuroscience >Transplantation of human central nervous system stem cells - neuroprotection in retinal degeneration.
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Transplantation of human central nervous system stem cells - neuroprotection in retinal degeneration.

机译:人中枢神经系统干细胞的移植-视网膜变性中的神经保护。

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Stem cells derived from the human brain and grown as neurospheres (HuCNS-SC) have been shown to be effective in treating central neurodegenerative conditions in a variety of animal models. Human safety data in neurodegenerative disorders are currently being accrued. In the present study, we explored the efficacy of HuCNS-SC in a rodent model of retinal degeneration, the Royal College of Surgeons (RCS) rat, and extended our previous cell transplantation studies to include an in-depth examination of donor cell behavior and phenotype post-transplantation. As a first step, we have shown that HuCNS-SC protect host photoreceptors and preserve visual function after transplantation into the subretinal space of postnatal day 21 RCS rats. Moreover, cone photoreceptor density remained relatively constant over several months, consistent with the sustained visual acuity and luminance sensitivity functional outcomes. The novel findings of this study include the characterization and quantification of donor cell radial migration from the injection site and within the subretinal space as well as the demonstration that donor cells maintain an immature phenotype throughout the 7 months of the experiment and undergo very limited proliferation with no evidence of uncontrolled growth or tumor-like formation. Given the efficacy findings and lack of adverse events in the RCS rat in combination with the results from ongoing clinical investigations, HuCNS-SC appear to be a well-suited candidate for cell therapy in retinal degenerative conditions.
机译:在多种动物模型中,已证明源自人脑并作为神经球生长的干细胞(HuCNS-SC)可有效治疗中枢神经退行性疾病。目前正在积累神经退行性疾病中的人类安全性数据。在本研究中,我们探讨了HuCNS-SC在啮齿动物视网膜变性模型,皇家外科医学院(RCS)大鼠中的功效,并扩展了我们以前的细胞移植研究,包括对供体细胞行为和移植后的表型。第一步,我们证明了HuCNS-SC在移植到出生后的第21天RCS大鼠的视网膜下腔中后,可以保护宿主感光器并保留视觉功能。此外,视锥细胞感光器密度在几个月内保持相对恒定,这与持续的视敏度和亮度敏感性功能结果一致。这项研究的新发现包括对供体细胞从注射部位和视网膜下间隙内放射状迁移的表征和定量,并证明了供体细胞在整个实验的7个月中均保持不成熟的表型,并在很短的时间内进行了有限的增殖。没有证据表明生长不受控制或肿瘤样形成。考虑到RCS大鼠的功效发现和不良事件的缺乏,以及正在进行的临床研究的结果,HuCNS-SC似乎是视网膜退行性疾病中细胞治疗的理想人选。

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