Regulating PCCA gene expression by modulation of pseudoexon splicing patterns to rescue enzyme activity in propionic acidemia
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M. Larsen | T. K. Doktor | L. L. Holm | B. Andresen | E. Richard | J. Havelund | N. Færgeman | L. Desviat | A. Martínez-Pizarro | M. Dembic | Ulrika S. S. Petersen