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Antisense Oligonucleotide Rescue of Deep-Intronic Variants Activating Pseudoexons in the 6-Pyruvoyl-Tetrahydropterin Synthase Gene.

Document Type

Article

Author

Martínez-Pizarro, Ainhoa; Leal, Fátima; Holm, Lise Lolle; Doktor, Thomas K.; Petersen, Ulrika S.S.; Bueno, María; Thöny, Beat; Pérez, Belén; Andresen, Brage S.; Desviat, Lourdes R.

Source

Nucleic Acid Therapeutics. Oct2022, Vol. 32 Issue 5, p378-390. 13p.

Subject

Language

ISSN

2159-3337

Online Access

Web of Science JCR 저널정보 Scopus Find it@PNU

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