Servei de Neurologia-Neuroimmunologia, Centre d'Esclerosi Múltiple de Catalunya (Cemcat), Institut de Recerca Vall d'Hebron (VHIR), Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
Servei de Neurologia-Neuroimmunologia, Centre d'Esclerosi Múltiple de Catalunya (Cemcat), Institut de Recerca Vall d'Hebron (VHIR), Hospital Universitari Vall d'Hebron, Universitat Autònoma de Barcelona, Barcelona, Spain.
J Neuroimmunol. 2020 Dec 15;349:577425. doi: 10.1016/j.jneuroim.2020.577425. Epub 2020 Oct 17.
Multiple Sclerosis (MS) is one of the leading causes of non-traumatic neurological disability among young adults. Due to its complex pathology and the lack of reliable disease models, there are no effective therapies for MS to prevent neurodegeneration or promote neuroprotection, and hence stop disease progression. The emergence of induced pluripotent stem cells (iPSC) has allowed the generation of patient-specific neural cell types for disease modelling, drug screening, and cell therapy. In this review, the challenges related with the use of iPSC-derived cells in MS are discussed, with a special focus on the functional studies performed, limitations and future perspectives.
多发性硬化症(MS)是导致青年成年人非外伤性神经残疾的主要原因之一。由于其复杂的病理学和缺乏可靠的疾病模型,目前尚无有效的治疗方法可预防神经退行性变或促进神经保护,从而阻止疾病进展。诱导多能干细胞(iPSC)的出现使得能够生成用于疾病建模、药物筛选和细胞治疗的患者特异性神经细胞类型。在这篇综述中,讨论了在 MS 中使用 iPSC 衍生细胞所面临的挑战,特别关注已进行的功能研究、局限性和未来展望。