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 DYSTROPHY

摘要

A nanoparticle of the core-shell type is described, which is suitable for delivering therapeu¬ tic oligonucleotides into muscle fibres, particularly antisense oligoribonucleotides capable of restoring, in all or in part, the expression of the dystrophin protein by the exon skipping mecha¬ nism, and thus effective in the treatment of Duchenne muscular dystrophy. Moreover, a nanoparti- cle/oligonucleotide complex effective in the treatment of Duchenne muscular dystrophy is de¬ scribed.