Pai Padmini, Nirmal Arnav, Mathias Lian, Jain Siya, Shetty Manasa Gangadhar, Sundara Babitha Kampa
Department of Biophysics, Manipal School of Life Sciences, Manipal Academy of Higher Education, Manipal, Karnataka, 576104, India.
Manipal School of Life Sciences, Manipal Academy of Higher Education, Manipal, Karnataka, 576104, India.
Mol Biotechnol. 2025 Feb;67(2):438-455. doi: 10.1007/s12033-024-01072-2. Epub 2024 Feb 20.
Histiocytosis represents a group of uncommon disorders characterized by the abnormal accumulation of specialized immune cells, such as macrophages, dendritic cells, or monocyte-derived cells, in various tissues and organs. Over 100 distinct subtypes have been documented, each displaying a broad spectrum of clinical presentations and histological characteristics. Till today, histiocytosis has been addressed through a combination of chemotherapy, radiotherapy, and surgery, with varying responses from individual patients. Due to its atypical symptoms, it has been prone to misdiagnosis. Advances in our understanding of the cellular and molecular aspects of these conditions are paving the way for improved diagnostic methods and targeted therapies. Researchers have extensively investigated various mutations in patient samples. However, no paper has yet provided a comprehensive summary of the collective analysis of mutations and pathways. Hence, this paper consolidates research efforts that specifically concentrate on gene mutations identified in patient samples of different subtypes of histiocytosis. These insights are essential for developing targeted therapies and improving diagnosis. Further, it provides potential insights to enhance the development of more effective therapeutic approaches for rare diseases.
组织细胞增多症是一组罕见的疾病,其特征是巨噬细胞、树突状细胞或单核细胞衍生细胞等特殊免疫细胞在各种组织和器官中异常积聚。已记录了100多种不同的亚型,每种亚型都表现出广泛的临床表现和组织学特征。直到今天,组织细胞增多症一直通过化疗、放疗和手术相结合的方式进行治疗,不同患者的反应各不相同。由于其非典型症状,它容易被误诊。我们对这些疾病的细胞和分子方面认识的进展为改进诊断方法和靶向治疗铺平了道路。研究人员对患者样本中的各种突变进行了广泛研究。然而,尚未有论文对突变和信号通路的综合分析进行全面总结。因此,本文整合了专门针对组织细胞增多症不同亚型患者样本中鉴定出的基因突变的研究成果。这些见解对于开发靶向治疗和改善诊断至关重要。此外,它为加强罕见病更有效治疗方法的开发提供了潜在的见解。