Chirivì Maila, Maiullari Fabio, Milan Marika, Ceraolo Maria Grazia, Fratini Nicole, Fasciani Alessandra, Bousselmi Salma, Stirm Michael, Scalera Francesca, Gervaso Francesca, Villa Michela, Viganò Raffaello, Brambilla Francesca, Mauri Pierluigi, De Falco Elena, Silvestre Dario Di, Costantini Marco, Wolf Eckhard, Bearzi Claudia, Rizzi Roberto
Department of Molecular Medicine, Sapienza University, Viale Regina Elena, 324, Rome, 00161, Italy.
Neurology Unit, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Via Francesco Sforza, 35, Milan, 20122, Italy.
Adv Healthc Mater. 2025 Apr;14(9):e2404251. doi: 10.1002/adhm.202404251. Epub 2025 Feb 17.
Advances in understanding the mechanisms behind genetic diseases like Duchenne muscular dystrophy (DMD) underscore the critical role of the extracellular matrix (ECM) composition in disease progression. Effective in vitro models must replicate the intercellular relationships and physicochemical properties of native ECM to fully capture disease-specific characteristics. Although recent biomaterials support the in vitro biofabrication of pathophysiological environments, they often lack disease-specific ECM features. In this study, DystroGel, a hydrogel derived from the cardiac ECM of a porcine DMD model, replicates the distinct molecular composition of dystrophic cardiac tissue for the first time. The findings indicate that the dystrophic ECM matrix exhibits a unique protein profile, impacting cellular processes critical to DMD pathology. This work demonstrates the importance of using a 3D substrate that recreates intercellular dynamics within a defined pathological environment, enhancing the ability to model genetic disorders and providing a valuable tool for advancing personalized therapeutic strategies.
在理解诸如杜兴氏肌肉营养不良症(DMD)等遗传疾病背后的机制方面取得的进展,凸显了细胞外基质(ECM)组成在疾病进展中的关键作用。有效的体外模型必须复制天然ECM的细胞间关系和物理化学性质,以充分捕捉疾病特异性特征。尽管最近的生物材料支持病理生理环境的体外生物制造,但它们往往缺乏疾病特异性的ECM特征。在这项研究中,DystroGel是一种源自猪DMD模型心脏ECM的水凝胶,首次复制了营养不良性心脏组织独特的分子组成。研究结果表明,营养不良性ECM基质呈现出独特的蛋白质谱,影响对DMD病理学至关重要的细胞过程。这项工作证明了使用三维基质在特定病理环境中重现细胞间动态的重要性,增强了对遗传疾病进行建模的能力,并为推进个性化治疗策略提供了有价值的工具。