肿瘤细胞治疗研究
英文原题：Recombinant AAV gene transfer: Emerging applications in immune modulation and cancer therapy.
使用重组腺相关病毒（rAAV）进行基因转移已取得进展。
重组腺相关病毒（rAAV）基因转移已取得进展。具有不同组织嗜性的rAAV可用于将目标基因安全、准确地递送至特定宿主组织。尽管rAAV载体主要用于治疗单基因疾病，但它们在免疫调节领域也具有潜力。通过rAAV载体进行基因递送，尤其是递送编码免疫调节分子的基因，不仅能减轻炎症、纠正免疫功能障碍，还能通过免疫调节抑制肿瘤生长并增强CAR-T 细胞疗法的疗效，从而发挥抗癌作用。基于rAAV的免疫治疗主要集中于自身免疫性疾病，包括类风湿关节炎、自身免疫性葡萄膜炎、多发性硬化及其他遗传性免疫疾病，但也可能用于治疗肝细胞癌、胶质瘤和卵巢癌。本综述旨在概述rAAV基因递送载体的优势与局限性及其在免疫治疗中的潜在应用。
Advancements have been achieved in gene transfer using recombinant adeno-associated virus (rAAV). rAAVs with distinct tissue tropisms are used to deliver target genes safely and accurately to specific host tissue. Although primarily used for treating monogenic disease, rAAV vectors also have potential in the field of immune modulation. Gene delivery through rAAV vectors, particularly those encoding immune regulatory molecules, not only mitigates inflammation and corrects immune dysfunction but also suppresses tumor growth and enhances the efficacy of Chimeric Antigen Receptor T cell therapy via immune modulation, thereby exerting anti-cancer effects. rAAV-based immune therapies primarily focus on autoimmune diseases, including rheumatoid arthritis, autoimmune uveitis, multiple sclerosis and other genetic immune diseases but may also be used to treat hepatocellular carcinoma, glioma and ovarian cancer. The present review aims to outline the advantages and limitations of rAAV gene delivery vectors and their potential applications in immune therapy.
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