misfolded

Arimoclomol (BRX-220 free base) 是热休克蛋白 (HSP) 的共诱导剂，可用于治疗肌萎缩侧索硬化症的研究。

Arimoclomol maleate (BRX-220) 是一种热休克蛋白 (HSP) 的共诱导剂。它通过增强 Hsp 表达来保护运动神经元，进而通过蛋白酶体-泛素系统直接影响蛋白质聚集和错误折叠装配体的清除

Lanosterol (3β-Hydroxy-8,24-lanostadiene) 是胆固醇合成的中间产物，能够诱导胆固醇合成的速率控制酶 HMG-CoA 还原酶的泛素化和降解。Lanosterol 可以抑制神经退行性疾病相关错误折叠蛋白的聚集和细胞毒性。

NT219 是一种胰岛素受体底物 1 2 (IRS1 2) 和STAT3的双重抑制剂，可增强错误折叠的朊病毒蛋白的聚集NT219 影响，可提高某些分子伴侣的水平，抑制 STAT3 磷酸化。NT219 具有研究癌症和神经退行性疾病。

PrPSc-IN-1 is a fluorescent probe, binds to the misfolded protein PrPSc, inhibits its accumulation(IC50 of 1.6 μM), with anti-prion activity.

TMU 35435 作为一种HDAC (组蛋白去乙酰化酶) 抑制剂，通过抑制DNA-PKcs（DNA 依赖性蛋白激酶催化亚基）的泛素化来阻断NHEJ通路。此外，该化合物通过促进TNBC中错误折叠蛋白的聚集及自噬作用(autophagy)，增强了对放射线的敏感性。

Simufilamum（亦称PTI-125）是一种针对改变构型的filamin A的小分子口服化合物候选物。它可以结合并逆转阿尔茨海默症大脑中的支架蛋白filamin A的改变构型，从而减缓阿尔茨海默症的病理发展。此外，PTI-125还能降低tau蛋白的超磷酸化、减少Aβ42的聚集沉积、神经纤维缠结和神经炎症。该化合物在体外实验中显示，对照组和阿尔茨海默病（AD）患者死后的海马切片具有浓度依赖性的效力，起始浓度为1 picomolar（pM）。PTI-125是首个优先结合并逆转蛋白病变改变构型的治疗候选物。

PBA-1105 TFA 是一种自噬靶向嵌合体 (AUTOTAC)，诱导p62自我寡聚化。它选择性结合错误折叠蛋白质的暴露疏水区域，通过自噬 (autophagy) 途径促进蛋白质的降解，并提升Ub结合聚集体的自噬通量。

16F16 is a protein disulfide isomerase (PDI) inhibitor.1It inhibits PDI reductase activity in an enzyme assay when used at concentrations ranging from 1 to 100 μg/ml.116F16 reduces PC12 cell apoptosis induced by the misfolded huntingtin protein HTTQ103. It suppresses PDI-dependent mitochondrial outer membrane permeabilization (MOMP) in isolated PC12 cell mitochondria. 16F16 (2, 3, 4, and 10 μM) reduces HTTN90Q73mutant huntingtin-induced medium spinal neuron death and MOMP in rat corticostriatal slices. It also reduces pyramidal neuron death induced by amyloid-β precursor protein (APP) in rat corticostriatal slices. 1.Hoffstrom, B.G., Kaplan, A., Letso, R., et al.Inhibitors of protein disulfide isomerase suppress apoptosis induced by misfolded proteinsNat. Chem. Biol.6(12)900-906(2010)

Endoplasmic reticulum (ER) stress, caused by accumulation of misfolded proteins and a disruption of calcium homeostasis, has been linked to several neuronal diseases including, Parkinson's, Alzheimer's, and prion diseases. A screen for protective activity against ER stress-dependent cell death identified termitomycamides, extracts from the fruiting bodies of T. titanicus, a notably large edible mushroom that is cultivated symbiotically in the nests of termites Termitomycamide B is a fatty acid isolated from T. titanicus that at a dose of 0.1 μg/ml protects against ER stress-dependent cell death in Neuro2a cells induced by tunicamycin.

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.

VCP p97 inhibitor-1, a highly effective compound, inhibits VCP p97 (also known as Cdc48, CDC-48, or Ter94) with an IC 50 of 54.7 nM. This inhibitor induces a disruption in protein homeostasis and interferes with the degradation process of misfolded polypeptides by the ubiquitin-proteasome system (UPS).

Arimoclomol citrate (BRX-220 citrate) is a heat shock protein (HSP) co-inducer that acts by enhancing Hsp expression and directly impacting protein aggregation, clearance of misfolded assemblies, and the proteasome-ubiquitin system. This compound is known for its ability to protect motor neurons.

BAY 1892005 是一种靶向 p53 凝聚体的 p53 蛋白调节剂，可用于研究与 p53 蛋白折叠错误相关的疾病

4-Br-Bnlm 是葡萄糖调节蛋白 (Grp94) 的选择性抑制剂，EC50值为 0.96 µM。它能够减少突变心肌蛋白和野生型心肌蛋白在细胞中的错误折叠水平，并促进毒性心肌素的清除，从而降低心肌毒性。