Printz M, Reynolds J, Mento S J, Jolly D, Kowal K, Sajjadi N
Viagene, Inc., San Diego, CA 92121-1204.
Gene Ther. 1995 Mar;2(2):143-50.
Many protocols for gene therapy employ recombinant retroviral vectors, which are replication-defective retroviruses engineered to serve as gene delivery vehicles. The use of retroviral vectors for human gene therapy requires careful screening of vector-producing cell lines and culture supernatants to ensure the absence of replication competent retrovirus (RCR) in clinical products. In this study we have examined several different culture assays routinely used to test for the presence of RCR. Results indicate that cocultivation of a vector-producing cell line with a permissive cell line can reproducibly detect a low level of contaminating RCR. RCR was detected less frequently in direct tests of cell-free culture supernatants from a contaminated vector-producing line. Further studies revealed that recombinant retroviral vector can interfere, to varying degrees, with the detection of low-level RCR in culture supernatants when a marker rescue assay, an extended mink S+L- assay or a PG-4 S+L- assay is used. Interference can be partially overcome by culturing the vector preparation with a permissive cell line for several days before testing on the indicator cell line. The interference phenomenon we have observed may also occur in other culture assays routinely used for the detection of RCR.
许多基因治疗方案都采用重组逆转录病毒载体,它们是经过工程改造的复制缺陷型逆转录病毒,用作基因传递载体。将逆转录病毒载体用于人类基因治疗,需要仔细筛选载体生产细胞系和培养上清液,以确保临床产品中不存在具有复制能力的逆转录病毒(RCR)。在本研究中,我们检测了几种常用于检测RCR的不同培养试验。结果表明,将载体生产细胞系与许可细胞系共培养能够重复检测到低水平的污染性RCR。在对受污染的载体生产系的无细胞培养上清液进行直接检测时,检测到RCR的频率较低。进一步研究表明,当使用标记拯救试验、扩展水貂S+L-试验或PG-4 S+L-试验时,重组逆转录病毒载体能够不同程度地干扰培养上清液中低水平RCR的检测。在指示细胞系上进行检测之前,将载体制剂与许可细胞系培养几天,可以部分克服这种干扰。我们观察到的干扰现象也可能发生在其他常用于检测RCR的培养试验中。