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Results for "

loss-of-function

" in MedChemExpress (MCE) Product Catalog:

8

Inhibitors & Agonists

3

Oligonucleotides

Cat. No. Product Name Target Research Areas Chemical Structure
  • HY-149555
    DNL343

    		Eukaryotic Initiation Factor (eIF) Neurological Disease
    DNL343 is a potent, selective, orally active and brain-penetrant activator of eukaryotic initiation factor eIF2B. DNL343 inhibits the activity of the integrated stress response (ISR) in the central nervous system (CNS) and reverses neurodegeneration and neuroinflammation. DNL343 also prevents motor dysfunction and premature death in eIF2B loss-of-function (LOF) mutant mice. DNL343 can be used in the study of neurodegenerative diseases .
    DNL343
  • HY-100886
    BAY1082439

    		PI3K Apoptosis Cancer
    BAY1082439 is an orally bioavailable, selective PI3Kα/β/δ inhibitor. BAY1082439 also inhibits mutated forms of PIK3CA. BAY1082439 is highly effective in inhibiting Pten-null prostate cancer growth .
    BAY1082439
  • HY-164221A
    Rapirosiran sodium

    ALN-HSD sodium

    		 17β-HSD Metabolic Disease
    Rapirosiran sodium is a N-acetylgalactosamine-conjugated small-interfering RNA targeting liver-expressed hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) mRNA. Loss-of-function variants in the HSD17B13 associated with reduced risk of chronic liver disease. Rapirosiran sodium can be used for the study of Non-alcoholic steatohepatitis.
    Rapirosiran sodium
  • HY-159709
    VL-422

    		Liposome Metabolic Disease
    VL-422 is an ionizable cationic lipid. VL-422 delivers CRISPR complementary single-guide RNA (sgRNA) and Cas9 mRNA to enable in vitro and in vivo gene editing. LNPs containing VL-422 loaded with Cas9 mRNA and sgRNA targeting the ANGPTL3 gene induce the deletion of premature stop codons within the ANGPTL3 gene in the liver of cynomolgus monkeys. Loss-of-function of ANGPTL3 leads to decreased levels of LDL, HDL and cholesterol in plasma. The VL-422 delivery system can be used for the research of gene editing strategies targeting lipid metabolism diseases .
    VL-422
  • HY-164221
    Rapirosiran

    ALN-HSD; ALN-288996; AD-288996

    		 17β-HSD Metabolic Disease
    Rapirosiran is a N-acetylgalactosamine-conjugated small-interfering RNA targeting liver-expressed hydroxysteroid 17-beta dehydrogenase 13 (HSD17B13) mRNA. Loss-of-function variants in the HSD17B13 associated with reduced risk of chronic liver disease. Rapirosiran can be used for the study of Non-alcoholic steatohepatitis.
    Rapirosiran
  • HY-115726
    FRM-024

    		γ-secretase Amyloid-β Neurological Disease
    FRM-024 is a central nervous system-penetrant, orally active γ-secretase modulator and Aβ42 lowering agent. FRM-024 is used for the research of familial Alzheimer's disease .
    FRM-024
  • HY-143615
    RET-IN-10

    		RET Cancer
    RET-IN-10 is a potent inhibitor of RET. RET loss of function mutations leads to Hirschsprung's disease, while its gain of function mutations is associated with a variety of human tumors. RET-IN-10 has the potential for the research of cancer diseases (extracted from patent WO2021135938A1, compound 18) .
    RET-IN-10
  • HY-100886R
    BAY1082439 (Standard)

    		PI3K Reference Standards Apoptosis Cancer
    BAY1082439 (Standard) is the analytical standard of BAY1082439 (HY-100886). This product is intended for research and analytical applications. BAY1082439 is an orally bioavailable, selective PI3Kα/β/δ inhibitor. BAY1082439 also inhibits mutated forms of PIK3CA. BAY1082439 is highly effective in inhibiting Pten-null prostate cancer growth .
    BAY1082439 (Standard)

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