通过 AAV9 疗法调节 Tau 可增强青光眼中 Akt/Erk 的存活信号,减轻视网膜变性表型。

IF 6.2 2区 医学 Q1 NEUROSCIENCES
Kanishka Pushpitha Maha Thananthirige, Nitin Chitranshi, Devaraj Basavarajappa, Rashi Rajput, Mojdeh Abbasi, Viswanthram Palanivel, Veer Bala Gupta, Joao A Paulo, Maya Koronyo-Hamaoui, Mehdi Mirzaei, Stuart L Graham, Vivek Gupta
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引用次数: 0

摘要

微管相关蛋白 Tau 是各种神经退行性疾病(包括阿尔茨海默病(AD)和 Tauopathies)的关键因素,其过度磷酸化会破坏神经元微管晶格的稳定性。青光眼是一种影响视网膜的神经退行性疾病,通过损害视网膜神经节细胞和视神经导致不可逆的视力丧失,通常与眼压升高有关。先前的研究表明,AD 和青光眼视网膜中的 Tau 表达和磷酸化都发生了改变,但 Tau 蛋白变化在这些病症中的致病性或下游性质仍不清楚。本研究探讨了正常和实验性青光眼条件下 Tau 蛋白调节对视网膜神经元的影响。利用 AAV9 介导的基因疗法来实现 Tau 的过表达和敲除,结果发现这两种操作都会对视网膜的结构和功能措施以及健康条件下的神经保护性 Akt/Erk 生存信号产生不利影响。在实验性青光眼模型中,Tau过表达会加剧视网膜内层的退化,而Tau沉默则能显著保护视网膜免受这些退化性变化的影响。这些发现强调了内源性 Tau 蛋白水平在维护视网膜完整性方面的关键作用,并强调了在青光眼病理中靶向 Tau 的治疗潜力。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Tau modulation through AAV9 therapy augments Akt/Erk survival signalling in glaucoma mitigating the retinal degenerative phenotype.

The microtubule-associated protein Tau is a key player in various neurodegenerative conditions, including Alzheimer's disease (AD) and Tauopathies, where its hyperphosphorylation disrupts neuronal microtubular lattice stability. Glaucoma, a neurodegenerative disorder affecting the retina, leads to irreversible vision loss by damaging retinal ganglion cells and the optic nerve, often associated with increased intraocular pressure. Prior studies have indicated Tau expression and phosphorylation alterations in the retina in both AD and glaucoma, yet the causative or downstream nature of Tau protein changes in these pathologies remains unclear. This study investigates the impact of Tau protein modulation on retinal neurons under normal and experimental glaucoma conditions. Employing AAV9-mediated gene therapy for Tau overexpression and knockdown, both manipulations were found to adversely affect retinal structural and functional measures as well as neuroprotective Akt/Erk survival signalling in healthy conditions. In the experimental glaucoma model, Tau overexpression intensified inner retinal degeneration, while Tau silencing provided significant protection against these degenerative changes. These findings underscore the critical role of endogenous Tau protein levels in preserving retinal integrity and emphasize the therapeutic potential of targeting Tau in glaucoma pathology.

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来源期刊
Acta Neuropathologica Communications
Acta Neuropathologica Communications Medicine-Pathology and Forensic Medicine
CiteScore
11.20
自引率
2.80%
发文量
162
审稿时长
8 weeks
期刊介绍: "Acta Neuropathologica Communications (ANC)" is a peer-reviewed journal that specializes in the rapid publication of research articles focused on the mechanisms underlying neurological diseases. The journal emphasizes the use of molecular, cellular, and morphological techniques applied to experimental or human tissues to investigate the pathogenesis of neurological disorders. ANC is committed to a fast-track publication process, aiming to publish accepted manuscripts within two months of submission. This expedited timeline is designed to ensure that the latest findings in neuroscience and pathology are disseminated quickly to the scientific community, fostering rapid advancements in the field of neurology and neuroscience. The journal's focus on cutting-edge research and its swift publication schedule make it a valuable resource for researchers, clinicians, and other professionals interested in the study and treatment of neurological conditions.
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