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儿童血液病中的细胞治疗

英文原题:Cell therapy in pediatric blood diseases.

查看英文原题

Cell therapy in pediatric blood diseases.

PubMed 2025/06/25(内容时间) Front Med (Lausanne) Q1 · IF 3.6(JCR 2025)

研究概要

与儿童血液肿瘤疾病死亡率的斗争是一部关于人权、学术、创新和技术进步的故事——其中科学界的女性发挥了关键作用。

中文摘要

与儿童血液肿瘤疾病死亡率作斗争的历史,是一部关于人权、学术、创新和技术进步的故事——其中女性科学工作者发挥了关键作用。她们以才华、毅力和敏锐,为新型细胞疗法研究做出了巨大贡献,拓展了治疗的前沿,为最脆弱的患者赢得了更多年的生命。本综述探讨多能细胞和单能细胞疗法,重点关注造血干细胞移植(HSCT)、间充质基质细胞移植(MSCT)以及嵌合抗原受体(CAR)-T细胞和自然杀伤(NK)细胞疗法。HSCT仍然是高危和复发患者的金标准,移植物来源包括骨髓(BM)、动员外周血(MPB)和脐带血(UCB)。MSCT的进展凸显了其在造血支持和免疫调节中的作用,降低了移植物抗宿主病(GVHD)风险。CAR-T细胞疗法已经彻底改变了白血病的治疗,尽管抗原逃逸、T细胞耗竭和治疗耐药等挑战仍然存在。包括CAR-NK细胞在内的新兴策略旨在提高疗效的同时尽量减少毒性。尽管取得了这些进展,细胞疗法仍然复杂且资源密集,需要进一步的创新以实现更广泛的普及,特别是在发展中地区。

展开英文摘要原文

The fight against mortality in pediatric oncohematological diseases is a story of human rights, academia, innovation, and technological advancement-one in which women in science have played a pivotal role. With talent, perseverance, and sensitivity, they greatly contributed to research in new cellular therapies, expanding the frontiers of treatment and giving the most vulnerable patients the gift of more years of life. This review explores multipotent and unipotent cell therapies, focusing on hematopoietic stem cell transplantation (HSCT), mesenchymal stromal cell transplantation (MSCT), and chimeric antigen receptor (CAR)-T cell and natural killer (NK) cell therapy. HSCT remains the gold standard for high-risk and relapsed cases, with graft sources including bone marrow (BM), mobilized peripheral blood (MPB), and umbilical cord blood (UCB). Advances in MSCT highlight its role in hematopoiesis support and immunomodulation, reducing graft-versus-host disease (GVHD) risks. CAR-T cell therapy has revolutionized leukemia treatment, although challenges such as antigen escape, T-cell exhaustion, and treatment resistance persist. Emerging strategies, including CAR-NK cells, seek to enhance efficacy while minimizing toxicity. Despite these advancements, cell therapy remains complex and resource-intensive, necessitating further innovations for broader accessibility, particularly in developing regions.

论文信息

作者
Romo-Rodríguez R、Pelayo R、Ramírez-Ramírez D
单位
Laboratory of Oncoimmunology and Cytomics of Childhood Cancer, Centro de Investigación Biomédica de Oriente, Instituto Mexicano del Seguro Social, Puebla, Mexico.Mexico
文献类型
综述
期刊
Frontiers in medicine2025
原文标识
PubMed 40636378 · DOI 10.3389/fmed.2025.1591287