Pumilio RNA-binding protein 1 (PUM1)

Pumilio RNA-binding protein 1 (PUM1) is a highly conserved member of the PUF family of RNA-binding proteins. It binds specific sequence motifs ("UGUA") within the 3′ untranslated regions of target mRNAs, regulating their stability, decay, and translation. PUM1 affects embryogenesis, cell development, stem cell self-renewal, and proper differentiation. It is mechanistically integral to cell cycle progression, genome stability, and responses to cellular stress. Dysregulation or mutation of PUM1 is implicated in cancer, neurological disorders such as spinocerebellar ataxia, and abnormal hematopoiesis. Its molecular structure features a signature Pumilio homology domain composed of eight repeats conferring RNA sequence specificity. Therapeutic targeting is in the exploratory stage, with no approved drugs currently.

Other names

Pumilio homolog 1PUM1KIAA0099PUMH1HsPUMPumilio-1HSPUMNEDMSFPUML1SCA47

Drugs (hypothetical/future) would interfere with RNA binding or modulation of mRNA decay/translation, altering cell-cycle regulation or stem cell maintenance. Experimental modulation could use antisense oligonucleotides, small molecules, or engineered proteins that block/prevent PUM1 interaction with target mRNAs.