Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria

Ainhoa Martínez-Pizarro, Mar Álvarez, Maja Dembic, Caroline A. Lindegaard, Margarita Castro, Eva Richard, Brage S. Andresen, Lourdes R. Desviat*

*Corresponding author for this work

Research output: Contribution to journalJournal articleResearchpeer-review

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Biochemistry, Genetics and Molecular Biology