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2026年6月9日,美国波士顿生物技术公司Life Biosciences宣布,其候选疗法ER-100已完成Ⅰ期临床试验的首例受试者给药。该试验并非以延缓全身衰老为目标,而是用于治疗开角型青光眼和非动脉炎性前部缺血性视神经病变(NAION),尝试修复受损的视网膜神经节细胞。

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(图片来源:《Nature》)

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    2026年6月9日,美国波士顿生物技术公司Life Biosciences宣布,其候选疗法ER-100已完成Ⅰ期临床试验的首例受试者给药。该试验并非以延缓全身衰老为目标,而是用于治疗开角型青光眼和非动脉炎性前部缺血性视神经病变(NAION),尝试修复受损的视网膜神经节细胞。

    . 让受损的视神经细胞重获年轻. 科技导报, 2026 , 44 (12) : 3 -3 .
    . Science & Technology Review, 2026 , 44 (12) : 3 -3 .
    2026年6月9日,美国波士顿生物技术公司Life Biosciences宣布,其候选疗法ER-100已完成Ⅰ期临床试验的首例受试者给药。该试验并非以延缓全身衰老为目标,而是用于治疗开角型青光眼和非动脉炎性前部缺血性视神经病变(NAION),尝试修复受损的视网膜神经节细胞。
    视网膜神经节细胞负责将视觉信号传递至大脑。在青光眼和NAION患者中,这类细胞会逐渐受损甚至死亡。现有治疗主要通过降低眼压、改善血供等方式控制风险,对已经受损的神经细胞缺少有效的修复手段。
    ER-100采用腺相关病毒载体,将OCT4SOX2KLF4 3种转录因子递送至细胞,并通过多西环素诱导系统控制其表达。研发人员希望借此调整细胞的表观遗传状态,提高神经细胞的存活和再生能力。
    该方法源自Sinclair团队2020年发表于《Nature》的研究。研究人员在小鼠视网膜神经节细胞中表达转录因子,观察到DNA甲基化模式和转录组向年轻状态恢复。视神经受损后的轴突再生能力有所增强,青光眼模型小鼠和老年小鼠的视觉功能也得到了改善。
    与诱导多能干细胞技术不同,ER-100并不将成熟细胞完全重置为类似胚胎干细胞的状态,而是采用“部分重编程”。其目标是在保留细胞原有身份的同时,恢复部分受衰老影响的功能。
    首都医科大学附属北京同仁医院眼底遗传性疾病诊疗中心、北京市眼科研究所金子兵和王晓薇长期从事遗传性视网膜疾病诊治及干细胞、基因治疗研究。金子兵和王晓薇在接受《科技导报》采访时表示,ER-100直接针对视网膜神经节细胞的退行性损伤,与主要控制眼压或血供等危险因素的现有疗法有所不同。
    他们认为,这种方法不针对某一种致病基因,而是通过调整细胞的表观遗传状态,提高神经元的生存和修复能力。因此,它有可能用于病因不同、但都伴有神经退行性损伤的眼病。
    然而,他们也表示该策略存在明确的适用边界与风险:以ABCA4基因突变导致的Stargardt病为例,其病因是特定基因缺陷,仅靠部分重编程很难解决问题,仍需采用基因增补或基因编辑等针对性治疗。在某些突变背景下,启动重编程还可能产生不利影响。
    ER-100也无法让已经死亡的细胞重新恢复。金子兵和王晓薇指出,这种疗法起效的前提是靶细胞仍然存活。对于衰老或受损但尚未死亡的神经元,它或许能够改善细胞状态;当疾病进展到大量细胞不可逆死亡时,则可能需要考虑细胞替代治疗。
    目前,ER-100仍处于Ⅰ期试验阶段,研究重点是评估单次给药的安全性和耐受性,并观察视觉功能变化。成体细胞表达重编程因子是否会增加肿瘤风险、引发免疫反应,以及作用能够维持多久,都要等待临床数据。
    (综合:《Nature》《Cell》、Life Biosciences公司)

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    2种不同金属材料的力学参数

    Family
    属数
    Number of
    genus
    种数
    Number of
    species
    占总种数比例
    Percentage of
    total species (%)

    Genus
    种数
    Number of
    species
    占总种数比例
    Percentage of total
    species (%)
    鹅膏菌科Amanitaceae 2 11 5.26 鹅膏菌属 Amanita 10 4.78
    小菇科 Mycenaceae 2 12 5.74 丝盖伞属 Inocybe 5 2.39
    多孔菌科 Polyporaceae 8 14 6.70 蜡蘑属 Laccaria 5 2.39
    红菇科 Russulaceae 3 23 11.00 小皮伞属 Marasmius 6 2.87
    小菇属 Mycena 11 5.26
    光柄菇属 Pluteus 5 2.39
    红菇属 Russula 17 8.13
    栓菌属 Trametes 5 2.39
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