Chen Li-Guo, Wang Zheng-Rong, Wan Chao-Min, Chao Lee, Chao Julie, Xing Hao-Yang
School of Basic Medicine and Forensics, Sichuan University, Chengdu, 610041, P.R. China.
J Gene Med. 2004 Jul;6(7):786-97. doi: 10.1002/jgm.563.
The success of any gene-therapy approach depends on the survival of the genetically engineered cells that are implanted in the patient to deliver the therapeutic product. Immunoisolation of nonautologous cells within a microcapsule is a unique approach for gene therapy.
We employed an immunoisolation device that protects nonautologous cells from destruction, to implant human atrial natriuretic peptide (hANP)-producing Chinese hamster ovary (CHO) cells in two-kidney, one-clip (2K1C) hypertensive rats. CHO cells transfected with the plasmid CMV-cANP were encapsulated in biocompatible polycaprolactone (PCL) capsules, and then the PCL capsules were implanted into 2K1C hypertensive rats intraperitoneally.
The implantation of encapsulated hANP-producing cells caused a significant delay of blood pressure (BP) increase 2 weeks post-implantation and the effect lasted for more than 5 months. The implantation of encapsulated hANP-producing cells also caused significant increases in renal blood flow (RBF), glomerular filtration rate (GFR), sodium output, urine excretion, and urinary cGMP levels. These beneficial effects were reflected morphologically by an attenuation of the glomerular sclerotic lesions, reduction in cardiomyocyte size, tubular injury and renal arterial thickening. Immunoreactive hANP can be detected in the blood of 2K1C rats after implantation of the PCL capsules containing hANP-producing cells.
This study demonstrates the usefulness of encapsulated ANP gene transfected cells as a new tool for ANP gene delivery in studying renovascular hypertension and cardiovascular diseases. Thus, our results may have important implications for clinical use of transgene cells as therapeutic agents in the treatment of cardiovascular diseases.
任何基因治疗方法的成功都取决于植入患者体内以递送治疗性产品的基因工程细胞的存活。在微胶囊内对非自体细胞进行免疫隔离是一种独特的基因治疗方法。
我们采用一种保护非自体细胞免受破坏的免疫隔离装置,将产生人心房钠尿肽(hANP)的中国仓鼠卵巢(CHO)细胞植入双肾单夹(2K1C)高血压大鼠体内。将用质粒CMV-cANP转染的CHO细胞封装在生物相容性聚己内酯(PCL)胶囊中,然后将PCL胶囊经腹腔植入2K1C高血压大鼠体内。
植入封装的产生hANP的细胞导致植入后2周血压(BP)升高显著延迟,且该效果持续超过5个月。植入封装的产生hANP的细胞还导致肾血流量(RBF)、肾小球滤过率(GFR)、钠排出量、尿排泄量和尿cGMP水平显著增加。这些有益作用在形态学上表现为肾小球硬化病变减轻、心肌细胞大小减小、肾小管损伤和肾动脉增厚。在植入含有产生hANP细胞的PCL胶囊后,可在2K1C大鼠的血液中检测到免疫反应性hANP。
本研究证明了封装的ANP基因转染细胞作为一种在研究肾血管性高血压和心血管疾病中进行ANP基因递送的新工具的有效性。因此,我们的结果可能对转基因细胞作为治疗剂在心血管疾病治疗中的临床应用具有重要意义。