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Human embryonic stem cell-derived retinal pigment epithelium transplants as a potential treatment for wet age-related macular degeneration

机译:人类胚胎干细胞衍生的视网膜色素上皮移植物作为湿性老年性黄斑变性的潜在治疗方法

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Stem cell therapy may provide a safe and promising treatment for retinal diseases. Wet age-related macular degeneration (wet-AMD) is a leading cause of blindness in China. We developed a clinical-grade human embryonic stem cell (hESC) line, Q-CTS-hESC-2, under xeno-free conditions that differentiated into retinal pigment epithelial cells (Q-CTS-hESC-2-RPE). A clinical trial with three wet-AMD patients was initiated in order to study the safety and tolerance to Q-CTS-hESC-2-RPE cell transplants. The choroidal neovascularization membrane was removed and then a suspension of 1?×?106 Q-CTS-hESC-2-RPE cells were injected into a subfoveal pocket. The patients were followed for 12 months during which no adverse effects resulting from the transplant were observed. Anatomical evidence suggested the existence of new RPE-like cell layer in the previously damaged area. Visual and physiological testing indicated limited functional improvement, albeit to different degrees between patients. This study provides some promising early results concerning the use of transplanted hESC-RPE cells to alleviate wet-AMD.
机译:干细胞疗法可为视网膜疾病提供安全且有希望的治疗方法。与年龄相关的湿性黄斑变性(wet-AMD)是中国失明的主要原因。我们在无异种条件下开发了临床级人类胚胎干细胞(hESC)系Q-CTS-hESC-2,该细胞分化为视网膜色素上皮细胞(Q-CTS-hESC-2-RPE)。为了研究对Q-CTS-hESC-2-RPE细胞移植的安全性和耐受性,开始了三名湿性AMD患者的临床试验。去除脉络膜新血管形成膜,然后将1××10 10 6 sup Q-CTS-hESC-2-RPE细胞的悬浮液注入到小凹下袋中。随访患者12个月,未观察到移植引起的不良反应。解剖学证据表明,在先前受损的区域中存在新的RPE样细胞层。视觉和生理测试表明功能改善有限,尽管患者之间程度不同。这项研究提供了有关使用移植的hESC-RPE细胞缓解湿性AMD的一些有希望的早期结果。

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