Departamento Biologia Celular, Genetica y Fisiologia, Facultad de Ciencias, Instituto de Investigacion Biomedica de Malaga-IBIMA, Universidad de Malaga, 29010 Malaga, Spain.
Centro de Investigacion Biomedica en Red Sobre Enfermedades Neurodegenerativas (CIBERNED), 28031 Madrid, Spain.
Future Med Chem. 2019 Jun;11(11):1305-1322. doi: 10.4155/fmc-2018-0520. Epub 2019 Jun 4.
Most neurodegenerative diseases are characterized by a complex and mostly still unresolved pathology. This fact, together with the lack of reliable disease models, has precluded the development of effective therapies counteracting the disease progression. The advent of human pluripotent stem cells has revolutionized the field allowing the generation of disease-relevant neural cell types that can be used for disease modeling, drug screening and, possibly, cell transplantation purposes. In this Review, we discuss the applications of human pluripotent stem cells, the development of efficient protocols for the derivation of the different neural cells and their applicability for robust disease modeling and drug screening platforms for most common neurodegenerative conditions.
大多数神经退行性疾病的特点是一种复杂且大多仍未解决的病理学。这一事实,再加上缺乏可靠的疾病模型,阻碍了对抗疾病进展的有效疗法的发展。人类多能干细胞的出现彻底改变了这一领域,使得能够产生与疾病相关的神经细胞类型,可用于疾病建模、药物筛选,以及可能的细胞移植目的。在这篇综述中,我们讨论了人类多能干细胞的应用,以及开发高效方案来衍生不同的神经细胞的进展,并讨论了它们在用于大多数常见神经退行性疾病的稳健疾病建模和药物筛选平台中的适用性。