neurodegenerative disorder

(Rac)-Neurodegenerative Disorder-Targeting Compound 1 is a calpain inhibitor.

Neurodegenerative Disorder-Targeting Compound 1 is a calpain inhibitor [1].

3β,5α,6β-Trihydroxycholanic acid, a metabolite derived from 5α,6β-dihydroxycholestanol, exhibits elevated levels in dried blood spots of patients diagnosed with Niemann-Pick disease type C, a neurodegenerative disorder characterized by the accumulation of cholesterol and sphingolipids in lysosomes.

BRD3731 is a selective inhibitor of GSK3β, demonstrating an IC50 value of 15 nM for GSK3β and 215 nM for GSK3α. Due to its inhibitory properties, BRD3731 holds promise for investigating various medical conditions, including post-traumatic stress disorder (PTSD), psychiatric disorders, diabetes, and neurodegenerative disorders [1].

β-Amyloid (11-22) is a peptide fragment of β-Amyloid. β-Amyloid peptide (Abeta), the primary component of amyloid plaques in the brains of Alzheimer’s patients, is believed to be the cause of Alzheimer’s Disease (AD), the most common neurodegenerative disease, affecting approximately 10% of the population over 60.

KMS88009 is an amyloid-β aggregation inhibitor that acts by ameliorating neurodegenerative disorder.

C20 Sphingomyelin is a naturally occurring sphingolipid. Levels of C20 sphingomyelin are upregulated in the hippocampus of rats with diabetes induced by streptozotocin and in human plasma, where it is positively correlated with insulin resistance in obese humans. C20 sphingomyelin is also upregulated in the liver of a mouse model of Niemann-Pick type C1 disease [a neurodegenerative cholesterol-sphingolipid lysosomal storage disorder]. The plasma concentration of C20 sphingomyelin is decreased in men with prostate cancer.

β-Amyloid (1-40) TFA, a principal protein in plaques observed in the brains of Alzheimer's disease patients [1], is pivotal in research focused on this neurodegenerative disorder.

Gedunin is an important limonoid present in several genera of the Meliaceae family, mainly in seeds. Gedunin is an Hsp90 inhibitor, and inhibits ovarian cancer cell proliferation.

Mu-6S-Palm-β-Glc is a fluorogenic substrate of palmitoyl-protein thioesterase (PPT, also known as CLN1), a lysosomal hydrolase that removes long-chain fatty acyl groups from modified cysteine residues in proteins. Upon cleavage by PPT CLN1, it releases the fluorescent moiety 4-methylumbelliferyl (4-MU). 4-MU fluorescence is pH-dependent, with excitation maxima of 320 and 360 nm at low (1.97-6.72) and high (7.12-10.3) pH, respectively, and an emission maximum ranging from 445 to 455 nM, increasing as pH decreases. This substrate is used in assays measuring PPT activity, commonly deficient in the neurodegenerative disorder known as infantile neuronal ceroid lipofuscinosis.

mHTT-IN-1 (Example 1) is a potent inhibitor of the mutant huntingtin protein (mHTT), which is primarily responsible for Huntington's disease (HD), an inherited autosomal dominant neurodegenerative disorder. This compound effectively reduces mHTT levels with an EC50 of 46 nM.

C16 Lactosylceramide is an endogenous bioactive sphingolipid. It forms membrane microdomains with Lyn kinase and the αi subunits of inhibitory G protein-coupled receptors (GPCRs), suggesting a role in cell signaling. Plasma levels of C16 lactosylceramide are elevated in insulin-resistant cattle. C16 Lactosylceramide is also upregulated in a mouse model of Niemann-Pick type C1 disease, a neurodegenerative cholesterol-sphingolipid lysosomal storage disorder.