nanoparticella type core - shell suitable for the channelling of oligonucleotidi therapeutic target tissue and its use for the preparation of a medicament for the treatment of duchenne muscular dystrophy.

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专利权人:

UNIVERSITA DEGLI STUDI DI FERRARA;UNIVERSITA' DEGLI STUDI DI FERRARA

发明人:

FERLINI ALESSANDRA,MEDICI ALESSANDRO,SPARNACCI KATIA,PERRONE DANIELA,TONDELLI LUISA,RIMESSI PAOLA,LAUS MICHELE

申请号：

ITTO20090782

公开号：

IT1397011B1

申请日:

2009.10.14

申请国别(地区):

IT

年份:

2012

代理人:

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.

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