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UPF1 silenced cellular model systems for screening of read-through agents active on β0 39 thalassemia point mutation

Nonsense mutations promote premature translational termination, introducing stop codons within the coding region of mRNAs and causing inherited diseases, including thalassemia. | UPF1 silenced cellular model systems for screening of read-through agents active on β0 39 thalassemia point mutation