superoxide dismutase [cu-zn]

AKV-9 是一种突变型 Cu Zn 超氧化物歧化酶 (SOD1) 抑制剂，能够抑制 SOD1 诱导的蛋白质聚集，并在 PC-12 细胞中展现对 SOD1 诱导的细胞毒性的保护作用，EC50 为 0.3 μM。AKV-9 可在小鼠模型中改善肌萎缩侧索硬化症 (ALS)，并延长其寿命。

The metallo-protein Cu/Zn-superoxide dismutase (SOD1) is a ubiquitous enzyme responsible for scavenging superoxide radicals. Mutations in SOD1, which alter its metal binding capacity and can result in protein misfolding and aggregation, have been linked to familial amyotrophic lateral sclerosis (ALS). Cu-ATSM is an orally bioavailable, blood-brain barrier permeable complex that has traditionally been used in cellular imaging experiments to selectively label hypoxic tissue via its susceptibility to reduction by oxygen-depleted mitochondria. More recently, Cu-ATSM has been reported to improve locomotor function and survival in a transgenic ALS mouse model by delivering copper specifically to cells in the spinal cords of mice producing misfolded SOD1 proteins. Copper chaperone for SOD (CCS) is presumed to utilize the copper from Cu-ATSM to prevent misfolding of the SOD1 protein.

SOD1 (147-153) human是一段在Cu Zn超氧化物歧化酶 (SOD1) 中倾向于形成淀粉样蛋白纤维的肽段.此肽段促进聚集体的形成,是导致家族性肌萎缩侧索硬化症 (fALS) 和散发性肌萎缩侧索硬化症 (sALS) 的关键分子.