defects

2BAct is a novel eif2b activator, preventing neurological defects caused by a chronic integrated stress response

B I09, an IRE-1 RNase inhibitor with an IC50 of 1230 nM, inhibits splicing of XBP1 mRNA in human WaC3 cells and expression of xbp-1 in LPS-stimulated B cells. B I09 can be used to simulate the defects of XBP-1 in CLL cells., an IRE-1 RNase inhibitor with an IC50 of 1230 nM, inhibits splicing of XBP1 mRNA in human WaC3 cells and expression of xbp-1 in LPS-stimulated B cells. B I09 can be used to simulate the defects of XBP-1 in CLL cells., an IRE-1 RNase inhibitor with an IC50 of 1230 nM, inhibits splicing of XBP1 mRNA in human WaC3 cells and expression of xbp-1 in LPS-stimulated B cells. B I09 can be used to simulate the defects of XBP-1 in CLL cells.

Mopidamol (RA 233) is a phosphodiesterase inhibitor, a dipyridamole derivative, with anticancer activity that prevents retinal vascular defects in experimental diabetes.

VRT-532 (CFpot-532) is an effective modulator of CFTR and is commonly used in studies of cystic fibrosis (CF) caused by CFTR defects. Modification of CFTR by this small molecule modulator can increase arylsulfatase B (ARSB), which is necessary to reduce the accumulation of sulphate glycosaminosaccharide (gag), thus reducing the accumulation of 4-chondroitin sulfate in cystic fibrosis.

VRT-325 can repair folding defects by promoting dimerization of the two NBDs or by promoting folding of the TMD.VRT-325 accelerates the healing rate of CF cell monolayers.

Folic acid is an orally active essential nutrient involved in the synthesis of DNA/RNA and the production and maintenance of new cells. It exhibits antidepressant-like effects and can reduce the risk of neural tube defects in newborns. Folate deficiency can cause megaloblastic anemia, macrocytic anemia, neural tube closure defects, tumors, and aging-related diseases. Folic acid is also commonly used in research on folate deficiency-related conditions and to induce acute kidney injury models.

Cholestenone is an intermediate oxidation product of cholesterol that is metabolized primarily in the liver. It can cause long-term defects in cell function. It is highly mobile in membranes and affects cholesterol turnover and efflux.

Pramiracetam (CI-879), derived from piracetam, is a more effective nootropic drug . According to reports, Pramiracetam improved cognitive defects related with traumatic brain wounds. Pramiracetam was also potent in the therapy of cognitive disorders of traumatic origins and cerebrovascular.

FKB04 is a telomeric repeat binding factor 2 (TRF2) inhibitor that exerts its antitumor activity by disrupting the telomere maintenance mechanisms in hepatocellular carcinoma cells. This leads to T-loop defects, inducing telomere shortening and cellular senescence. Additionally, FKB04 inhibits tumor growth in a human liver cancer xenograft mouse model (by implanting Huh-7 cells in BALB c mice). This compound is utilized in research focused on liver cancer.

YJ1206 is a highly potent and selective CDK12 13 PROTAC degrader (IC50 =12.55 nM) with the advantages of oral administration and low toxicity, which triggers gene-length-dependent transcription elongation defects, leading to DNA damage and cell-cycle arrest, and inhibits the proliferation of a subpopulation of prostate cancer cells. YJ1206 can synergize with AKT pathway inhibitors to effectively inhibit prostate cancer.

YJ9069 is a selective CDK12 CDK13P ROTAC degrader with an IC50 of 22.22 nM in VCaP cells. Rapid degradation of CDK12 13 by this compound triggers transcription elongation defects dependent on gene length, leading to DNA damage and cell cycle arrest. YJ9069 effectively inhibits the proliferation of prostate cancer cell subpopulations and significantly suppresses prostate tumor growth in vivo.

EpoY (SD-142) is an irreversible inhibitor of the primary brain microtubule-associated tyrosine carboxypeptidase (TCP), a complex formed by vasohibin-1 (VASH1) and small vasohibin-binding protein (SVBP). By inhibiting TCP with an IC50 value of approximately 500 nM, EpoY effectively reduces the levels of detyrosinated α-tubulin, which is crucial for microtubule dynamics and neuronal differentiation. This inhibition results in significant differentiation defects and is linked to potential issues related to cancer and cardiomyopathy.

PZ-3022 is an orally active, conformational PanK activator that counteracts C3-CoA inhibition. In a mouse model of propionic acidemia, it elevates hepatic CoASH and C2-CoA levels while reducing C3-CoA, making it useful for studying mitochondrial defects in propionic acidemia.

Etaden exhibits a positive impact on protein synthesis, facilitating the accelerated healing of ulcerative defects.

Valproic acid hydroxamate (VPA-HA) exhibits anticonvulsant activity in a mouse model of neural tube defects and lacks teratogenic activity.

HDAC8-IN-7 (H7E) is an inhibitor of HDAC8 that provides retinal protection against glaucoma damage by improving abnormal Müller glial cell activity and reducing oxidative stress. HDAC8-IN-7 also alleviates both functional and structural defects in the inner layer of the retina.

Chlopynostat (Compound 6c) is an HDAC1 inhibitor with an IC50 value of 67 nM. It induces apoptosis by inhibiting HDAC1, thereby reversing STAT4/p66Shc defects.

RTx-152 is an allosteric Polθ-pol inhibitor (IC50: 6.2 nM) that targets Polθ captured on DNA. It selectively kills cancer cells with homologous recombination (HR) defects and inhibits resistance to PARP inhibitors across various genetic backgrounds, including HR-proficient cells. Additionally, RTx-152 selectively eliminates BRCA2-deficient cells.

Fluconazole (hydrate) is a triazole antifungal agent used for oral treatment and prevention of both superficial and systemic fungal infections, including but not limited to balanitis, various Candida infections, Cryptococcus infections, dermatomycosis, and fungal infections of the respiratory and urinary tracts. As the hydrate salt form of fluconazole, it exhibits antifungal activity by inhibiting the growth and development of pathogenic fungi, such as Candida species, at minimal inhibitory concentrations often exceeding 100 mg/l. Notably, it disrupts the branching and hyphal formation in C. albicans at concentrations as low as 0.3 mg/l, indicating its potency compared to other antifungals like miconazole and ketoconazole which are effective at 100-fold lower concentrations. Clinical studies have not shown a significant increase in the risk of most birth defects with oral fluconazole, although there may be an associated risk of tetralogy of Fallot. Its efficacy against Cryptococcus gattii in koalas suggests it may be ineffective as a sole therapeutic agent at a dosage of 10 mg/kg orally every 12 h. Toxicity symptoms from overdose can include hallucinations and paranoid behavior.

Hexaglutamate folate is a naturally occurring form of folic acid. The bioavailability of dietary folate may be limited by the need for the glutamate moieties to be deconjugated before absorption. Folate deficiency in humans results in anemia, neural tube

Hydroxyellipticine-1a, a selective r(CGG)exp probe, improves pre-mRNA splicing defects, inhibits formation of nuclear inclusions and RANT in a FXTAS cellular mode.l

SB236057 is a potent and selective inverse agonist for the 5-HT receptor, acting especially at 5-HT autoreceptors on nerve terminals. It produces a rapid increase in 5-HT levels in the brain. SB236057 also acts as a potent teratogen, producing severe musc

Hyperphyllin specifically mimics the branch defects of AMP1, including plastid reduction and expansion and proliferation of the branch meristem.

Levomefolate calcium pentahydrate is the calcium salt of 5-methyltetrahydrofolic acid, a biologically active form of folic acid that functions, in conjunction with Vitamin B12. Levomefolate is included in formulations of certain oral contraceptives to ens