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Results for "

hereditary transthyretin amyloidosis

" in MedChemExpress (MCE) Product Catalog:

7

Inhibitors & Agonists

1

Peptides

6

Oligonucleotides

Targets Recommended:
Cat. No. Nom du produit Target Áreas de investigación Chemical Structure
  • HY-132609
    Patisiran sodium
    1 Publications Verification

    		 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-132608
    Inotersen sodium

    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-112974
    Inotersen

    GSK-2998728; ISIS-420915

    		 Transthyretin (TTR) Cardiovascular Disease Neurological Disease
    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 .
    Inotersen
  • HY-150151A
    GalNac-siRNA negative control (21nt)

    		LYTACs Cardiovascular Disease
    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 .
    GalNac-siRNA negative control (21nt)
  • HY-153609
    AS-Patisiran sodium

    		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-132590A
    Revusiran sodium

    ALN-TTRSC sodium

    		 Small Interfering RNA (siRNA) Transthyretin (TTR) Neurological Disease
    Revusiran (ALN-TTRSC) sodium is an RNA interference agent targeting the mRNA of transthyretin (Transthyretin, TTR). Revusiran sodium mediates sequence-specific degradation of TTR mRNA through RNA interference, reduces the synthesis of TTR protein, binds to GalNAc ligands, and is taken up by hepatocytes via the asialoglycoprotein receptor. Revusiran sodium exhibits favorable nonclinical safety profiles. Revusiran sodium can be used in studies related to transthyretin-mediated amyloidosis .
    Revusiran sodium
  • HY-P11698
    Guanidino-G-Clamp-PNA

    		DNA Alkylator/Crosslinker Transthyretin (TTR) Cancer
    Guanidino-G-Clamp-PNA is a highly efficient sequence-specific RNA binder and gene silencer. Guanidino-G-Clamp-PNA precisely targets such targets as miR-155 or transthyretin (TTR) mRNA through base pairing: the former regulates tumor-related signaling pathways by reducing microRNA activity, while the latter inhibits the translation of harmful proteins via steric hindrance. Guanidino-G-Clamp-PNA effectively stabilizes DNA/RNA duplexes, induces cancer cell apoptosis, and suppresses tumor growth. In addition, Guanidino-G-Clamp-PNA can be conjugated with targeting ligands to improve tissue-specific delivery and reduce in vivo adverse reactions, and it can also enhance the splicing regulation efficacy of other oligonucleotide platforms (such as PMO) when integrated into them. Guanidino-G-Clamp-PNA is applicable to the research of various diseases including diffuse large B-cell lymphoma and hereditary transthyretin-related amyloidosis .
    Guanidino-G-Clamp-PNA

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