Gene therapy is increasingly used in the clinic, but specific delivery to kidney cells remains an important obstacle to broader implementation in nephrology. In a new study, Tobias Huber and colleagues describe a new adeno-associated virus (AAV) vector that specifically targets glomerular cells.

To test the therapeutic potential of AAV2-GEC, the researchers used it to carry the gene encoding IdeS, the Streptococcus pyogenes protease that cleaves IgG heavy chains. In a mouse model of glomerulonephritis induced by injecting anti-glomerular basement membrane (GBM) sheep serum, treatment with AAV2-GEC-IdeS one day after induction was renoprotective. Treated mice had lower and less persistent albuminuria, and weaker deposition of mouse and sheep IgG on the GBM, compared with control mice injected with AAV2-GEC without IdeS.

基因治疗在临床上得到越来越多的应用，但特异性递送至肾细胞仍然是肾病学更广泛实施的重要障碍。在一项新研究中，Tobias Huber 及其同事描述了一种专门针对肾小球细胞的新型腺相关病毒 （AAV） 载体。

为了测试 AAV2-GEC 的治疗潜力，研究人员用它来携带编码 IdeS 的基因，IdeS 是裂解 IgG 重链的化脓性链球菌蛋白酶。在注射抗肾小球基底膜 （GBM） 绵羊血清诱导的肾小球肾炎小鼠模型中，诱导后 1 天用 AAV2-GEC-IdeS 治疗具有肾脏保护作用。与注射不含 IdeS 的 AAV2-GEC 的对照小鼠相比，治疗小鼠的白蛋白尿较低且持久性较低，小鼠和绵羊 IgG 在 GBM 上的沉积较弱。