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Rendu J, Brocard J, Denarier E, Monnier N, Piétri-Rouxel F, Beley C, Roux-Buisson N, Gilbert-Dussardier B, Perez MJ, Romero N, Garcia L, Lunardi J, Fauré J, Fourest-Lieuvin A, Marty I. Exon Skipping as a Therapeutic Strategy Applied to anRYR1Mutation with Pseudo-Exon Inclusion Causing a Severe Core Myopathy. Human Gene Therapy. 2013 Jul;24(7):702–13. doi: 10.1089/hum.2013.052. |