光遗传视觉恢复中天然和假体视觉反应的相互作用。

IF 6.3 1区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL
JCI insight Pub Date : 2025-04-15 eCollection Date: 2025-06-09 DOI:10.1172/jci.insight.190785
Eleonora Carpentiero, Steven Hughes, Jessica Rodgers, Nermina Xhaferri, Sumit Biswas, Michael J Gilhooley, Mark W Hankins, Moritz Lindner
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引用次数: 0

摘要

退行性视网膜疾病导致不可逆的光感受器死亡是失明的常见原因。光遗传基因疗法旨在通过将光敏视蛋白引入视网膜内存活的神经元中来恢复受影响个体的视力。虽然到目前为止,光基因治疗的主要焦点一直是在晚期失明的个体上,但在存在残余天然视力的阶段进行治疗可能有几个优势。然而,如果同时存在,残余的天然视觉和光遗传视觉如何相互作用仍是未知的。利用在on双极细胞中表达光遗传学工具ReaChR的转基因小鼠,我们通过视网膜电图(ERG)和视觉诱发电位(VEP)研究了这种相互作用。我们发现光遗传反应显示出一种特殊的ERG特征,并且在没有光感受器丢失的视网膜中增强。相反,在ReaChR的存在下,自然反应受到抑制。此外,在VEP记录中,我们发现光遗传反应到达皮层与自然反应是异步的。这些发现应该在规划未来的临床试验时加以考虑,并可能指导未来的临床前研究,以优化光遗传学方法来恢复视力。此外,确定的ERG特征可以用于跟踪临床试验中的治疗效率。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Interaction between native and prosthetic visual responses in optogenetic visual restoration.

Degenerative retinal disorders leading to irreversible photoreceptor death are a common cause of blindness. Optogenetic gene therapy aims to restore vision in affected individuals by introducing light-sensitive opsins into the surviving neurons of the inner retina. While up until now, the main focus of optogenetic therapy has been on terminally blind individuals, treating at stages where residual native vision is present could have several advantages. However, it is still unknown how residual native and optogenetic vision would interact if present at the same time. Using transgenic mice expressing the optogenetic tool ReaChR in ON-bipolar cells, we herein examine this interaction through electroretinography (ERG) and visually evoked potentials (VEP). We find that optogenetic responses show a peculiar ERG signature and are enhanced in retinas without photoreceptor loss. Conversely, native responses are dampened in the presence of ReaChR. Moreover, in VEP recordings, we find that optogenetic responses reach the cortex asynchronous to the native response. These findings should be taken into consideration when planning future clinical trials and may direct future preclinical research to optimize optogenetic approaches for visual restoration. The identified ERG signatures, moreover, may serve to track treatment efficiency in clinical trials.

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来源期刊
JCI insight
JCI insight Medicine-General Medicine
CiteScore
13.70
自引率
1.20%
发文量
543
审稿时长
6 weeks
期刊介绍: JCI Insight is a Gold Open Access journal with a 2022 Impact Factor of 8.0. It publishes high-quality studies in various biomedical specialties, such as autoimmunity, gastroenterology, immunology, metabolism, nephrology, neuroscience, oncology, pulmonology, and vascular biology. The journal focuses on clinically relevant basic and translational research that contributes to the understanding of disease biology and treatment. JCI Insight is self-published by the American Society for Clinical Investigation (ASCI), a nonprofit honor organization of physician-scientists founded in 1908, and it helps fulfill the ASCI's mission to advance medical science through the publication of clinically relevant research reports.
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