肿瘤细胞治疗研究
英文原题：RNA-Based Therapeutic Strategies in Multiple Myeloma: From Molecular Targets to Delivery and Clinical Translation.
多发性骨髓瘤（MM）是一种治疗棘手的血液系统恶性肿瘤，以克隆性浆细胞增殖为特征，常在全球范围内导致显著的发病率和死亡率。
多发性骨髓瘤（MM）是一种棘手的血液系统恶性肿瘤，其特征为浆细胞克隆增殖，在全球常导致显著发病和死亡。尽管化疗和CAR-T疗法有所进展，MM仍无法治愈，原因包括肿瘤异质性、免疫逃逸及微环境重塑，并受到细胞因子释放综合征和骨髓抑制等毒性的加剧。这一迫切未满足需求要求创新策略。本综述评估用于调节MM的前沿RNA疗法，并综合mRNA疫苗、小干扰RNA（siRNA）、反义寡核苷酸（ASO）和微小RNA（miRNA）模拟物/抑制剂等治疗方式的临床前及临床证据。我们还探讨RNA工程化细胞疗法，例如瞬时CAR-T平台以及利用脂质纳米颗粒递送、靶向骨髓生态位的系统。综合这些见解，我们强调RNA技术具有变革性潜力，可实现持久缓解、克服耐药并降低成本，为复发难治性MM的个体化、更安全治疗铺平道路。
Multiple myeloma (MM) is a challenging hematologic malignancy characterized by clonal plasma cell proliferation, often leading to significant morbidity and mortality worldwide. Despite advances in chemotherapy and CAR-T therapies, MM remains incurable due to tumor heterogeneity, immune evasion, and microenvironment remodeling-exacerbated by toxicities like cytokine release syndrome and myelosuppression. This urgent unmet need demands innovative strategies. In this review, we assess cutting-edge RNA-based therapeutics for MM modulation, drawing on preclinical and clinical evidence on modalities including mRNA vaccines, small interfering RNAs (siRNAs), antisense oligonucleotides (ASOs), and microRNA (miRNA) mimics/inhibitors. We further explore RNA-engineered cell therapies, such as transient CAR-T platforms and lipid nanoparticle-delivered systems targeting the bone marrow niche. By integrating these insights, we underscore RNA technologies' transformative potential to achieve durable remissions, overcome resistance, and reduce costs-paving the way for personalized, safer treatments in refractory MM.
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