negamycin and Disease-Models--Animal

In this paper I review the results of the treatments directed to modify the mRNA of dystrophin with the goal of converting the severe Duchenne type to the milder Becker muscular dystrophy. Antisense oligomers potential to modify Duchenne muscular dystrophy (DMD) gene expression and therapeutic strategies to induce ribosomal read-through of nonsense mutations (PTC124) are described. They are an important advance in the treatment of DMD, so far unspecific. Significant expression of new dystrophin is observed in biopsies of peripheral muscle, although the functional improvement is not so encouraging. New modification of chemistries are expected to improve the liberation, broad distribution in muscles, as well as their efficacy and safety enough to allow a positive chronic treatment of DMD.

Topics: Amino Acids, Diamino; Aminoglycosides; Animals; Clinical Trials, Phase I as Topic; Clinical Trials, Phase II as Topic; Codon, Nonsense; Disease Models, Animal; Dogs; Dystrophin; Exons; Gene Expression Regulation; Genetic Therapy; Humans; Mice; Mice, Inbred mdx; Morpholinos; Muscle, Skeletal; Muscular Dystrophy, Duchenne; Oligonucleotides; Oligonucleotides, Antisense; Oxadiazoles; Protein Biosynthesis; RNA Splicing; Suppression, Genetic