《生命科学》 2018, 30(3): 248-260

人胚胎干细胞来源的视网膜色素上皮细胞质量控制研究

纳　涛1#，王　磊2，3#，郝　捷2，张可华1，韩晓燕1，周　琪2，3，王　柳2，3*，袁宝珠1*

(1 中国食品药品检定研究院细胞资源保藏研究中心，卫生部生物技术产品检定方法及其标准化重点实验室，北京 100050； 2 中国科学院动物研究所干细胞与生殖生物学国家重点实验室，北京100101；3 中国科学院大学，北京 100049)

摘　要：摘　要：人视网膜色素上皮细胞(retinal pigment epithelial cells， RPEs) 移植是目前被认为最有希望延缓或治愈视网膜变性疾病的治疗策略，而由人胚胎干细胞诱导分化来源的RPE 细胞(human embryonic stem cells-derived RPE， hESC-RPEs) 可为RPEs 移植提供数量充足、质量可控的细胞来源。为确保hESC-RPEs 临床应用的安全性、 有效性和稳定性，这类细胞在临床前研究阶段需满足明确的细胞鉴别特征、RPEs 基本生物学特征、微生物学安全性、生物学安全性及生物学有效性的总体质量要求，而每一类总体质量要求是由多个关键质量属性要求组成。考虑到人ESCs 来源因素，hESC-RPEs 细胞生物学安全性需考虑终未分化的RPEs 中的hESCs 残留问题。现就hESC-RPEs 细胞总体质量要求及各相关质量属性进行讨论，目的是帮助在我国建立临床研究用hESC-RPEs 质量控制评价体系，和探索适合于所有hESCs 诱导分化细胞临床前阶段质量评价的基本方法。

The quality control studies on human embryonic stem cells-derived retinal pigment epithelial cells

NA Tao1#， WANG Lei 2，3#， HAO Jie2， ZHANG Ke-Hua1， HAN Xiao-Yan1， ZHOU Qi 2，3， WANG Liu2，3*， YUAN Bao-Zhu1*

(1 The Cell Collection and Research Center， National Institutes for Food and Drug Control， Key Laboratory of the Ministry of Health for Research on Quality and Standardization of Biotech Products， Beijing 100050， China; 2 State Key Laboratory of Stem Cell and Reproductive Biology， Institute of Zoology， Chinese Academy of Sciences， Beijing 100101， China; 3 University of Chinese Academy of Sciences， Beijing 100049， China)

Abstract: Abstract: Transplantation of human retinal pigmented epithelial cells (RPEs) represents the most promising strategy for treating various retinal degenerative diseases. Human embryonic stem cells-derived RPEs (hESC-RPEs) serve as a sufficient and quality-ensured source of RPEs to be used in treatments. To ensure clinical safety， effectiveness and stability of hESC-RPEs， the hESC-RPEs must meet four categories of key quality requirements， which are the general cell biology features， microbiology safety， biology safety and biological effectiveness， during preclinical stage of product development. Each key quality requirement consists of a group of inter-supportive key quality attributes. In this review， we discussed the key quality requirements and the associated key quality attributes with goals of establishing a comprehensive quality assessment system in China for hESC-RPEs to be used in patients as well as developing a common methodology for evaluating the qualities of all hESC-derived cells required before clinical uses.