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UPF3A regulator of nonsense-mediated mRNA decay (UPF3A) is a protein involved in the nonsense-mediated mRNA decay (NMD) pathway, which is essential for mRNA quality control by degrading transcripts that harbor premature stop codons[7][1][3][2][5]. UPF3A is one of two paralogs in vertebrates (the other being UPF3B) and has structural domains enabling RNA binding and interaction with core NMD proteins such as UPF2[3]. While UPF3A is dispensable for canonical NMD activity under most conditions (particularly when UPF3B is present), it can have minor, tissue-specific roles in modulating NMD efficiency and may compensate for UPF3B deficiency in certain contexts[1][2][5]. The functional interplay between UPF3A and UPF3B is complex: UPF3B is generally the stronger NMD activator; UPF3A may have weak NMD-activating or even context-dependent antagonistic effects[1][2][5]. Mutations in UPF3B can lead to neurodevelopmental disorders, and altered UPF3A expression often appears as a compensatory mechanism in these cases[3][4][5]. UPF3A is not a typical therapeutic target such as a receptor or enzyme, and no clinically relevant drugs are known to interact with it directly.
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