胚胎干细胞对黄斑变性试验:初步报告
Steven D Schwartz1, Jean-Pierre Hubschman, Gad Heilwell
1Jules Stein Eye Institute Retina Division, Department of Ophthalmology, David Geffen School of Medicine, University of California, Los Angeles, CA 90095, USA.
Lancet (London, England)
|January 28, 2012
概括
人类胚胎干细胞衍生的视网膜色素表皮质 (hESC-RPE) 细胞被移植到患有黄斑缩症的患者体内. 在早期临床试验中,hESC-RPE细胞显示出安全性和潜在的视力改善.
科学领域:
- 眼科医生 眼科 眼科
- 再生医学是一种再生医学.
- 干细胞生物学 干细胞生物学
背景情况:
- 人类胚胎干细胞 (hESCs) 在13年前被发现.
- 这份报告详细介绍了首次将hESC衍生细胞移植到人类患者身上.
- 这项研究侧重于hESC衍生的视网膜色素上皮质 (RPE),用于治疗失明症.
研究的目的:
- 评估hESC衍生的RPE的亚皮移植的安全性和耐受性.
- 评估hESC-RPE在患有Stargardt黄斑缩和干老年性黄斑退行症的患者中的潜在疗效.
- 建立hESC-RPE移植的临床方案.
主要方法:
- 开始了两个前性临床研究 (临床试验.gov:NCT01345006,NCT01344993).
- 超99%纯度,受控的hESC分化的RPE细胞的视网膜下移植.
- 术前和术后的眼科检查,包括视力敏度,光素血管学,OCT和视野测试.
主要成果:
- 移植的hESC-RPE细胞集成到宿主RPE层中并持续存在.
- 在4个月内,没有观察到过度扩散,瘤性或免疫排斥的迹象.
- 两位患者的视觉敏度都得到改善;Stargardt的患者从手部运动改善到20/800,而AMD患者从21到28个ETDRS字母得到改善.
结论:
- 由hESC衍生的RPE细胞在患有黄斑缩症的患者中是安全的,并且耐受性良好.
- 早期证据表明,视力改善和光受体的救援是有可能的.
- 未来的治疗策略旨在更早地干预疾病进展.
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