Preferred Label : TMPRSS6 RNAi SLN124;
NCIt synonyms : RNA Interference Therapeutic SLN124; GalNAc-anti-TMPRSS6 siRNA Conjugate SLN124;
NCIt definition : An RNA interference (RNAi) therapeutic composed of the liver-targeted ligand N-acetylgalactosamine
(GalNAc) conjugated to small-interfering RNAs (siRNAs) directed against the serine
protease TMPRSS6, that may potentially be used in the treatment of iron overload associated
with certain disorders such as beta thalassemia and myelodysplastic syndromes. Upon
administration of TMPRSS6 RNAi SLN124, the GalNAc moiety targets and binds with high
affinity to asialoglycoprotein receptors (ASGPRs) expressed on hepatocytes. Once inside
the cell, the siRNAs bind to and silence TMPRSS6 mRNA and inhibit both the translation
and expression of the TMPRSS6 protein. This prevents TMPRSS6-mediated suppression
of the bone morphogenetic protein (BMP)-SMAD signaling pathway and leads to an increase
in hepcidin levels. Hepcidin decreases the uptake of dietary iron and the release
of iron from cellular storage, which may normalize iron levels. Hepcidin plays a key
role in the homeostasis of systemic iron; low levels of hepcidin are associated with
iron overload in certain disorders.;
Molecule name : SLN-124; SLN 124;
NCI Metathesaurus CUI : CL1411948;
Origin ID : C174396;
UMLS CUI : C5418083;
Semantic type(s)
- concept_is_in_subset
