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Stem cells in clinical trials for treatment of retinal degeneration

机译：干细胞治疗视网膜变性的临床试验

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Introduction: After decades of basic science research involving the testing of regenerative strategies in animal models of retinal degenerative diseases, a number of clinical trials are now underway, with additional trials set to begin shortly. These efforts will evaluate the safety and preliminary efficacy of cell-based products in the eyes of patients with a number of retinal conditions, notably including age-related macular degeneration, retinitis pigmentosa and Stargardt's disease.Areas covered: This review considers the scientific work and early trials with fetal cells and tissues that set the stage for the current clinical investigatory work, as well the trials themselves, specifically those either now completed, underway or close to initiation. The cells of interest include retinal pigment epithelial cells derived from embryonic stem or induced pluripotent stem cells, undifferentiated neural or retinal progenitors or cells from the vascular/bone marrow compartment or umbilical cord tissue.Expert opinion: Degenerative diseases of the retina represent a popular target for emerging cell-based therapeutics and initial data from early stage clinical trials suggest that short-term safety objectives can be met in at least some cases. The question of efficacy will require additional time and testing to be adequately resolved.
机译：简介：经过数十年的基础科学研究，包括在视网膜退行性疾病的动物模型中测试再生策略，现在正在进行许多临床试验，并且即将开始其他试验。这些努力将评估许多视网膜疾病患者眼中基于细胞的产品的安全性和初步疗效，特别是与年龄相关的黄斑变性，色素性视网膜炎和Stargardt病患者。胎儿细胞和组织的早期试验为当前的临床研究工作奠定了基础，以及试验本身，特别是那些已经完成，正在进行或即将开始的试验。感兴趣的细胞包括源自胚胎干或诱导性多能干细胞的视网膜色素上皮细胞，未分化的神经或视网膜祖细胞或来自血管/骨髓腔或脐带组织的细胞。专家意见：视网膜退行性疾病代表了流行的靶标对于新兴的基于细胞的疗法，早期临床试验的初步数据表明，至少在某些情况下可以达到短期安全目标。效力问题将需要额外的时间和测试才能得到充分解决。

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