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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
67 Results for "
hereditary
" in MedChemExpress (MCE) Product Catalog:
Products (67)
67 Results for "hereditary" in MCE Product Catalog:
131
131 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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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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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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2
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Alkaloids Other Alkaloids Metabolic Disease Leguminosae Extract Plants Vicia faba L. Disease Research Fields
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1
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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Sebetralstat (KVD900) is an orally active and selective plasma kallikrein inhibitor, with an IC50 of 6.0 nM and a Ki of 3.0 nM against the human target. Sebetralstat binds to the active site of plasma kallikrein, inducing a conformational flip of Trp215 to form a U-shaped conformation; its P1 group interacts with the S1 pocket through hydrophobic interactions and displacement of high-energy water molecules, independent of the Asp189 ionic bond. Sebetralstat inhibits plasma kallikrein-kinin system activation in whole plasma, rapidly relieves laryngeal and abdominal attack symptoms of hereditary angioedema (HAE-C1INH), and reduces attack severity. Sebetralstat can be used in research related to hereditary angioedema (HAE) .
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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 .
1
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 .
1
1 Cited Publications
Cat. No.: HY-P73526
Purity: ≥ 95%, as determined by reducing SDS-PAGE.
Synonyms: SERPING1; Serine (Or Cysteine) Proteinase Inhibitor, Clade G (C1 Inhibitor), Member 1, (Angioedema, hereditary); Prev. C1NH; Serine/Cysteine Proteinase Inhibitor Clade G Member 1 Splice Variant 2; Plasma Protease C1 Inhibitor; Serine/Cysteine Proteinase I
Species:
Human
Source:
HEK293
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 .
306-N16B is a selective lung-targeted lipid nanoparticle that reversibly targets lung endothelial cells and specific immune cells through selective adsorption of a protein corona mediated by differences in tail structure (such as fibrinogen β/γ chain). 306-N16B binds to specific plasma proteins in the blood to form a protein corona, which guides the particles to be enriched in the lungs, releases mRNA and promotes target cell gene expression, exerts efficient lung cell transfection activity, and can precisely regulate gene delivery of different cell types in the lungs (such as endothelial cells and macrophages). 306-N16B can be used in gene therapy technologies for hereditary lung diseases including pulmonary lymphangioleiomyomatosis (LAM), restoring tumor suppressor function by delivering Tsc2 mRNA, and can also be used for lung-specific mRNA vaccines and gene editing therapies .
Cat. No.: HY-P99110
CAS No.: 1426055-14-2
Synonyms: SHP643; DX-2930; X124-G01
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Cat. No.: HY-P1488
CAS No.: 23815-89-6
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Bradykinin (1-5) is a metabolite of Bradykinin (HY-P0206) and a PAR inhibitor that inhibits thrombin-induced platelet aggregation by preventing protease-activated receptor (Protease Activated Receptor) cleavage and inhibiting Thrombin (MW 37kDa) (HY-114164). Bradykinin (1-5) is used in research on thrombosis, carrageenan-induced pleurisy, nasal allergy, and hereditary angioedema .
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-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-153609
Purity: 92.51%
Research Areas:
AS-Patisiran sodium is an antisense strand of Patisiran. Patisiran is a double-stranded small interfering RNA that targets a sequence within the transthyretin (TTR) messenger RNA. Patisiran specifically inhibits hepatic synthesis of mutant and wild-type TTR. Patisiran can be used for the research of hereditary TTR amyloidosis .
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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
