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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
59 Results for "
hereditary
" in MedChemExpress (MCE) Product Catalog:
Products (59)
59 Results for "hereditary" in MCE Product Catalog:
130
130 Publications Verification
Synonyms: STA-4783
Research Areas:
Elesclomol (STA-4783) is a potent copper ionophore and promotes copper-dependent cell death (cuproptosis). Elesclomol specifically binds ferredoxin 1 (FDX1) α2/α3 helices and β5 strand. Elesclomol inhibits FDX1-mediated Fe-S cluster biosynthesis. Elesclomol is an oxidative stress inducer that induces cancer cell apoptosis. Elesclomol is a reactive oxygen species (ROS) inducer. Elesclomol can be used for Menkes and associated disorders of hereditary copper deficiency research .
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Avoralstat (BCX4161), a potent and orally active plasma kallikrein (PKK) inhibitor, is used for hereditary angioedema research .
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3 Cited Publications
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DMT1 blocker 1 is an orally active blocker of divalent metal transporter 1 (DMT1) with an IC50 of 0.64 μM. DMT1 blocker 1 inhibits intestinal cell absorption of non-heme iron, thereby alleviating iron overload by blocking the DMT1 transporter. DMT1 blocker 1 demonstrates significant efficacy in rodent models of acute iron hyperabsorption. DMT1 blocker 1 is useful for studying iron overload disorders such as hereditary hemochromatosis and thalassemia .
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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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2
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Alkaloids Leguminosae Other Alkaloids Metabolic Disease Plants Vicia faba L. Disease Research Fields
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1 Cited Publications
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Patisiran sodium is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran sodium specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran sodium can be used for the research of hereditary TTR amyloidosis .
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Berotralstat (BCX7353) is an orally active plasma kallikrein inhibitor. Berotralstat can reduce brain edema and is being studied for glioblastoma and hereditary angioedema .
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1 Cited Publications
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gamma-secretase modulator 1 is a Gamma-secretase modulator. gamma-secretase modulator 1 can be used in the research of Alzheimer's disease, multi-infarct dementia and Down syndrome .
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1 Cited Publications
Synonyms: BCX7353 dihydrochloride
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Berotralstat dihydrochloride (BCX7353 dihydrochloride) is an orally active plasma kallikrein inhibitor. Berotralstat can reduce brain edema and is being studied for glioblastoma and hereditary angioedema .
Cat. No.: HY-P10272
CAS No.: 1628323-80-7
Synonyms: PTG-300
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Synonyms: ISIS-420915 sodium
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Inotersen (ISIS-420915) sodium is a 2′-O-methoxyethyl-modified antisense oligonucleotide. Inotersen sodium inhibits the production of transthyretin (TTR) protein by targeting the TTR RNA transcript and reduces the levels of the TTR transcript. Inotersen sodium can be used for the research of hereditary TTR amyloidosis polyneuropathy .
Cat. No.: HY-112974
CAS No.: 1492984-65-2
Synonyms: GSK-2998728; ISIS-420915
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Inotersen (GSK-2998728; ISIS-420915) is a 2'-O-methoxyethyl-modified antisense oligonucleotide and transthyretin (TTR) inhibitor with low genotoxicity. Inotersen triggers RNase H1-mediated degradation by binding to TTR mRNA, thereby effectively reducing the production of both mutant and wild-type transthyretin in the liver. Inotersen significantly reduces amyloid fiber deposition, yet specific toxicities such as inflammation or tumors are observed at high doses in some animal models. Inotersen is used in studies of hereditary transthyretin amyloidosis and the associated polyneuropathy and cardiomyopathy .
Cat. No.: HY-173189A
Synonyms: 2′,5′-ApApA pentasodium; 2′,5′-trioligoadenylate pentasodium; 5'-O-Triphosphoryladenylyl-(2'→5')-adenylyl-(2'→5')-adenosine pentasodium
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2-5A (2′,5′-ApApA) pentasodium solution 100 mM is a high-affinity (Ka=0.04 nM) RNase L binder. 2-5A pentasodium solution 100 mM activates the nuclease activity of latent RNase L to cleave single-stranded RNA by inducing conformational changes and dimerization of latent RNase L, thereby mediating critical antiviral responses and enhancing interferon effects. 2-5A pentasodium solution 100 mM not only effectively inhibits Rauscher murine leukemia virus replication, reduces viremia and splenomegaly, but also enhances antileukemic efficacy when combined with amphotericin B and exhibits favorable high-dose tolerance. 2-5A pentasodium solution 100 mM is an important tool molecule for investigating the pathological mechanisms of viral infection, hereditary prostate cancer and leukemia .
Cat. No.: HY-P99110
CAS No.: 1426055-14-2
Synonyms: SHP643; DX-2930; X124-G01
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Cat. No.: HY-150151A
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GalNac-siRNA negative control (21nt) is the negative control form of GalNac-siRNA. GalNac-siRNA is an Asialoglycoprotein receptor (ASGPR)-targeted inhibitor conjugate. GalNac-siRNA is promising for research of hereditary transthyretin amyloidosis, acute hepatic porphyria, hemophilia and hypercholesterolemia .
Cat. No.: HY-139787
CAS No.: 2304692-48-4
Synonyms: ISIS-721744 free acid; IONIS-PKK-LRX free acid
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Donidalorsen (ISIS-721744; IONIS-PKK-LRX free acid) is an antisense oligonucleotide targeting prekallikrein (PKK). Donidalorsen inhibits kallikrein activity and reduces the production of Bradykinin (HY-P0206) by specifically binding to and degrading PKK mRNA in the liver. Donidalorsen can be used in the research of hereditary angioedema .
Cat. No.: HY-139787A
CAS No.: 2304701-45-7
Synonyms: ISIS-721744; IONIS-PKK-LRX
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Donidalorsen (ISIS-721744; IONIS-PKK-LRX) sodium is an antisense oligonucleotide targeting prekallikrein (PKK). Donidalorsen sodium inhibits kallikrein activity and reduces the production of Bradykinin (HY-P0206) by specifically binding to and degrading PKK mRNA in the liver. Donidalorsen sodium can be used in the research of hereditary angioedema .
Cat. No.: HY-U00461
CAS No.: 2102501-84-6
Synonyms: Ketohexokinase inhibitor 1
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PF-06835919 (Ketohexokinase inhibitor 1) is a ketohexokinase (KHK) inhibitor, with IC50 values of 0.028 μM and 0.17 μM against human KHK-C and KHK-A, respectively, and an IC50 value of 0.21 μM against rat KHK. PF-06835919 blocks fructose (HY-N0395) phosphorylation and fructose metabolism. PF-06835919 can be used for research on metabolic dysfunction-associated steatotic liver disease, type 2 diabetes, hereditary fructose intolerance, and related conditions .
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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
