{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T02:47:50Z","timestamp":1790736470186,"version":"4.1.0"},"reference-count":33,"publisher":"SAGE Publications","issue":"6","license":[{"start":{"date-parts":[[2023,11,24]],"date-time":"2023-11-24T00:00:00Z","timestamp":1700784000000},"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":[[2023,12,1]]},"abstract":"<jats:p>\n                    One advantage of antisense oligonucleotides (ASOs) for drug development is their long-lasting gene knockdown after administration\n                    <jats:italic toggle=\"yes\">in vivo<\/jats:italic>\n                    . In this study, we examine the effect on gene expression after intraocular injection in target tissues in the eye. We examined expression levels of the\n                    <jats:italic toggle=\"yes\">Malat1<\/jats:italic>\n                    gene after intracameral or intravitreal (IV) injection of an anti-\n                    <jats:italic toggle=\"yes\">Malat1<\/jats:italic>\n                    ASO in corneal epithelium\/stroma, corneal endothelium, lens capsule epithelium, neurosensory retina, and retinal pigment epithelium\/choroid of the mouse eye. We assessed potency of the compound at 7 days as well as duration of the gene knockdown at 14, 28, 60, 90, and 120 days. The ASO was more potent when delivered by IV injection relative to intracameral injection, regardless of whether the tissues analyzed were at the front or back of the eye. For corneal endothelium, inhibition was &gt;50% after 120 days for ASO at 50\u2009\u03bcg. At IV dosages of 6\u2009\u03bcg, we observed &gt;75% inhibition of gene expression in the retina and lens epithelium for up to 120 days. ASOs have potential as long-lasting gene knockdown agents in the mouse eye, but efficacy varies depending on the specific ocular target tissue and injection protocol.\n                  <\/jats:p>","DOI":"10.1089\/nat.2023.0044","type":"journal-article","created":{"date-parts":[[2023,11,2]],"date-time":"2023-11-02T11:29:07Z","timestamp":1698924547000},"page":"339-347","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":11,"title":["Modulation of Gene Expression in the Eye with Antisense Oligonucleotides"],"prefix":"10.1177","volume":"33","author":[{"given":"Jiaxin","family":"Hu","sequence":"first","affiliation":[{"name":"Department of Pharmacology and Biochemistry, University of Texas, Southwestern Medical Center, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xin","family":"Gong","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, and University of Texas, Southwestern Medical Center, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yan","family":"Fan","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, and University of Texas, Southwestern Medical Center, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Selina","family":"Aguilar","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Biochemistry, University of Texas, Southwestern Medical Center, Dallas, Texas, USA."},{"name":"Department of Ophthalmology, and University of Texas, Southwestern Medical Center, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank","family":"Rigo","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thahza P.","family":"Prakash","sequence":"additional","affiliation":[{"name":"Ionis Pharmaceuticals, Carlsbad, California, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8973-493X","authenticated-orcid":false,"given":"David R.","family":"Corey","sequence":"additional","affiliation":[{"name":"Department of Pharmacology and Biochemistry, University of Texas, Southwestern Medical Center, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"V. Vinod","family":"Mootha","sequence":"additional","affiliation":[{"name":"Department of Ophthalmology, and University of Texas, Southwestern Medical Center, Dallas, Texas, USA."},{"name":"Eugene McDermott Center for Human Growth and Development, University of Texas, Southwestern Medical Center, Dallas, Texas, USA."}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2023,11,24]]},"reference":[{"key":"e_1_3_6_2_2","doi-asserted-by":"publisher","DOI":"10.1093\/nar\/gkx1239"},{"key":"e_1_3_6_3_2","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-pharmtox-010716-104846"},{"key":"e_1_3_6_4_2","doi-asserted-by":"publisher","DOI":"10.1038\/nn.4508"},{"key":"e_1_3_6_5_2","doi-asserted-by":"publisher","DOI":"10.1177\/0883073815627882"},{"key":"e_1_3_6_6_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1702752"},{"key":"e_1_3_6_7_2","doi-asserted-by":"publisher","DOI":"10.1146\/annurev-vision-091718-014852"},{"key":"e_1_3_6_8_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamaophthalmol.2015.4776"},{"key":"e_1_3_6_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ophtha.2017.08.015"},{"key":"e_1_3_6_10_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ophtha.2016.04.049"},{"key":"e_1_3_6_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ophtha.2009.06.021"},{"key":"e_1_3_6_12_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ophtha.2010.08.012"},{"key":"e_1_3_6_13_2","doi-asserted-by":"publisher","DOI":"10.1371\/journal.pone.0049083"},{"key":"e_1_3_6_14_2","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.13-12611"},{"key":"e_1_3_6_15_2","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.14-16222"},{"key":"e_1_3_6_16_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamaophthalmol.2015.3430"},{"key":"e_1_3_6_17_2","doi-asserted-by":"publisher","DOI":"10.1097\/ICO.0000000000000018"},{"key":"e_1_3_6_18_2","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.17-22350"},{"key":"e_1_3_6_19_2","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M114.621607"},{"key":"e_1_3_6_20_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ajhg.2018.02.010"},{"key":"e_1_3_6_21_2","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.19-27641"},{"key":"e_1_3_6_22_2","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddy018"},{"key":"e_1_3_6_23_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2018.0764"},{"key":"e_1_3_6_24_2","doi-asserted-by":"publisher","DOI":"10.1089\/nat.2019.0838"},{"key":"e_1_3_6_25_2","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.17-22178"},{"key":"e_1_3_6_26_2","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.14-15390"},{"key":"e_1_3_6_27_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.preteyeres.2020.100883"},{"key":"e_1_3_6_28_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1414264"},{"key":"e_1_3_6_29_2","doi-asserted-by":"publisher","DOI":"10.1001\/jamaophthalmol.2016.1351"},{"key":"e_1_3_6_30_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0002-9394(02)01325-9"},{"key":"e_1_3_6_31_2","doi-asserted-by":"publisher","DOI":"10.1167\/iovs.10-6019"},{"key":"e_1_3_6_32_2","doi-asserted-by":"publisher","DOI":"10.1159\/000484126"},{"key":"e_1_3_6_33_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.crmeth.2021.100125"},{"key":"e_1_3_6_34_2","doi-asserted-by":"publisher","DOI":"10.1016\/0014-4835(83)90043-X"}],"container-title":["Nucleic Acid Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1089\/nat.2023.0044","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/nat.2023.0044","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1089\/nat.2023.0044","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,1]],"date-time":"2026-05-01T21:03:08Z","timestamp":1777669388000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1089\/nat.2023.0044"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,11,24]]},"references-count":33,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2023,12,1]]}},"alternative-id":["10.1089\/nat.2023.0044"],"URL":"https:\/\/doi.org\/10.1089\/nat.2023.0044","relation":{},"ISSN":["2159-3337","2159-3345"],"issn-type":[{"value":"2159-3337","type":"print"},{"value":"2159-3345","type":"electronic"}],"subject":[],"published":{"date-parts":[[2023,11,24]]}}}