Goureau Olivier, Orieux Gaël
Institut de la Vision, Sorbonne Université, Inserm, CNRS, 17 rue Moreau, F-75012 Paris, France.
Med Sci (Paris). 2020 Jun-Jul;36(6-7):600-606. doi: 10.1051/medsci/2020097. Epub 2020 Jul 2.
In inherited retinal diseases such retinitis pigmentosa, characterized by progressive loss of light sensitive neurons (photoreceptors), cell therapy is now considered as an attractive strategy. Photoreceptor cell replacement would be valuable for restoring function to retinas in a way that is independent from the cause of the disease. With advances in stem cell biology, considerable strides have been made towards the generation of retinal cells, in particular with the development of 3D culture systems allowing the generation of retinal organoids from pluripotent stem cells. In this review, we present a state-of-the art of preclinical strategies conducted in animal models for photoreceptor replacement from stem cell-derived photoreceptors and we discuss the important obstacles to overcome in the future.
在诸如色素性视网膜炎等遗传性视网膜疾病中,其特征是光敏感神经元(光感受器)逐渐丧失,细胞疗法目前被认为是一种有吸引力的策略。光感受器细胞替代对于以独立于疾病病因的方式恢复视网膜功能将是有价值的。随着干细胞生物学的进展,在生成视网膜细胞方面已经取得了长足的进步,特别是随着3D培养系统的发展,使得从多能干细胞生成视网膜类器官成为可能。在这篇综述中,我们展示了在动物模型中从干细胞衍生的光感受器进行光感受器替代的临床前策略的最新进展,并讨论了未来需要克服的重要障碍。