Department of Molecular Medicine, National Institute of Genetic Engineering and Biotechnology (NIGEB), Tehran, Iran.
Blood Transfusion Research Center, High Institute for Research and Education in Transfusion Medicine, Tehran, Iran.
PLoS One. 2024 Oct 24;19(10):e0311102. doi: 10.1371/journal.pone.0311102. eCollection 2024.
Degenerative eye diseases cause partial or complete blindness due to photoreceptor degeneration. Optogenetic gene therapy is a revolutionary technique combining genetics and optical methods to control the function of neurons. Due to the inherent risk of photochemical damage, the light intensity necessary to activate Opto-mGluR6 surpasses the safe threshold for retinal illumination. Conversely, red-shifted lights pose a significantly lower risk of inducing such damage compared to blue lights. We designed red-shifted Opto-mGluR6 photopigments with a wide, red-shifted working spectrum compared to Opto-mGluR6 and examined their excitation capability in vitro. ROM19, ROM18 and ROM17, red-shifted variants of Opto-mGluR6, were designed by careful bioinformatics/computational studies. The predicted molecules with the best scores were selected, synthesised and cloned into the pAAV-CMV-IRES-EGFP vector. Expression of constructs was confirmed by functional assessment in engineered HEK-GIRK cells. Spectrophotometry and patch clamp experiments demonstrated that the candidate molecules were sensitive to the desired wavelengths of the light and directly coupled light stimuli to G-protein signalling. Herein, we introduce ROM17, ROM18 and ROM19 as newly generated, red-shifted variants with maximum excitation red-shifted of ~ 40nm, 70 nm and 126 nm compared to Opto-mGluR6.
退行性眼病会导致光感受器变性而导致部分或完全失明。光遗传学基因治疗是一种将遗传学和光学方法结合起来控制神经元功能的革命性技术。由于光化学损伤的固有风险,激活 Opto-mGluR6 所需的光强超过视网膜照明的安全阈值。相比之下,与蓝光相比,红移光引发这种损伤的风险要低得多。我们设计了与 Opto-mGluR6 相比具有更宽、红移工作光谱的红移 Opto-mGluR6 光感色素,并在体外研究了它们的激发能力。通过仔细的生物信息学/计算研究设计了 ROM19、ROM18 和 ROM17 这三种 Opto-mGluR6 的红移变体。选择预测得分最高的分子进行合成和克隆到 pAAV-CMV-IRES-EGFP 载体中。通过在工程化的 HEK-GIRK 细胞中的功能评估来确认构建体的表达。分光光度法和膜片钳实验表明,候选分子对所需波长的光敏感,并将光刺激直接与 G 蛋白信号偶联。在此,我们引入了 ROM17、ROM18 和 ROM19,它们是新生成的红移变体,与 Opto-mGluR6 相比,最大激发红移分别约为 40nm、70nm 和 126nm。