A nanoparticle of the core-shell type is described, which is suitable for delivering therapeu not 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 not 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 not scribed
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
LAUS, Michele;SPARNACCI, Katia
2010-01-01
Abstract
A nanoparticle of the core-shell type is described, which is suitable for delivering therapeu not 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 not 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 not scribedFile in questo prodotto:
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