Department of Ophthalmology, University of Lausanne, Jules-Gonin Eye Hospital, FAA, Unit of Gene Therapy & Stem Cell Biology, Avenue de France 15, 1004 Lausanne, Switzerland.
École Polytechnique Fédérale de Lausanne, Institut Suisse de Recherche Expérimentale sur le Cancer, 1015 Lausanne, Switzerland.
Stem Cell Reports. 2014 May 22;2(6):853-65. doi: 10.1016/j.stemcr.2014.04.010. eCollection 2014 Jun 3.
Retinal degenerative diseases resulting in the loss of photoreceptors are one of the major causes of blindness. Photoreceptor replacement therapy is a promising treatment because the transplantation of retina-derived photoreceptors can be applied now to different murine retinopathies to restore visual function. To have an unlimited source of photoreceptors, we derived a transgenic embryonic stem cell (ESC) line in which the Crx-GFP transgene is expressed in photoreceptors and assessed the capacity of a 3D culture protocol to produce integration-competent photoreceptors. This culture system allows the production of a large number of photoreceptors recapitulating the in vivo development. After transplantation, integrated cells showed the typical morphology of mature rods bearing external segments and ribbon synapses. We conclude that a 3D protocol coupled with ESCs provides a safe and renewable source of photoreceptors displaying a development and transplantation competence comparable to photoreceptors from age-matched retinas.
导致光感受器丧失的视网膜退行性疾病是失明的主要原因之一。光感受器替代疗法是一种很有前途的治疗方法，因为现在可以将视网膜来源的光感受器移植到不同的鼠类视网膜病变中，以恢复视觉功能。为了获得无限的光感受器来源，我们构建了一个转 Crx-GFP 基因的胚胎干细胞（ESC）系，其中 GFP 基因在光感受器中表达，并评估了 3D 培养方案产生整合能力光感受器的能力。该培养系统允许产生大量的光感受器，再现体内发育。移植后，整合细胞表现出成熟的杆状细胞的典型形态，具有外部节段和带状突触。我们得出结论，3D 方案与 ESC 相结合为光感受器提供了一种安全且可再生的来源，这些光感受器表现出与年龄匹配的视网膜光感受器相当的发育和移植能力。
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