Treating Duchenne Muscular Dystrophy: The Promise of Stem Cells, Artificial Intelligence, and Multi-Omics.
Treating Duchenne Muscular Dystrophy: The Promise of Stem Cells, Artificial Intelligence, and Multi-Omics.
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DOI：
10.3389/fcvm.2022.851491
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发表时间：
2022
影响因子：
3.6
通讯作者：
Wu JC
中科院分区：
文献类型：
--
作者：
Vera CD;Zhang A;Pang PD;Wu JC
肌肉营养不良是由渐进性肌肉萎缩引起的慢性衰弱疾病。Duchenne肌营养不良症(DMD)是最常见的类型。DMD是一种特征明确的遗传性疾病，由Dstrophin缺失引起。尽管有一些疗法可以治疗这些症状，而且正在努力纠正潜在的分子缺陷，但肌肉营养不良患者将从针对直接导致肌肉疾病的特定途径的新疗法中受益匪浅。三项新进展有望改变DMD等肌营养不良症的治疗格局。首先，人类诱导多能干细胞(IPSCs)的出现使研究人员能够设计有效的治疗策略，弥补传统的“一刀切”策略所遗漏的空白。通过用单细胞分辨率来表征组织改变，并拥有针对各种细胞类型的治疗的分子图谱，临床研究人员可以设计多管齐下的干预措施，不仅可以延缓退化过程，还可以再生健康的组织。其次，人工智能(AI)将在未来的治疗开发中发挥重要作用，它允许聚集和合成大型和不同的数据集，以帮助揭示潜在的分子机制。第三，疾病模型使用了从不同来源收集的大量多组学数据，携带了关于汇聚和分离路径的有价值的信息。使用这些新工具，以前和正在出现的研究结果将催化基于精确医学的药物开发，这些药物可以治疗DMD等破坏性疾病。
Muscular dystrophies are chronic and debilitating disorders caused by progressive muscle wasting. Duchenne muscular dystrophy (DMD) is the most common type. DMD is a well-characterized genetic disorder caused by the absence of dystrophin. Although some therapies exist to treat the symptoms and there are ongoing efforts to correct the underlying molecular defect, patients with muscular dystrophies would greatly benefit from new therapies that target the specific pathways contributing directly to the muscle disorders. Three new advances are poised to change the landscape of therapies for muscular dystrophies such as DMD. First, the advent of human induced pluripotent stem cells (iPSCs) allows researchers to design effective treatment strategies that make up for the gaps missed by conventional “one size fits all” strategies. By characterizing tissue alterations with single-cell resolution and having molecular profiles for therapeutic treatments for a variety of cell types, clinical researchers can design multi-pronged interventions to not just delay degenerative processes, but regenerate healthy tissues. Second, artificial intelligence (AI) will play a significant role in developing future therapies by allowing the aggregation and synthesis of large and disparate datasets to help reveal underlying molecular mechanisms. Third, disease models using a high volume of multi-omics data gathered from diverse sources carry valuable information about converging and diverging pathways. Using these new tools, the results of previous and emerging studies will catalyze precision medicine-based drug development that can tackle devastating disorders such as DMD.
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