{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,13]],"date-time":"2026-05-13T02:04:58Z","timestamp":1778637898992,"version":"3.51.4"},"reference-count":53,"publisher":"MDPI AG","issue":"10","license":[{"start":{"date-parts":[[2021,10,9]],"date-time":"2021-10-09T00:00:00Z","timestamp":1633737600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"European Union\u2019s Horizon 2020 research and innovation programme under the Marie Sk\u0142odowska-Curie grant agreement","award":["722717"],"award-info":[{"award-number":["722717"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pharmaceutics"],"abstract":"<jats:p>Barded-Biedl syndrome (BBS) is a rare genetic disorder with an unmet medical need for retinal degeneration. Small-molecule drugs were previously identified to slow down the apoptosis of photoreceptors in BBS mouse models. Clinical translation was not practical due to the necessity of repetitive invasive intravitreal injections for pediatric populations. Non-invasive methods of retinal drug targeting are a prerequisite for acceptable adaptation to the targeted pediatric patient population. Here, we present the development and functional testing of a non-invasive, topical, magnetically assisted delivery system, harnessing the ability of magnetic nanoparticles (MNPs) to cargo two drugs (guanabenz and valproic acid) with anti-unfolded protein response (UPR) properties towards the retina. Using magnetic resonance imaging (MRI), we showed the MNPs\u2019 presence in the retina of Bbs wild-type mice, and their photoreceptor localization was validated using transmission electron microscopy (TEM). Subsequent electroretinogram recordings (ERGs) demonstrated that we achieved beneficial biological effects with the magnetically assisted treatment translating the maintained light detection in Bbs\u2212\/\u2212 mice (KO). To our knowledge, this is the first demonstration of efficient magnetic drug targeting in the photoreceptors in vivo after topical administration. This non-invasive, needle-free technology expands the application of SMDs for the treatment of a vast spectrum of retinal degenerations and other ocular diseases.<\/jats:p>","DOI":"10.3390\/pharmaceutics13101650","type":"journal-article","created":{"date-parts":[[2021,10,10]],"date-time":"2021-10-10T21:37:29Z","timestamp":1633901849000},"page":"1650","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":19,"title":["Magnetically Assisted Drug Delivery of Topical Eye Drops Maintains Retinal Function In Vivo in Mice"],"prefix":"10.3390","volume":"13","author":[{"given":"Marco","family":"Bassetto","sequence":"first","affiliation":[{"name":"OZ Biosciences, Parc Scientifique de Luminy, Case 922, Zone Entreprise, CEDEX 9, 13288 Marseille, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Daniel","family":"Ajoy","sequence":"additional","affiliation":[{"name":"INSERM, Ciliopathies Modeling and Associated Therapies Group, Laboratoire de G\u00e9n\u00e9tique M\u00e9dicale, UMRS_U1112, F\u00e9d\u00e9ration de M\u00e9dicine Translationelle de Strasbourg, Universit\u00e9 de Strasbourg, 67085 Strasbourg, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Florent","family":"Poulhes","sequence":"additional","affiliation":[{"name":"OZ Biosciences, Parc Scientifique de Luminy, Case 922, Zone Entreprise, CEDEX 9, 13288 Marseille, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7349-8749","authenticated-orcid":false,"given":"Cathy","family":"Obringer","sequence":"additional","affiliation":[{"name":"INSERM, Ciliopathies Modeling and Associated Therapies Group, Laboratoire de G\u00e9n\u00e9tique M\u00e9dicale, UMRS_U1112, F\u00e9d\u00e9ration de M\u00e9dicine Translationelle de Strasbourg, Universit\u00e9 de Strasbourg, 67085 Strasbourg, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Aurelie","family":"Walter","sequence":"additional","affiliation":[{"name":"OZ Biosciences, Parc Scientifique de Luminy, Case 922, Zone Entreprise, CEDEX 9, 13288 Marseille, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nadia","family":"Messadeq","sequence":"additional","affiliation":[{"name":"INSERM, Institute of Genetics and Molecular and Cellular Biology (IGBMC), 67640 Illkrich-Graffenstaden, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Amir","family":"Sadeghi","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonranta 1C, 70211 Kuopio, Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jooseppi","family":"Puranen","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonranta 1C, 70211 Kuopio, Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marika","family":"Ruponen","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonranta 1C, 70211 Kuopio, Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mikko","family":"Kettunen","sequence":"additional","affiliation":[{"name":"Kuopio Biomedical Imaging Unit, A.I. Virtanen Institute for Molecular Sciences, University of Eastern Finland, Neulaniementie 2, 70150 Kuopio, Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elisa","family":"Toropainen","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonranta 1C, 70211 Kuopio, Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Arto","family":"Urtti","sequence":"additional","affiliation":[{"name":"School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Yliopistonranta 1C, 70211 Kuopio, Finland"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"H\u00e9l\u00e8ne","family":"Dollfus","sequence":"additional","affiliation":[{"name":"INSERM, Ciliopathies Modeling and Associated Therapies Group, Laboratoire de G\u00e9n\u00e9tique M\u00e9dicale, UMRS_U1112, F\u00e9d\u00e9ration de M\u00e9dicine Translationelle de Strasbourg, Universit\u00e9 de Strasbourg, 67085 Strasbourg, France"},{"name":"Laboratoire de G\u00e9n\u00e9tique M\u00e9dicale, UMRS_U1112, Institut de G\u00e9n\u00e9tique M\u00e9dicale d\u2019Alsace, F\u00e9d\u00e9ration de M\u00e9dicine Translationelle de Strasbourg, Hopiaux Universitaires de Strasbourg, Universit\u00e9 de Strasbourg, 67085 Strasbourg, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3838-0198","authenticated-orcid":false,"given":"Olivier","family":"Zelphati","sequence":"additional","affiliation":[{"name":"OZ Biosciences, Parc Scientifique de Luminy, Case 922, Zone Entreprise, CEDEX 9, 13288 Marseille, France"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vincent","family":"Marion","sequence":"additional","affiliation":[{"name":"INSERM, Ciliopathies Modeling and Associated Therapies Group, Laboratoire de G\u00e9n\u00e9tique M\u00e9dicale, UMRS_U1112, F\u00e9d\u00e9ration de M\u00e9dicine Translationelle de Strasbourg, Universit\u00e9 de Strasbourg, 67085 Strasbourg, France"},{"name":"ALMS Therapeutics, Parc d\u2019Innovation, 650 Boulevard Gonthier d\u2019Andernach, 67400 Illkirch-Graffenstaden, France"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"1968","published-online":{"date-parts":[[2021,10,9]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1016\/j.preteyeres.2016.12.001","article-title":"Pharmacokinetic aspects of retinal drug delivery","volume":"57","author":"Heikkinen","year":"2017","journal-title":"Prog. 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