Department of Genetics, UCL Institute of Ophthalmology, 11-43 Bath Street, London EC1V 9EL, UK.
Biotechnol Adv. 2014 Mar-Apr;32(2):485-91. doi: 10.1016/j.biotechadv.2014.01.001. Epub 2014 Jan 8.
Despite very different aetiologies, age-related macular degeneration (AMD) and most inherited retinal disorders culminate in the same final common pathway, loss of the light-sensitive photoreceptors. There are few clinical treatments and none can reverse the loss of vision. Photoreceptor replacement by transplantation is proposed as a broad treatment strategy applicable to all degenerations. The past decade has seen a number of landmark achievements in this field, which together provide strong justification for continuing investigation into photoreceptor replacement strategies. These include proof of principle for restoring vision by rod-photoreceptor transplantation in mice with congenital stationary night blindness and advances in stem cell biology, which have led to the generation of complete optic structures in vitro from embryonic stem cells. The latter represents enormous potential for generating suitable and renewable donor cells with which to achieve the former. However, there are still challenges presented by the degenerating recipient retinal environment that must be addressed as we move to translating these technologies towards clinical application.
尽管病因非常不同，但年龄相关性黄斑变性 (AMD) 和大多数遗传性视网膜疾病最终都通向同一个最终共同途径，即光敏感感光器的丧失。目前几乎没有临床治疗方法，而且没有任何方法可以逆转视力丧失。通过移植来替代感光器被提出作为一种广泛的治疗策略，适用于所有变性。过去十年中，该领域取得了一些里程碑式的成就，这些成就共同为继续研究感光器替代策略提供了强有力的依据。其中包括通过向先天性静止性夜盲症小鼠的杆状感光器移植来恢复视力的原理证明，以及干细胞生物学的进展，这些进展已从胚胎干细胞体外产生完整的视神经结构。后者为生成合适且可再生的供体细胞提供了巨大的潜力，这些供体细胞可用于实现前者。然而，在将这些技术推向临床应用时，我们仍然面临着由变性受者视网膜环境带来的挑战，这些挑战必须得到解决。
