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The Poly(rC)-binding protein 1 (PCBP1)–Cystic fibrosis transmembrane conductance regulator (CFTR) mRNA regulatory complex is a ribonucleoprotein assembly involving the PCBP1 protein, also known as hnRNP E1, and the messenger RNA of the CFTR gene [Holla et al., 2015]. PCBP1 binds to specific cytosine-rich (C-rich) elements within the 3' untranslated region (3' UTR) of the CFTR mRNA, where it acts as a critical post-transcriptional regulator [Holla et al., 2015; UniProt: Q15365]. This interaction is essential for determining the stability and translational efficiency of the CFTR transcript, directly influencing the amount of functional CFTR protein produced and localized to the cell membrane [Holla et al., 2015]. In the context of Cystic Fibrosis, where mutations often lead to insufficient protein levels, modulating this complex represents a therapeutic strategy to increase the pool of available CFTR mRNA and protein [Amaral & Farinha, 2013]. Experimental approaches, such as the use of antisense oligonucleotides (ASOs), aim to stabilize the mRNA or enhance translation by targeting the regulatory sites within this complex [Ramalho et al., 2022].
Stabilization of CFTR mRNA and enhancement of translation through binding to the 3' untranslated region (UTR)
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