Tibbles Lee Anne, Spurrell Jason C L, Bowen Gloria P, Liu Qiang, Lam Mindy, Zaiss Anne K, Robbins Stephen M, Hollenberg Morley D, Wickham Thomas J, Muruve Daniel A
Department of Medicine. Libin Gene Therapy Unit, University of Calgary Calgary, Alberta T2N 4N1, Canada.
J Virol. 2002 Feb;76(4):1559-68. doi: 10.1128/jvi.76.4.1559-1568.2002.
The use of adenovirus vectors for human gene therapy is limited by potent inflammatory responses that result in significant morbidity. In kidney-derived epithelial cells (REC), activation of extracellular signal-regulated kinase 1/2 (ERK) and p38 kinase (p38) pathways occurred within 20 min of transduction with the serotype 5 adenovirus vector AdCMV beta gal. Inhibition of ERK and p38 with U0126 and SB203580, respectively, reduced the expression of IP-10 mRNA following transduction with AdCMV beta gal. To determine the role of the coxsackievirus-adenovirus receptor (CAR) or alpha(v) integrins in the activation of ERK and p38 and the expression of IP-10, REC cells were transduced with the fiber-modified and RGD-deleted adenovirus vectors AdL.F(RAEK-HA) and AdL.PB(HA), respectively. Compared with the wild-type capsid vector Ad5Luc, transduction with AdL.F(RAEK-HA) and AdL.PB(HA) resulted in reduced ERK-p38 activation and less IP-10 mRNA expression. The decreased IP-10 expression induced by the tropism-modified vectors was due to diminished transduction, since increasing multiplicity of infection resulted in increased IP-10 expression. Inhibition of adenovirus penetration with bafilomycin A1 or ammonium chloride attenuated the activation of ERK-p38 and IP-10 mRNA expression following infection, suggesting that endosomal escape was required to trigger these pathways. In vivo, direct inhibition of ERK and p38 signaling pathways inhibited adenovirus vector-induced IP-10 expression in mouse liver 1 h following transduction. These results demonstrate the importance of signaling via ERK and p38 in the early host response to adenovirus vectors and will permit the development of novel strategies to improve the safety and efficacy of these agents in human gene therapy.
腺病毒载体用于人类基因治疗受到强烈炎症反应的限制,这种反应会导致显著的发病率。在肾源性上皮细胞(REC)中,用5型腺病毒载体AdCMVβ半乳糖苷酶转导后20分钟内,细胞外信号调节激酶1/2(ERK)和p38激酶(p38)信号通路被激活。分别用U0126和SB203580抑制ERK和p38,可降低AdCMVβ半乳糖苷酶转导后IP-10 mRNA的表达。为了确定柯萨奇病毒-腺病毒受体(CAR)或α(v)整合素在ERK和p38激活以及IP-10表达中的作用,分别用纤维修饰且缺失RGD的腺病毒载体AdL.F(RAEK-HA)和AdL.PB(HA)转导REC细胞。与野生型衣壳载体Ad5Luc相比,用AdL.F(RAEK-HA)和AdL.PB(HA)转导导致ERK-p38激活减少,IP-10 mRNA表达降低。嗜性修饰载体诱导的IP-10表达降低是由于转导减少,因为增加感染复数会导致IP-10表达增加。用巴弗洛霉素A1或氯化铵抑制腺病毒穿透可减弱感染后ERK-p38的激活和IP-10 mRNA的表达,这表明内体逃逸是触发这些信号通路所必需的。在体内,转导后1小时直接抑制ERK和p38信号通路可抑制腺病毒载体诱导的小鼠肝脏IP-10表达。这些结果证明了ERK和p38信号在宿主对腺病毒载体早期反应中的重要性,并将有助于开发新策略以提高这些药物在人类基因治疗中的安全性和有效性。