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Genome editing: the breakthrough technology for inherited retinal disease?

机译：基因组编辑：遗传性视网膜疾病的突破性技术？

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Introduction: Genetic alterations resulting in a dysfunctional retinal pigment epithelium and/or degenerating photoreceptors cause impaired vision. These juxtaposed cells in the retina of the posterior eye are crucial for the visual cycle or phototransduction. Deficits in these biochemical processes perturb neural processing of images capturing the external environment. Notably, there is a distinct lack of clinically approved pharmacological, cell- or gene-based therapies for inherited retinal disease. Gene editing technologies are rapidly advancing as a realistic therapeutic option.Areas covered: Recent discovery of endonuclease-mediated gene editing technologies has culminated in a surge of investigations into their therapeutic potential. In this review, the authors discuss gene editing technologies and their applicability in treating inherited retinal diseases, the limitations of the technology and the research obstacles to overcome before editing a patient's genome becomes a viable treatment option.Expert opinion: The ability to strategically edit a patient's genome constitutes a treatment revolution. However, concerns remain over the safety and efficacy of either transplanting iPSC-derived retinal cells following ex vivo gene editing, or with direct gene editing in vivo. Ultimately, further refinements to improve efficacy and safety profiles are paramount for gene editing to emerge as a widely available treatment option.
机译：简介：导致功能障碍视网膜颜料上皮和/或退化感光感导致的遗传改变导致视力受损。后眼视网膜中的这些并置细胞对于视觉循环或光晶体来说是至关重要的。这些生物化学过程中的缺陷捕获外部环境的图像的神经处理。值得注意的是，临床批准的临床缺乏缺乏缺乏遗传性视网膜疾病的药理学，细胞或基因疗法。基因编辑技术正在迅速推进，作为一种现实的治疗选择。覆盖：最近的内核酸酶介导的基因编辑技术的发现终止于调查的治疗潜力。在本文中，作者讨论了基因编辑技术及其在治疗遗传性视网膜疾病方面的适用性，在编辑患者的基因组之前，技术的局限性和克服的研究障碍成为可行的治疗选项.Pert意见：战略性地编辑的能力患者的基因组构成治疗革命。然而，担心仍然是移植IPSC衍生的视网膜细胞的安全性和功效，或在体内进行直接基因编辑。最终，进一步的改进以改善效力和安全谱是基因编辑作为广泛可用的治疗选择的基因。

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