Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency
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Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency
Brasil S, Viecelli HM, Meili D, Rassi A, Desviat LR, Pérez B, Ugarte M, Thöny B. Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency. Hum Mutat. 2011 May 3. doi: 10.1002/humu.21529. [Epub ahead of print]
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