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Regulating PCCA gene expression by modulation of pseudoexon splicing patterns to rescue enzyme activity in propionic acidemia

Document Type

Article

Author

Spangsberg Petersen, Ulrika Simone; Dembic, Maja; Martínez-Pizarro, Ainhoa; Richard, Eva; Holm, Lise Lolle; Havelund, Jesper Foged; Doktor, Thomas Koed; Larsen, Martin Røssel; Færgeman, Nils J.; Desviat, Lourdes Ruiz; Andresen, Brage Storstein

Source

In Molecular Therapy - Nucleic Acids 12 March 2024 35(1)

Subject

Language

ISSN

2162-2531

Online Access

Full Text (ScienceDirect 종량제) Open Access (EBSCO) Full Text (ScienceDirect O/A) Web of Science JCR 저널정보 Scopus Find it@PNU

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