Human embryonic stem cell-derived cells rescue visual function in dystrophic RCS rats
Human embryonic stem cell-derived cells rescue visual function in dystrophic RCS rats
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DOI：
10.1089/clo.2006.8.189
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发表时间：
2006-09-01
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影响因子：
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通讯作者：
Lanza, Robert
中科院分区：
文献类型：
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作者：
Lund, Raymond D.;Wang, Shaomei;Lanza, Robert
胚胎干细胞有望为人类临床研究提供具有良好特性和可重复性的替代组织来源。这项技术的早期潜在应用是使用视网膜色素上皮(RPE)治疗视网膜退行性疾病，如黄斑变性。在这里，我们展示了可重复生成的RPE(从18个不同的HES细胞系建立的67种可传代培养物)；来自NIH批准的HES细胞(H9)的批量RPE被测试，并被证明能够在视网膜疾病的动物模型-皇家外科学院(RCS)大鼠中广泛地挽救光感受器，在该动物模型中，光感受器的丢失是由邻近视网膜色素上皮的缺陷引起的。与未经治疗的对照组相比，视觉表现的改善是100%(空间敏锐度大约是正常非营养不良大鼠的70%)，没有不好的病理证据。使用体细胞核移植(SCNT)和/或建立复杂性较低的人类白细胞抗原(HL A)HES-RPE系可以最大限度地减少或消除对免疫抑制药物和/或免疫调节方案的需求。
Embryonic stem cells promise to provide a well-characterized and reproducible source of replacement tissue for human clinical studies. An early potential application of this technology is the use of retinal pigment epithelium (RPE) for the treatment of retinal degenerative diseases such as macular degeneration. Here we show the reproducible generation of RPE (67 passageable cultures established from 18 different hES cell lines); batches of RPE derived from NIH-approved hES cells (H9) were tested and shown capable of extensive photoreceptor rescue in an animal model of retinal disease, the Royal College of Surgeons (RCS) rat, in which photoreceptor loss is caused by a defect in the adjacent retinal pigment epithelium. Improvement in visual performance was 100% over untreated controls (spatial acuity was approximately 70% that of normal nondystrophic rats) without evidence of untoward pathology. The use of somatic cell nuclear transfer (SCNT) and/or the creation of banks of reduced complexity human leucocyte antigen (HLA) hES-RPE lines could minimize or eliminate the need for immunosuppressive drugs and/or immunomodulatory protocols.