Stroncek David F, Jin Ping, Wang Ena, Ren Jiagiang, Sabatino Marianna, Marincola Francesco M
Department of Transfusion Medicine, Clinical Center, National Institutes of Health, Bethesda, Maryland 20892, USA.
Mol Diagn Ther. 2009;13(3):181-93. doi: 10.2165/01250444-200913030-00003.
Potency testing is an important part of the evaluation of cellular therapy products. Potency assays are quantitative measures of a product-specific biologic activity that is linked to a relevant biologic property and, ideally, a product's in vivo mechanism of action. Both in vivo and in vitro assays can be used for potency testing. Since there is often a limited period of time between the completion of production and the release from the laboratory for administration to the patient, in vitro assays such as flow cytometry, ELISA, and cytotoxicity are typically used. Better potency assays are needed to assess the complex and multiple functions of cellular therapy products, some of which are not well understood. Gene expression profiling using microarray technology has been widely and effectively used to assess changes of cells in response to stimuli and to classify cancers. Preliminary studies have shown that the expression of non-coding microRNA (miRNA), which plays an important role in cellular development, differentiation, metabolism, and signal transduction, can distinguish between different types of stem cells and leukocytes. Both gene and miRNA expression profiling have the potential to be important tools for testing the potency of cellular therapies. Potency testing, the complexities associated with potency testing of cellular therapies, and the potential role of gene and miRNA expression microarrays in potency testing of cellular therapies are discussed.
效力测试是细胞治疗产品评估的重要组成部分。效力测定是对与相关生物学特性相关联的产品特异性生物活性的定量测量，理想情况下，也是对产品体内作用机制的定量测量。体内和体外测定均可用于效力测试。由于从生产完成到从实验室放行以供患者使用之间的时间通常有限，因此通常使用体外测定，如流式细胞术、酶联免疫吸附测定（ELISA）和细胞毒性测定。需要更好的效力测定方法来评估细胞治疗产品复杂多样的功能，其中一些功能尚未完全了解。使用微阵列技术的基因表达谱分析已被广泛有效地用于评估细胞对刺激的反应变化以及癌症分类。初步研究表明，在细胞发育、分化、代谢和信号转导中起重要作用的非编码微小RNA（miRNA）的表达可以区分不同类型的干细胞和白细胞。基因和miRNA表达谱分析都有可能成为测试细胞治疗效力的重要工具。本文将讨论效力测试、细胞治疗效力测试相关的复杂性以及基因和miRNA表达微阵列在细胞治疗效力测试中的潜在作用。
