{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,28]],"date-time":"2026-09-28T19:32:43Z","timestamp":1790623963480,"version":"4.1.0"},"reference-count":43,"publisher":"SAGE Publications","issue":"3","license":[{"start":{"date-parts":[[2021,6,1]],"date-time":"2021-06-01T00:00:00Z","timestamp":1622505600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Nucleic Acid Therapeutics"],"published-print":{"date-parts":[[2021,6,1]]},"abstract":"<jats:p>Orphan drugs, including antisense oligonucleotides (AONs), siRNAs\/miRNAs, Cas9 nuclease, and recombinant genes, have recently been made available for rare diseases. However, the main bottleneck for these new therapies is delivery. Drugs\/synthetic genes need to reach the affected tissues with minimal off-target effects and immune reactions. AON molecules are currently delivered as backboned naked compounds or via viral vectors. Nanocarriers are considered promising vehicles, able to improve drug distribution by organ targeting and limiting safety issues.<\/jats:p>\n                  <jats:p>\n                    We tested perfluoropentane-based nanobubbles (NBs) as vehicles for loading phosphorodiamidate morpholino (PMO) AON to suppress DUX4 expression in a facioscapulohumeral muscular dystrophy cell model.\n                    <jats:italic toggle=\"yes\">In vitro<\/jats:italic>\n                    cell-free analysis demonstrated a good loading capacity of PMO into NBs, while experiments in cell cultures showed lack of therapeutic effect since expression of DUX4 and its targets remained unmodified. We conclude that these types of chitosan-shelled NBs do not release PMO-AON and are therefore not ideal for PMO AON-related therapies.\n                  <\/jats:p>","DOI":"10.1089\/nat.2020.0862","type":"journal-article","created":{"date-parts":[[2020,7,17]],"date-time":"2020-07-17T17:49:23Z","timestamp":1595008163000},"page":"201-207","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":15,"title":["Chitosan-Shelled Nanobubbles Irreversibly Encapsulate Morpholino Conjugate Antisense Oligonucleotides and Are Ineffective for Phosphorodiamidate Morpholino-Mediated Gene Silencing of\n                    <i>DUX4<\/i>"],"prefix":"10.1177","volume":"31","author":[{"given":"Maria Sofia","family":"Falzarano","sequence":"first","affiliation":[{"name":"Unit of Medical Genetics, University of Ferrara, Ferrara, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica","family":"Argenziano","sequence":"additional","affiliation":[{"name":"Department of Scienza e Tecnologia del Farmaco, University of Torino, Torino, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anne Chalotte","family":"Marsollier","sequence":"additional","affiliation":[{"name":"NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Virginie","family":"Mariot","sequence":"additional","affiliation":[{"name":"NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Davide","family":"Rossi","sequence":"additional","affiliation":[{"name":"Department of Scienza e Tecnologia del Farmaco, University of Torino, Torino, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rita","family":"Selvatici","sequence":"additional","affiliation":[{"name":"Unit of Medical Genetics, University of Ferrara, Ferrara, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Julie","family":"Dumonceaux","sequence":"additional","affiliation":[{"name":"NIHR Biomedical Research Centre, University College London, Great Ormond Street Institute of Child Health and Great Ormond Street Hospital NHS Trust, London, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roberta","family":"Cavalli","sequence":"additional","affiliation":[{"name":"Department of Scienza e Tecnologia del Farmaco, University of Torino, Torino, Italy."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Ferlini","sequence":"additional","affiliation":[{"name":"Unit of Medical Genetics, University of Ferrara, Ferrara, Italy."},{"name":"The Dubowitz Neuromuscular Centre, UCL Street Institute of Child Health, London, United Kingdom."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2021,6,4]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41573-019-0049-9"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.3390\/cells9010137"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2016.0657"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1186\/s40348-018-0081-6"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddy171"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1177\/1756285618754501"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.3390\/ijms19051347"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.1007\/s13311-013-0194-5"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkx1239"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkw236"},{"key":"e_1_3_5_12_2","doi-asserted-by":"publisher","DOI":"10.15252\/emmm.201607199"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.3390\/molecules22040563"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.2174\/1381612824666180110151318"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.addr.2016.04.001"},{"key":"e_1_3_5_16_2","first-page":"458","article-title":"Polymer nanoparticles for smart drug delivery","author":"Bennet D","year":"2014","unstructured":"Bennet D, , and K Sanghyo. 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