defects

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

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.

D-I03 is a selective RAD52 inhibitor with a Kd of 25.8 µM. D-I03 inhibits RAD52-dependent single-strand annealing (SSA) and D-loop formation, with IC₅₀ values of 5 µM and 8 µM, respectively. D-I03 exerts significant growth inhibition on BRCA1/2-deficient cancer cells but has no effect on RAD51. D-I03 can be used in combination therapy studies for tumors with DNA damage repair defects.

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 is a CFTR modulator with the ability to promote ΔF508-CFTR folding and endoplasmic reticulum trafficking. VRT-325 restores chloride transport in bronchial epithelial cells from cystic fibrosis patients and accelerates healing of CF cell monolayers, used for experimental research in cystic fibrosis.

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.

AqF026 is an AQP1 agonist that enhances AQP1-mediated water transport. It is utilized in research related to peritoneal dialysis and diseases associated with local water metabolism defects, such as subretinal edema and cerebral edema.

EXO1-IN-1 (Compound F684) is a potent and selective inhibitor of exonuclease 1 (EXO1) with an IC50 of 15.7 μM. It inhibits DNA end resection, promotes the accumulation of DNA double-strand breaks, and induces S-phase polyadenylation, thereby disrupting DNA repair pathways in homologous recombination-deficient (HRD) cancer cells. This selectivity effectively targets and kills tumor cells with homologous recombination gene defects, such as BRCA1/2 mutations. EXO1-IN-1 holds promise for researching cancers with homologous recombination deficiencies, including BRCA-related tumors.

KI-DX-014 is a DDX21 inhibitor with an IC50 of 3.31 μM. It effectively disrupts the interaction between DDX21 and RNA, modulates ATPase activity, and influences the formation of biomolecular condensates. This compound reduces the release of DDX21-dependent P-TEFb from the 7SK snRNP complex, inhibits RNA polymerase II CTD phosphorylation, and induces developmental defects in zebrafish embryos. KI-DX-014 is applicable in research related to cancer and neurodegenerative diseases.

MU262 is a structurally novel polycyclic heterocyclic small-molecule compound and a MUS81 inhibitor with an IC₅₀ value of 0.87 μM, which directly inhibits MUS81 catalytic activity through non-interfering DNA binding, inducing genomic instability in tumor cells and specifically inhibiting the HR/BIR repair pathways. MU262 can serve as a chemical biology tool for studying MUS81 function and as a lead compound for developing anticancer therapies that exploit DNA repair defects in cancer cells.

T-1228 is a highly selective inhibitor of LpxC. It effectively blocks the synthesis of LPS, leading to structural defects in the bacterial outer membrane, increased membrane permeability, and ultimately bacterial cell death. T-1228 is used in research on Gram-negative bacterial infections.