Department of Neurology, University Medical Center Goettingen, 37075 Goettingen, Germany.
Institute of Pharmacology and Toxicology, University Medical Center Goettingen, 37075 Goettingen, Germany.
Cells. 2022 Apr 5;11(7):1233. doi: 10.3390/cells11071233.
Many neuromuscular disease entities possess a significant disease burden and therapeutic options remain limited. Innovative human preclinical models may help to uncover relevant disease mechanisms and enhance the translation of therapeutic findings to strengthen neuromuscular disease precision medicine. By concentrating on idiopathic inflammatory muscle disorders, we summarize the recent evolution of the novel in vitro models to study disease mechanisms and therapeutic strategies. A particular focus is laid on the integration and simulation of multicellular interactions of muscle tissue in disease phenotypes in vitro. Finally, the requirements of a neuromuscular disease drug development workflow are discussed with a particular emphasis on cell sources, co-culture systems (including organoids), functionality, and throughput.
许多神经肌肉疾病实体具有显著的疾病负担，治疗选择仍然有限。创新的人类临床前模型可能有助于揭示相关的疾病机制，并加强治疗发现的转化，以加强神经肌肉疾病的精准医学。通过集中研究特发性炎症性肌肉疾病，我们总结了新型体外模型研究疾病机制和治疗策略的最新进展。特别关注的是体外疾病表型中肌肉组织的多细胞相互作用的整合和模拟。最后，讨论了神经肌肉疾病药物开发工作流程的要求，特别强调了细胞来源、共培养系统（包括类器官）、功能和通量。
