RNA-BASE GENE THERAPY OF PRIMARY MYOPATHIES: USE OF ANTISENSE RNAS FOR THE CORRECTION, AT THE POST-TRANSCRIPTIONAL LEVEL, OF MUTATED DYSTROPHIN PRE-MRNAS
- 2 Anni 1999/2001
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Pubblicazioni Scientifiche
- PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Δ48-50 DMD cells
