genetic

5-Fluoroorotic acid is an inhibitor of thymidylate synthase. 5-Fluoroorotic is a selective agent in yeast molecular genetics.5-Fluoroorotic possesses a well-expressed anticandidal effect close to that of 5-fluorocytosine, as well as moderate antidermatophytal effects.

Epigenetic Multiple Ligand is a cell-permeable inhibitor of substrate processing by several chromatin-associated enzymes, including SIRT1 2, H3 SET7, H3 p300 CBP, H4 RmtA, PABP1 CARM1, and H4 PRMT1. It acts by inducing either apoptosis or granulocytic differentiation.

Epigenetic factor-IN-1 (40569Z) is an inhibitor that exhibits potent binding affinity for SIRT7 and is utilized in the study of liver cancer [1].

CFTR corrector 8 is a highly effective modulator of the cystic fibrosis transmembrane conductance regulator (CFTR). This compound is specifically designed for utilization in research related to cystic fibrosis, a genetic disorder primarily affecting the lungs and digestive system [1].

Moxnidazole, a 5-nitroimidazole, is mutagenic in microbial assays and in Drosophila, inducing genetic alterations in mouse somatic cells.

PG-11047 2HCl is an apoptosis-stimulating agent used in the treatment of genitourinary disorders, immune system disorders, genetic disorders and malformations, ocular disorders, and disorders of the blood and lymphatic systems, and in the study of lymphomas and prostate cancer.

L-Pipecolic acid (L-Homoproline) is a breakdown product of lysine, accumulates in body fluids of infants with generalized genetic peroxisomal disorders, such as Zellweger syndrome, neonatal adrenoleukodystrophy.

N-Acetyl-L-phenylalanine (N-Ac-Phenylalanine) is an essential amino acid produced for medical, feed, and nutritional applications. It appears in large amount in urine of patients with phenylketonuria which is a human genetic disorder due to the lack of phenylalanine hydroxylase, the enzyme necessary to metabolize phenylalanine to tyrosine. Acetylphenylalanine is a product of enzyme phenylalanine N-acetyltransferase in the pathway phenylalanine metabolism.

Homocystine is the double-bonded form of homocysteine, but it occurs only transiently before being converted to the harmless cystathionine via a vitamin B6-dependent enzyme. Increasing evidence supports a role for an elevation of homocysteine in schizophr

N-Acetyl-L-glutamic acid (Ac-L-Glu-OH) (NAcGlu) is an acetylated amino acid. NAcGlu is biosynthesized from glutamic acid and acetyl-CoA by the enzyme N-acetyl glutamate synthase (NAGS). NAcGlu activates carbamoyl phosphate synthetase in the urea cycle. A deficiency in N-acetyl glutamate synthase or a genetic mutation in the gene coding for the enzyme will lead to urea cycle failure in which ammonia is not converted to urea, but rather accumulated in the blood leading to the condition called Type I hyperammonemia.

D-Glutamine is the D-type stereoisomer of cell-permeable glutamine, one of the 20 amino acids encoded by the standardized genetic code.

N-Phenylthiourea (Phenylthiocarbamide) is a phenyl ring-containing organothiourea that acts as an EC 1.14.18.1 (tyrosinase) inhibitor, a diphenolase inhibitor, and a non-competitive inhibitor of the PvdP tyrosinase of Pseudomonas, which inhibits melanogenesis, and is able to be used in genetic taste testing.

DB1255 2TFA is an ERG DNA binding inhibitor with an unusual and potent monomer binding pattern at the minor groove site for the study of genetic disorders.

Sortin1, a chemical genetic-hit compound, specifically induces mislocalization of both soluble and membrane-associated vacuolar markers in plants and yeast.This compound is unstable in powder form and other related salt forms are recommended.

L-Alloisoleucine (L-allo-Isoleucine) is a branched chain amino acid and is a stereoisomer of L-isoleucine. It is a common constituent of human plasma (albeit at low levels). L-alloisoleucine is produced as a byproduct of isoleucine transamination. Plasma L-alloisoleucine, which is derived from L-isoleucine in vivo, can be used for the diagnosis of maple syrup urine disease (MSUD), a genetic disorder. Indeed, plasma L-alloisoleucine levels above 5 μmol L is the most specific and most sensitive diagnostic marker for all forms of MSUD.

BGB-102 (JNJ-26483327) is a kinase inhibitor targeting FLT3 and YES1 and an antagonist targeting EGFR and VEGFR3, which may be useful in the study of macular degeneration and diseases associated with genetic disorders and malformations.

PF-5006739 is an effective and selective inhibitor of CK1δ ε (IC50s: 3.9 nM and 17.0 nM, respectively). PF-5006739 is a potential therapeutic agent for a range of psychiatric disorders with low nanomolar in vitro potency for CK1δ ε and high kinome selecti

cIAP1 Ligand-Linker Conjugates 3 are chemical compounds comprising an IAP ligand designed specifically for the E3 ubiquitin ligase and a PROTAC linker. These conjugates, denoted as cIAP1 Ligand-Linker Conjugates 3, find utility in the design of SNIPERs, short for Specific and Non-genetic IAP-dependent Protein Erasers [1].

PROTAC ERα Degrader-2 is composed of a cIAP1 ligand binding group, a linker, and an estrogen receptor α (ERα) binding group, serving as an ERα degrader. It achieves maximal ERα degradation in human mammary tumor MCF7 cells at a concentration of 30 μM. Degradation inducers that utilize cIAP1 are referred to as specific and non-genetic IAP-dependent protein erasers (SNIPERs)[1].

PROTAC RAR Degrader-1, an RAR degrader, consists of a cIAP1 ligand binding group, a linker, and an RAR ligand binding group. It achieves maximal RAR degradation at a concentration of 30 μM in HT1080 cells. Degradation inducers that utilize cIAP1 are known as specific and non-genetic IAP-dependent protein erasers (SNIPERs)[1].

ELX-02 disulfate (also referred to as NB-124 disulfate) is a synthetic eukaryotic ribosome selective glycoside (ERSG). This compound is currently in development as a therapy specifically designed to address genetic diseases resulting from nonsense mutations.

ELX-02 sulfate, also known as NB-124 sulfate, is a novel compound currently under development as a therapeutic agent for genetic diseases triggered by nonsense mutations. This investigational compound, an advanced synthetic eukaryotic ribosome selective glycoside (ERSG), shows promising potential for targeted treatment.

POSH-IN-2 (MIDI) is a mitochondrial fission inhibitor and a DRP1 inhibitor that effectively prevents mitochondrial fragmentation caused by cellular stress and genetic mitochondrial damage. It covalently interacts with DRP1-C367 to target the interaction of DRP1 with multiple receptors.

5-Fluoroorotic acid (monohydrate) (5-Fluorouracil-6-carboxylic acid Mono(hydrate); 5-FOA) is employed to detect the expression of the URA3 gene in yeast molecular genetic constructs. Yeast possessing an active URA3 gene (which encodes orotidine-5'-monophosphate decarboxylase) convert 5-FOA into 5-fluorodeoxyuridine, a substance toxic to cells. Yeast strains with mutations in the URA3 gene that confer resistance can grow in the presence of 5-FOA if uracil is added to the medium.