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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, U.S.; Dembic, M.; Holm, L.L.; Havelund, J.F.; Doktor, T.K.; Larsen, M.R.; Færgeman, N.J.; Andresen, B.S.; Martínez-Pizarro, A.; Richard, E.; Desviat, L.R.

Source

In: Molecular Therapy Nucleic Acids. (Molecular Therapy Nucleic Acids, 12 March 2024, 35(1))

Subject

Language

English

ISSN

21622531

Online Access

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

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