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hereditary

" in MedChemExpress (MCE) Product Catalog:

28

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2

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1

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1

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2

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6

Oligonucleotides

Cat. No. Product Name Target Research Areas Chemical Structure
  • HY-16735
    Avoralstat
    4 Publications Verification

    BCX4161

    Kallikrein Others
    Avoralstat (BCX4161), a potent and orally active plasma kallikrein (PKK) inhibitor, is used for hereditary angioedema research .
    Avoralstat
  • HY-P99631
    Garadacimab
    1 Publications Verification

    CSL312

    Factor Xa Cardiovascular Disease
    Garadacimab (CSL312) is a first-in-class, fully human IgG4 monoclonal antibody targeting activated factor XII (FXIIa). Garadacimab has the potential for hereditary angioedema research .
    Garadacimab
  • HY-P10272

    PTG-300

    Ferroportin Others
    Rusfertide is a peptide mimetic of natural hepcidin, which targets and degrades ferroportin, reduces serum iron and transferrin-saturation, and thus regulates the production of red blood cells. Rusfertide ameliorates the polycythemia vera, β-thalassemia and hereditary hemochromatosis .
    Rusfertide
  • HY-132608

    ISIS-420915 sodium

    Transthyretin (TTR) Neurological Disease
    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 .
    Inotersen sodium
  • HY-P99110

    SHP643; DX-2930

    Kallikrein Inflammation/Immunology
    Lanadelumab (SHP643) is a human IgG1 monoclonal antibody against plasma kallikrein (pKal) with an Ki value of 0.12 nM. Lanadelumab inhibits both free and HMWK (high molecular weight kininogen)-bound pKal. Lanadelumab has the potential for the research of hereditary angioedema .
    Lanadelumab
  • HY-12040
    Elesclomol
    Maximum Cited Publications
    55 Publications Verification

    STA-4783

    Reactive Oxygen Species Apoptosis Cuproptosis Cancer
    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 .
    Elesclomol
  • HY-163464

    Kallikrein Cardiovascular Disease
    Plasma kallikrein-IN-5 (Compound 20) is a potent covalent inhibitor of plasma potassium kinin peptide (Pka) with IC50 values of 66 nM and 70 pM at 1 minute and 24 hours, respectively. Plasma kallikrein-IN-5 can be used IN the study of hereditary angioedema (HAE) .
    Plasma kallikrein-IN-5
  • HY-143476

    Kallikrein Cardiovascular Disease Metabolic Disease
    Plasma kallikrein-IN-2 (Compound 198) is a potent plasma kallikrein (PKal) inhibitor with an IC50 of 0.1 nM. Plasma kallikrein-IN-2 can be used for hereditary angioedema, diabetic macular edema, and diabetic retinopathy research .
    Plasma kallikrein-IN-2
  • HY-163329

    Biochemical Assay Reagents Neurological Disease
    BCH-HSP-C01 is a lead compound that restores AP-4-dependent protein trafficking in neuronal models of adapter protein complex 4 (AP-4)-associated hereditary spastic paraplegia. BCH-HSP-C01 can be used for the research of autophagy-associated diseases .
    BCH-HSP-C01
  • HY-141876

    Histone Methyltransferase Cancer
    PRMT5-IN-14 is a PRMT5 inhibitor to research cancer, sickle cell, and hereditary persistence of foetal hemoglobin (HPFH) mutations.
    PRMT5-IN-14
  • HY-109127
    Berotralstat
    1 Publications Verification

    BCX7353

    Kallikrein Cancer
    Berotralstat (BCX7353) is an orally active plasma kallikrein inhibitor. Berotralstat can reduce brain edema and is being studied for glioblastoma and hereditary angioedema .
    Berotralstat
  • HY-139787A

    ISIS-721744; IONIS-PKK-LRX

    Kallikrein Others
    Donidalorsen (sodium) is an antisense oligonucleotide designed to reduce the production of prekallikrein (PKK). PKK plays an important role in the activation of inflammatory mediators associated with acute attacks of Hereditary angioedema (HAE).
    Donidalorsen sodium
  • HY-150052

    Kallikrein Cardiovascular Disease Metabolic Disease
    Plasma kallikrein-IN-3 is a plasma kallikrein inhibitor (IC50: 0.15 μM). Plasma kallikrein-IN-3 can be used for hereditary angioedema, diabetic macular edema, and diabetic retinopathy research .
    Plasma kallikrein-IN-3
  • HY-101207

    GABA Receptor Neurological Disease
    NCS-382 is a potent GABA receptor antagonist and also a GHBR receptor antagonist. NCS-382 has anticonvulsant and antisedative activity. NCS-382 is used in the related research of hereditary nervous system diseases .
    NCS-382
  • HY-132609

    Small Interfering RNA (siRNA) Transthyretin (TTR) Neurological Disease
    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 .
    Patisiran sodium
  • HY-W414498

    Endogenous Metabolite Neurological Disease
    1-Deoxysphingosine is elevated in lymphoblasts of individuals with hereditary sensory neuropathy type 1 (HSAN1) disorder; it features a (4E) double bond in its structure and is produced through the catabolism of 1-deoxyceramide catalyzed by the ceramidase enzyme, classifying it as an unsaturated deoxy-sphingoid base.
    1-Deoxysphingosine
  • HY-153609

    Transthyretin (TTR) Small Interfering RNA (siRNA) Neurological Disease
    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 .
    AS-Patisiran sodium
  • HY-B0607
    Nitisinone
    2 Publications Verification

    NTBC; Nitisone; SC0735

    HPPD Metabolic Disease
    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 .
    Nitisinone
  • HY-109127A
    Berotralstat dihydrochloride
    1 Publications Verification

    BCX7353 dihydrochloride

    Kallikrein Others
    Berotralstat dihydrochloride is a low toxicity, effective, highly specific, second-generation, synthetic and orally active plasma kallikrein inhibitor used for the research of hereditary angioedema (HAE) attacks. Berotralstat dihydrochloride works by blocking the enzymatic activity of plasma kallikrein in releasing bradykinin, the major biologic peptide that promotes swelling and pain associated with attacks of HAE .
    Berotralstat dihydrochloride
  • HY-128530

    Cuproptosis Cardiovascular Disease Metabolic Disease Inflammation/Immunology Cancer
    Tetrathiomolybdate, an orally active anti-copper agent, reduces copper levels in the body. Tetrathiomolybdate has a protective effect on collagen-induced arthritis in mice. Tetrathiomolybdate also reduces blood sugar, but has no effect on mice with hereditary diabetes (db/db). Tetrathiomolybdate inhibit angiogenesis, also shows antiangiogenic effects in malignant pleural mesothelioma .
    Tetrathiomolybdate
  • HY-111302

    Progesterone Receptor Endocrinology
    Norgestrienone is a progestin receptor agonist. Norgestrienone is commonly used as a progesterone compound in birth control pills and can be combined with ethinylestradiol. Norgestrienone is a click chemical. It contains Alkyne groups and can undergo copper-catalyzed azide-alkyne cycloaddition reactions (CuAAc) with molecules containing Azide groups. Norgestrienone can be used in the study of hereditary angioneurotic edema .
    Norgestrienone
  • HY-N2093
    Vicine
    1 Publications Verification

    Drug Metabolite Cardiovascular Disease Metabolic Disease
    Vicine, an alkaloid glycoside found mainly in fava beans, is toxic in individuals who have a hereditary loss of the enzyme glucose-6-phosphate dehydrogenase and may cause haemolytic anaemia.Vicine is an inactive compound that is hydrolysed by the intestinal microflora to a highly reactive free radical generating compound, the aglycone divicine when Vicine enters the body through food .
    Vicine
  • HY-145343A

    γ-secretase Amyloid-β Neurological Disease
    (9R)-RO7185876 (Compound example 16) is a γ-secretase inhibitor. (9R)-RO7185876 inhibits Αβ42 secretion. (9R)-RO7185876 can be used for the researches of Alzheimer's disease, cerebral amyloid angiopathy, hereditary cerebral hemorrhage with amyloidosis, multi-infarct dementia, dementia pugilistica or Down syndrome .
    (9R)-RO7185876
  • HY-B0607R

    NTBC (Standard); Nitisone (Standard); SC56735 (Standard)

    HPPD Metabolic Disease
    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 .
    Nitisinone (Standard)
  • HY-B0607S

    NTBC-d3; Nitisone-d3; SC0735-d3

    Isotope-Labeled Compounds HPPD Metabolic Disease
    Nitisinone-d3 (NTBC-d3) is deuterium labeled Nitisinone. 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 .
    Nitisinone-d3
  • HY-109127R

    Ser/Thr Protease Others
    Berotralstat (Standard) is the analytical standard of Berotralstat. This product is intended for research and analytical applications. Berotralstat (BCX7353) is a low toxicity, effective, highly specific, second-generation, synthetic and orally active plasma kallikrein inhibitor used for the research of hereditary angioedema (HAE) attacks. Berotralstat works by blocking the enzymatic activity of plasma kallikrein in releasing bradykinin, the major biologic peptide that promotes swelling and pain associated with attacks of HAE .
    Berotralstat (Standard)
  • HY-160928

    E1/E2/E3 Enzyme Cardiovascular Disease
    Smurf1 modulator-1 (Compound 20) is a selective inhibitor for Smad ubiquitination regulatory factor 1 (Smurf1), with IC50 of 180 nM .
    Smurf1 modulator-1
  • HY-150229

    Liposome Cancer
    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 .
    306-N16B