Ramezani Ali, Hawley Teresa S, Hawley Robert G
Department of Anatomy and Regenerative Biology, The George Washington University Medical Center, Washington, DC 20037, USA.
Stem Cells. 2008 Dec;26(12):3257-66. doi: 10.1634/stemcells.2008-0258. Epub 2008 Sep 11.
Insertional mutagenesis by retroviral vectors has emerged as a serious impediment to the widespread application of hematopoietic stem cell gene transfer for the treatment of hematologic diseases. Here we report the development of a 77-base pair element, FII/BEAD-A (FB), which contains the minimal enhancer-blocking components of the chicken beta-globin 5'HS4 insulator and a homologous region from the human T-cell receptor alpha/delta BEAD-1 insulator. With a new flow cytometry-based assay, we show that the FB element is as effective in enhancer-blocking activity as the prototypical 1.2-kilobase 5'HS4 insulator fragment. When incorporated into the residual U3 region of the 3' long terminal repeat (LTR) of a self-inactivating (SIN) gammaretroviral vector, the FB element was stably transferred to the 5' LTR during reverse transcription, flanking the integrated transgene expression cassette. Notably, using a recently established in vitro insertional mutagenesis assay involving primary murine hematopoietic cells, we found that SIN gammaretroviral vectors, as well as SIN lentiviral vectors, containing the FB element exhibited greatly reduced transforming potential-to background levels under the experimental conditions used-compared with their unshielded counterparts. These results suggest that the FB element-mediated enhancer-blocking modification is a promising approach to dramatically improve the safety of retroviral vectors for therapeutic gene transfer.
逆转录病毒载体介导的插入诱变已成为造血干细胞基因转移广泛应用于治疗血液系统疾病的严重障碍。在此,我们报告了一种77个碱基对元件FII/BEAD-A(FB)的开发,它包含鸡β-珠蛋白5'HS4绝缘子的最小增强子阻断成分以及来自人T细胞受体α/δ BEAD-1绝缘子的同源区域。通过一种基于流式细胞术的新检测方法,我们表明FB元件在增强子阻断活性方面与典型的1.2千碱基5'HS4绝缘子片段一样有效。当FB元件整合到自失活(SIN)γ逆转录病毒载体的3'长末端重复序列(LTR)的残余U3区域时,在逆转录过程中它被稳定转移到5' LTR,位于整合的转基因表达盒两侧。值得注意的是,使用最近建立的涉及原代小鼠造血细胞的体外插入诱变检测方法,我们发现与未屏蔽的对应载体相比,含有FB元件的SINγ逆转录病毒载体以及SIN慢病毒载体在所用实验条件下的转化潜能大大降低至背景水平。这些结果表明,FB元件介导的增强子阻断修饰是一种有望显著提高逆转录病毒载体用于治疗性基因转移安全性的方法。