A nanoparticle of the core-shell type is described, which is suitable for delivering therapeutic oligonucleotides into muscle fibers, particularly antisense oligoribonucleotides capable of restoring, in all or in part, the expression of the dystrophin protein by the exon skipping mechanism, and thus effective in the treatment of Duchenne muscular dystrophy. Moreover, a nanoparticle/oligonucleotide complex effective in the treatment of Duchenne muscular dystrophy is described.
Nanoparticle of the core-shell type suitable for delivering therapeutic oligonucleotides to target tissues and the use thereof for the preparation of a medicament for treating duchenne muscular distrophy
FERLINI, Alessandra;MEDICI, Alessandro;PERRONE, Daniela;RIMESSI, Paola;
2011
Abstract
A nanoparticle of the core-shell type is described, which is suitable for delivering therapeutic oligonucleotides into muscle fibers, particularly antisense oligoribonucleotides capable of restoring, in all or in part, the expression of the dystrophin protein by the exon skipping mechanism, and thus effective in the treatment of Duchenne muscular dystrophy. Moreover, a nanoparticle/oligonucleotide complex effective in the treatment of Duchenne muscular dystrophy is described.File in questo prodotto:
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