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There are several classes of antisense oligonucleotides (AONs) with therapeutic use, such as double-stranded RNAs (interfering RNAs, utilized for gene silencing, and single-stranded AONs with various chemistries, which are useful for antisense targeting of micro-RNAs and mRNAs. In particular, the use of AONs for exon skipping, by targeting pre-mRNA, is proving to be a highly promising therapy for some genetic disorders like Duchenne muscular dystrophy and spinal muscular atrophy. However, AONs are unable to cross the plasma membrane unaided, and several other obstacles still remain to be overcome, in particular their instability due to their nuclease sensitivity and their lack of tissue specificity. Various drug delivery systems have been explored to improve the bioavailability of nucleic acids, and nanoparticles (NPs) have been suggested as potential vectors for DNA\/RNA. This review describes the recent progress in AON conjugation with natural and synthetic delivery systems, and provides an overview of the efficacy of NP-AON complexes as an exon-skipping treatment for Duchenne muscular dystrophy.<\/jats:p>","DOI":"10.1089\/nat.2013.0450","type":"journal-article","created":{"date-parts":[[2014,2,7]],"date-time":"2014-02-07T15:20:37Z","timestamp":1391786437000},"page":"87-100","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":49,"title":["Nanoparticle Delivery of Antisense Oligonucleotides and Their Application in the Exon Skipping Strategy for Duchenne Muscular Dystrophy"],"prefix":"10.1177","volume":"24","author":[{"given":"Maria Sofia","family":"Falzarano","sequence":"first","affiliation":[{"name":"University of Ferrara","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chiara","family":"Passarelli","sequence":"additional","affiliation":[{"name":"University of Ferrara","place":["Italy"]},{"name":"Bambino Ges\u00f9 Children's Hospital","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Ferlini","sequence":"additional","affiliation":[{"name":"University of Ferrara","place":["Italy"]}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2014,2,7]]},"reference":[{"key":"e_1_3_3_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/978-1-61779-767-5_8"},{"key":"e_1_3_3_3_1","doi-asserted-by":"publisher","DOI":"10.1261\/rna.653607"},{"key":"e_1_3_3_4_1","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.696"},{"key":"e_1_3_3_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.coph.2005.07.001"},{"key":"e_1_3_3_6_1","first-page":"897076","article-title":"Persistent dystrophin protein restoration 90 days after a course of intraperitoneally administered naked 2'OMePS AON and ZM2 NP-AON complexes in mdx mice","author":"BASSI E.","year":"2012","unstructured":"BASSI E., , FALZARANO S., , FABRIS M., , GUALANDI F., , MERLINI L., , VATTEMI G., , PERRONE D., , MARCHESI E., , SABATELLI P., , SPARNACCI K., et al. 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