Neural Circuit Laboratories, Friedrich Miescher Institute for Biomedical Research, Basel, Switzerland.
Gene Ther. 2012 Feb;19(2):169-75. doi: 10.1038/gt.2011.155. Epub 2011 Oct 13.
Retinitis pigmentosa (RP) refers to a diverse group of progressive, hereditary diseases of the retina that lead to incurable blindness and affect two million people worldwide. Artificial photoreceptors constructed by gene delivery of light-activated channels or pumps ('optogenetic tools') to surviving cell types in the remaining retinal circuit has been shown to restore photosensitivity in animal models of RP at the level of the retina and cortex as well as behaviorally. The translational potential of this optogenetic approach has been evaluated using in vitro studies involving post-mortem human retinas. Here, we review recent developments in this expanding field and discuss the potential and limitations of optogenetic engineering for the treatment of RP.
色素性视网膜炎(RP)是一组不同的进行性、遗传性视网膜疾病,可导致无法治愈的失明,影响全球 200 万人。通过基因传递光激活通道或泵(“光遗传学工具”)到剩余视网膜回路中存活的细胞类型构建的人工光感受器,已在 RP 的动物模型中显示出在视网膜和皮层水平以及行为上恢复光敏性。这种光遗传学方法的转化潜力已经通过涉及死后人类视网膜的体外研究进行了评估。在这里,我们回顾了这个不断发展的领域的最新进展,并讨论了光遗传学工程治疗 RP 的潜力和局限性。