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Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!2025-02-06
20 Results for "
genetic disorders
" in MedChemExpress (MCE) Product Catalog:
Products (20)
20 Results for "genetic disorders" in MCE Product Catalog:
3
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Research Areas:
Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC50 of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .
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1 Cited Publications
Cat. No.: HY-101203
CAS No.: 1459687-89-8
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GJ103 is a read-through compound that can induce read through of premature stop codons. GJ103 has potential for the research of genetic disorders caused by nonsense mutations .
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1 Cited Publications
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GJ103 sodium is a read-through compound that can induce read through of premature stop codons. GJ103 sodium has potential for the research of genetic disorders caused by nonsense mutations .
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PF-5006739 is a potent and selective inhibitor of CK1δ/ε with IC50s of 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 selectivity. PF-5006739 attenuats opioid agent-seeking behavior in a rodent operant reinstatement model in animals in a dose-dependent manner . PF-5006739 improves glucose tolerance in both diet-induced obesity (DIO) and genetic (ob/ob) mice models of obesity .
Cat. No.: HY-W019815
CAS No.: 759-73-9
Synonyms: ENU; N-Nitroso-N-ethylurea
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N-Ethyl-N-nitrosourea (ENU) is a DNA alkylating agent. N-Ethyl-N-nitrosourea induces leukemia by alkylating nucleobases, disrupting DNA, and resulting in bone marrow suppression and the formation of leukemic cells. N-Ethyl-N-nitrosourea is teratogenic in vivo, inducing tumor formation and paw malformations in pregnant rats. N-Ethyl-N-nitrosourea cause central nervous system (CNS) tumors and genetic disorders .
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Cat. No.: HY-B0607R
CAS No.: 104206-65-7
Synonyms: NTBC (Standard); Nitisone (Standard); SC0735 (Standard)
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Nitisinone (Standard) is the analytical standard of Nitisinone. This product is intended for research and analytical applications. Nitisinone is an orally active, competitive and reversible 4-hydroxyphenylpyruvate dioxygenase (4-HPPD) inhibitor with an IC50 of 173 nM. Nitisinone promotes tyrosine accumulation in a dose-dependent manner. nitisinone can be used in studies of hereditary tyrosinemia type 1 (HT-1) (a rare genetic disorder) and albinism .
Cat. No.: HY-177058
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DSP30 is a phosphorothioate cpG-oligodeoxynucleotide and a TLR9 agonist. DSP30 can activate immune system cells, including B cells and dendritic cells, by inducing proliferation and cytokine production.DSP30 can enhance the immunosuppressive function of bone marrow-multipotent mesenchymal stromal cells (BM-MSC). DSP30 combined with interleukin 2 (IL2) is an effective mitotic stimulant in B-cell disorders. DSP30 can be used for the genetic characteristic research and analysis of chronic lymphocytic leukemia (CLL) .
Cat. No.: HY-164977
CAS No.: 3093519-24-2
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JCS-1 is a potent DcpS PROTAC degrader. JCS-1 non-covalently binds DcpS with a RG3039-based warhead and recruits the E3
ligase VHL. JCS-1 promotes ubiquitination and degradation of DcpS at nanomolar concentrations (DC50 in MOLM-14 cells: 87 nM). JCS-1 can be used for the research of AML and other DcpS-dependent genetic disorders .
Cat. No.: HY-123359
CAS No.: 414909-09-4
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RTC14 is a read-through compound (RTC) that can induce ribosomes to bypass nonsense mutations in mRNA and allow the production of full-length functional proteins. RTC14 has the potential to be used in the research of various genetic disorders, such as nonsense mutations in the ataxia-telangiectasia mutated (ATM) gene and the dystrophin gene .
Cat. No.: HY-171981
CAS No.: 1934241-32-3
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VU6002703 (Compound 17) is a BBB-penetrable M4 mAChR positive allosteric modulator (PAM) with an EC50 of 0.6 μM for hM4. VU6002703 can be used for neuropsychiatric and rare genetic CNS disorders research .
Cat. No.: HY-153057
CAS No.: 380192-76-7
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PRMT5-IN-46 (compound 278) inhibits PRMT5 with IC50s of 1-10 μM. PRMT5 is a methyltransferase which is associated with specific genetic alterations. PRMT5-IN-46 can be used for research of proliferative diseases, metabolic disorders, and blood diseases .
Cat. No.: HY-16771B
CAS No.: 1639208-51-7
Synonyms: NBI-98854 dihydrochloride
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Valbenazine (NBI-98854) dihydrochloride is a VMAT2 inhibitor, used to treat tardive dyskinesia. Valbenazine dihydrochloride demonstrates potential benefit in alleviating symptoms associated with movement disorders resulting from chronic dopamine receptor blocking medications. Valbenazine dihydrochloride has been supported by evidence from preclinical models examining its role in the genetic factors linked to tardive dyskinesia.
Cat. No.: HY-B0607S
Synonyms: NTBC-d3; Nitisone-d3; SC0735-d3
Cat. No.: HY-W777526
CAS No.: 1246815-63-3
Cat. No.: HY-101203AR
CAS No.: 1459687-96-7
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GJ103 (sodium) (Standard) is the analytical standard of GJ103 (sodium) (HY-101203A). This product is intended for research and analytical applications. GJ103 sodium is a read-through compound that can induce read through of premature stop codons. GJ103 sodium has potential for the research of genetic disorders caused by nonsense mutations .
Cat. No.: HY-L090
2,618 compounds
Transcription is the essential first step in the conversion of the genetic information in the DNA into protein and the major point at which gene expression is controlled. Transcription of protein-coding genes is accomplished by the multi-subunit enzyme RNA polymerase II and an ensemble of ancillary proteins, called transcription factors (TFs). Transcription factors play an important role in the long-term regulation of cell growth, differentiation and responses to environmental cues. Deregulated transcription factors contribute to the pathogenesis of a plethora of human diseases, ranging from diabetes, inflammatory disorders and cardiovascular disease to many cancers, and thus these proteins hold great therapeutic potential.
MCE offers a unique collection of 2,618 compounds with validated transcription factor targets modulating properties. MCE transcription factor-targeted compound library is an effective tool for researching transcription factors as drug targets as well as modulation of TFs for different therapeutic applications.
Cat. No.: HY-L267
1,244 compounds
Protein synthesis is a fundamental process that converts genetic information into functional proteins and is essential for maintaining cellular growth, proliferation, differentiation, and metabolic homeostasis. Protein synthesis is precisely regulated by a complex network of signaling pathways, including mTOR, PI3K/Akt, MAPK/ERK, and eIF (eukaryotic translation initiation factor)-mediated signaling. These pathways finely control cellular protein synthesis by regulating translation initiation, ribosomal activity, and the expression and phosphorylation status of key translation factors. Dysregulation of protein synthesis is closely associated with the development and progression of various diseases, including cancer, metabolic disorders, neurodegenerative diseases, and immune-related disorders. Therefore, key proteins, kinases, and translation regulators within these pathways are of significant value for research and drug development.
MCE offers 1,244 small-molecule compounds targeting key components of protein synthesis signaling pathways, covering multiple targets involved in protein synthesis and translational regulation, as well as diverse modes of target modulation and functions. By selectively modulating the activity or function of different pathway components, these compounds can be used to investigate protein synthesis and translational regulatory networks, elucidate the roles of specific targets in cellular physiology and disease processes, and support disease mechanism studies, potential drug target validation, candidate drug evaluation, and the exploration of therapeutic strategies.
Cat. No.: HY-L102
1,899 compounds
Rare diseases are an important public-health issue and a challenge for the medical community. Most rare diseases are genetic disorders, which are often severely disabling, substantially affect life expectancy, and impair physical and mental abilities. Currently, there are about 7,000 identified rare diseases, together affecting 10% of the population. However, fewer than 6% of all rare diseases have an approved treatment option, highlighting their tremendous unmet needs in drug development. The process of repurposing drugs for new indications, compared with the development of novel orphan drugs, is a time-saving and cost-efficient method resulting in higher success rates, which can therefore drastically reduce the risk of drug development for rare diseases.
MCE carefully collects a unique of 1,899 compounds studied in preclinical, clinical trials or approved used in rare diseases treatment. MCE rare diseases drug library is a useful tool for the research of rare diseases. All compounds can provide corresponding indications for rare diseases.
Cat. No.: HY-L917
5,619 compounds
RNA is crucial for the regulation of numerous cellular processes and functions. With the in-depth study of disease mechanisms, processes such as RNA expression, splicing, translation, and stability regulation have become new targets for disease intervention. RNA has provided new therapeutic modalities for metabolic diseases, genetic disorders, and cancer patients, resulting in several innovative drugs.
MCE R&D team collected small molecules targeting RNA from the PDB, R-BIND, ROBIN, and internal database as the positive dataset, and non-targeting RNA small molecules from ROBIN as the negative dataset. Based on the GeminiMol pre-trained model, we encoded the molecules and calculated over 1700 molecular descriptors using Mordred as inputs for the model. Subsequently, we employed 13 deep learning models to learn from the data. All of which yielded good training results, with AUROCs greater than 0.75. Ultimately, we selected the Finetune model to screen HY-L901P, which exhibited the best classification performance, achieving an AUROC of 0.82 and a prediction accuracy of 0.76. We then applied filtering based on StaR rules (with at least two of the following properties: cLogP ≥ 1.5, Molar Refractivity ≥ 4, Relative Polar Surface Area ≤ 0.3) to obtain a library containing approximately 5,000 small molecule compounds targeting RNA. This library serves as a valuable tool for screening small molecules that interact with RNA.
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Protocols
Targeted therapy! The Capetin Prize winning "Click Chemistry" can be used like this!Targeted therapy! The Nobel Prize winning "Click Chemistry" can be used like this!A suitable model is crucial in drug screening experiments. Organs can mimic the three-dimensional functional structure of internal organs, have similar spatial organization to corresponding organs, maintain some key characteristics, and reproduce some physiological functions. They are widely used for modeling and personalized drug screening physiological functions.2025-02-06
