Splice-Switching Antisense Oligonucleotides Correct Phenylalanine Hydroxylase Exon 11 Skipping Defects and Rescue Enzyme Activity in Phenylketonuria
Article in Nucleic Acid Therapeutics (April 2024)
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Article in Nucleic Acid Therapeutics (April 2024)
Article in Nucleic Acids Research (February 2024)
Book chapter (February 2024)