CCA tRNA nucleotidyltransferase 1, mitochondrial (TRNT1)

CCA tRNA nucleotidyltransferase 1, mitochondrial (TRNT1) is an essential nucleotidyltransferase enzyme that catalyzes the addition and repair of the conserved CCA sequence at the 3' end of tRNA molecules, a modification required for tRNA aminoacylation and subsequent protein translation. It is a mitochondrial enzyme encoded by the nuclear genome, playing a central role in both tRNA maturation and tRNA quality control by distinguishing stable tRNAs (adding CCA) from unstable or defective tRNAs (adding CCACCA, leading to degradation). Mutations in TRNT1 cause inherited diseases such as sideroblastic anemia with B-cell immunodeficiency, periodic fevers, and developmental delay, highlighting its crucial role in cellular and mitochondrial function[1][2][4][7]. No approved drugs directly target TRNT1, reflecting its essential housekeeping role and the likely toxicity of its inhibition.

Other names

tRNA nucleotidyltransferase 1TRNA Nucleotidyl Transferase 1CCA1MtCCACGI-47SIFDATP(CTP):tRNA nucleotidyltransferaseCCA tRNA nucleotidyltransferase 1, mitochondrialtRNA CCA Nucleotidyl Transferase 1mt tRNA CCA-Pyrophosphorylase

Drugs targeting this enzyme would theoretically inhibit or modify the CCA-adding function, interfering with tRNA maturation and mitochondrial protein synthesis (no therapeutic drugs currently known)