自身免疫性脑炎（autoimmune encephalitis，AE）是一类由免疫系统攻击中枢神经系统所导致的炎症性疾病，常见临床表现包括精神行为异常、认知与运动障碍以及癫痫发作等。传统一线治疗主要依赖糖皮质激素、静脉注射免疫球蛋白和血浆置换，然而部分患者治疗效果不佳或复发率较高，迫切需要开发新的治疗策略。近年来，靶向治疗在AE领域进展迅速，其策略主要针对B细胞、T细胞、补体系统及关键炎症因子等免疫环节。抗CD20单克隆抗体已在难治性AE中显示出良好的临床疗效；诸如奥瑞珠单抗、依帕珠单抗、泰它西普及达雷妥尤单抗等新型药物，进一步为B细胞及浆细胞的精准清除提供了新策略。同时，靶向细胞因子的药物、Janus激酶抑制剂及蛋白酶体抑制剂硼替佐米等在调节异常免疫应答中表现出潜在价值；嵌合抗原受体T细胞免疫疗法（chimeric antigen receptor T cell immuno-therapy，CAR-T）也逐步成为前沿探索的重要方向。展望未来，结合神经修复策略与人工智能辅助诊疗决策，靶向治疗有望推动AE从传统的经验性免疫抑制迈向精准干预的新阶段，从而实现更高效、个体化的疾病管理。

Autoimmune encephalitis (AE) is a group of inflammatory disorders caused by immune-mediated attacks on the central nervous system, with common clinical manifestations including psychiatric and behavioral abnormalities, cognitive and motor impairments and epileptic seizures. Conventional first-line therapies mainly rely on corticosteroids, intravenous immunoglobulin, and plasma exchange. However, a subset of patients exhibits suboptimal responses or high relapse rates, underscoring the urgent need for the development of novel therapeutic strategies. In recent years, targeted therapeutic approaches have advanced rapidly in the field of AE, primarily focusing on key immune components such as B cells, T cells, the complement cascade, and key inflammatory cytokines. Anti-CD20 monoclonal antibodies have demonstrated promising efficacy in refractory AE. Moreover, emerging agents such as ocrelizumab, epratuzumab, telitacicept, and daratumumab have provided refined strategies for the precise depletion of B cells and plasma cells. In parallel, cytokine-targeting biologics, Janus kinase inhibitors, and the proteasome inhibitor bortezomib have shown potential value for modulating aberrant immune responses. In addition, chimeric antigen receptor (CAR)-T cell immunotherapy is gradually emerging as an important frontier in exploratory research. Looking ahead, by integrating neurorestorative strategies with artificial intelligence-assisted diagnostic and therapeutic decision-making, targeted therapies are expected to drive AE management from traditional empirical immunosuppression toward a new era of precision intervention, thereby enabling more efficient and personalized disease management.