Tragin Margot, Degrelle Séverine A, Periou Baptiste, Bader-Meunier Brigitte, Barnerias Christine, Bodemer Christine, Desguerre Isabelle, Rodero Mathieu Paul, Authier François Jérôme, Gitiaux Cyril
Bioinformatics Facilities Imagine Institute, SFR Necker, 75015 Paris, France.
Inovarion, 75005 Paris, France.
Cells. 2025 Jun 19;14(12):939. doi: 10.3390/cells14120939.
This study aimed to investigate the spatial heterogeneity of molecular signature in the muscle of juvenile dermatomyositis (JDM) patients before and after treatment. Unsupervised reference-free deconvolution of spatial transcriptomics and standardized morphometry were performed in two JDM muscle biopsies with different clinical severity at disease onset and compared to healthy muscle. Identified signatures were scored in two additional JDM muscle biopsies from the same patient before and after remission. Disappearance of the normal muscle signature mostly corresponding to mitochondrial biology was observed in JDM. Three pathological transcriptomic signatures were isolated, related to "myofibrillar stress", "muscle remodeling" and "interferon signaling" signatures. The "myofibrillar stress signature" was prominent in the most severe biopsy while the "muscle remodeling" signature was mostly present in the biopsy from the patient with good outcome. These signatures unveiled genes not previously associated with JDM including ANKRD1 and FSLT1 for "myofibrillar stress" and "muscle remodeling" signatures, respectively. Post-treatment analysis of muscle after two years remission showed a persistence of pathological signatures. This pilot study of JDM muscle identified spatially distributed pathological signatures that persist after remission. This work paves the way for a better understanding of the pathophysiology in affected muscle and the identification of biomarkers that predict relapse.
本研究旨在调查幼年皮肌炎(JDM)患者治疗前后肌肉中分子特征的空间异质性。对疾病发作时具有不同临床严重程度的两份JDM肌肉活检样本进行了空间转录组学的无监督无参考反卷积和标准化形态测量,并与健康肌肉进行比较。在同一患者缓解前后的另外两份JDM肌肉活检样本中对鉴定出的特征进行评分。在JDM中观察到主要与线粒体生物学相对应的正常肌肉特征消失。分离出三种病理转录组特征,分别与“肌原纤维应激”、“肌肉重塑”和“干扰素信号”特征相关。“肌原纤维应激特征”在最严重的活检样本中最为突出,而“肌肉重塑”特征主要存在于预后良好患者的活检样本中。这些特征揭示了以前与JDM无关的基因,“肌原纤维应激”特征对应的是ANKRD1,“肌肉重塑”特征对应的是FSLT1。缓解两年后肌肉的治疗后分析显示病理特征持续存在。这项关于JDM肌肉的初步研究确定了缓解后仍持续存在的空间分布病理特征。这项工作为更好地理解受影响肌肉的病理生理学以及识别预测复发的生物标志物铺平了道路。