NFU1 iron-sulfur cluster scaffold protein (NFU1)

NFU1 iron-sulfur cluster scaffold protein is a mitochondrial assembly factor required for the maturation of a specific subset of iron-sulfur ([4Fe–4S]) cluster-containing proteins. It acts late in the iron-sulfur cluster (Fe–S) biogenesis pathway, receiving [2Fe–2S] cluster equivalents from precursor proteins (ISCU2 and ISCA1) and facilitating their assembly into [4Fe–4S] clusters to be delivered to client proteins, such as lipoic acid synthase and components of mitochondrial respiratory chain complexes I and II[1][2][4][5]. Mutations in NFU1 disrupt this maturation process, causing multiple mitochondrial dysfunctions syndrome, a disorder characterized by combined defects in mitochondrial energy metabolism—typically manifesting as fatal infantile encephalopathy, lactic acidosis, and failure of critical metabolic pathways[1][3][4]. NFU1 is not considered a classical drug target such as an enzyme, receptor, or transporter in therapeutic pharmacology, but is rather a key assembly factor essential for mitochondrial biology[1][4][5]. Notes on fields left ‘null’: - No approved or candidate drugs, nor established mechanisms of drug action targeting NFU1, are reported in the literature. - No established direct pharmacodynamic or patient-selection biomarkers (beyond deleterious variants for diagnostic purposes) are reported.

Other names

NFU1 iron-sulfur cluster scaffold homolog, mitochondrialHIRA-interacting protein 5HIRIP5CGI-33NifUNIFUCMMDFSMMDS1SPG93NifU-like C-terminal domain containingiron-sulfur cluster scaffold protein