Associated Papers
Antisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene.
Martínez-Pizarro A et al.
Nucleic Acid Therapeutics, 2022
Pseudoexon exclusion by antisense therapy in 6-pyruvoyl-tetrahydropterin synthase deficiency.
Brasil S et al.
Human Mutation, 2011
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