{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T16:07:06Z","timestamp":1788883626233,"version":"build-2803163510"},"reference-count":60,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T00:00:00Z","timestamp":1788220800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,7,17]],"date-time":"2026-07-17T00:00:00Z","timestamp":1784246400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007745","name":"Massachusetts Lions Eye Research Fund Inc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007745","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000053","name":"National Eye Institute","doi-asserted-by":"publisher","award":["P30EYE003790"],"award-info":[{"award-number":["P30EYE003790"]}],"id":[{"id":"10.13039\/100000053","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Nucleic Acids"],"published-print":{"date-parts":[[2026,9]]},"DOI":"10.1016\/j.omtn.2026.103024","type":"journal-article","created":{"date-parts":[[2026,7,21]],"date-time":"2026-07-21T15:27:52Z","timestamp":1784647672000},"page":"103024","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Development of an antisense oligonucleotide targeting EFEMP1 in models relevant to macular degeneration"],"prefix":"10.1016","volume":"37","author":[{"given":"William P.","family":"Miller","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Said","family":"Arevalo-Alquichire","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula","family":"Perez-Corredor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rose","family":"Lin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anil","family":"Upreti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeysson","family":"Sanchez-Suarez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tim E.","family":"Vanderleest","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Marino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donovan","family":"Spencer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matthew","family":"Scribner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jebgy","family":"Vargas","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andres","family":"Muriel-Torres","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xinyao","family":"Hu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3899-7064","authenticated-orcid":false,"given":"Yvonne","family":"Adu-Rutledge","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bryan A.","family":"Kaplan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Karim W.","family":"Barake","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Audrey L.","family":"Gunawan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Aruvi","family":"Vijikumar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Reeya","family":"Shrestha","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anton","family":"Lennikov","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Leo A.","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joseph F.","family":"Arboleda-Velasquez","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"Peterson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4275-2510","authenticated-orcid":false,"given":"Sudhir","family":"Agrawal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vinod","family":"Vathipadiekal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carlos","family":"Loya","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2833-3697","authenticated-orcid":false,"given":"Michael","family":"O\u2019Hare","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2026.103024_bib1","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1093\/hmg\/ddt395","article-title":"Mouse genetics and proteomic analyses demonstrate a critical role for complement in a model of DHRD\/ML, an inherited macular degeneration","volume":"23","author":"Garland","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib2","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1093\/hmg\/ddx392","article-title":"Changes in extracellular matrix cause RPE cells to make basal deposits and activate the alternative complement pathway","volume":"27","author":"Fernandez-Godino","year":"2018","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib3","doi-asserted-by":"crossref","first-page":"2411","DOI":"10.1093\/hmg\/ddm198","article-title":"The R345W mutation in EFEMP1 is pathogenic and causes AMD-like deposits in mice","volume":"16","author":"Fu","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib4","doi-asserted-by":"crossref","first-page":"2423","DOI":"10.1093\/hmg\/ddm199","article-title":"Formation and progression of sub-retinal pigment epithelium deposits in Efemp1 mutation knock-in mice: a model for the early pathogenic course of macular degeneration","volume":"16","author":"Marmorstein","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib5","doi-asserted-by":"crossref","first-page":"13067","DOI":"10.1073\/pnas.202491599","article-title":"Aberrant accumulation of EFEMP1 underlies drusen formation in Malattia Leventinese and age-related macular degeneration","volume":"99","author":"Marmorstein","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2026.103024_bib6","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1038\/9722","article-title":"A single EFEMP1 mutation associated with both Malattia Leventinese and Doyne honeycomb retinal dystrophy","volume":"22","author":"Stone","year":"1999","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib7","doi-asserted-by":"crossref","first-page":"31","DOI":"10.1167\/iovs.65.6.31","article-title":"A Long-Term Retrospective Natural History Study of EFEMP1-Associated Autosomal Dominant Drusen","volume":"65","author":"de Guimar\u00e3es","year":"2024","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.omtn.2026.103024_bib8","doi-asserted-by":"crossref","first-page":"1455","DOI":"10.1167\/iovs.16-20955","article-title":"Deletion of Efemp1 Is Protective Against the Development of Sub-RPE Deposits in Mouse Eyes","volume":"58","author":"Stanton","year":"2017","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.omtn.2026.103024_bib9","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0068088","article-title":"Interaction of complement factor h and fibulin3 in age-related macular degeneration","volume":"8","author":"Wyatt","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.omtn.2026.103024_bib10","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1556\/EuJMI.2.2012.2.2","article-title":"The complement system: history, pathways, cascade and inhibitors","volume":"2","author":"Nesargikar","year":"2012","journal-title":"Eur. J. Microbiol. Immunol."},{"key":"10.1016\/j.omtn.2026.103024_bib11","doi-asserted-by":"crossref","first-page":"30469","DOI":"10.1074\/jbc.M403026200","article-title":"Tissue inhibitor of metalloproteinases-3 (TIMP-3) is a binding partner of epithelial growth factor-containing fibulin-like extracellular matrix protein 1 (EFEMP1). Implications for macular degenerations","volume":"279","author":"Klenotic","year":"2004","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2026.103024_bib12","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1038\/ng1294-352","article-title":"Mutations in the tissue inhibitor of metalloproteinases-3 (TIMP3) in patients with Sorsby's fundus dystrophy","volume":"8","author":"Weber","year":"1994","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib13","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1097\/IAE.0000000000000263","article-title":"Comparison of drusen and modifying genes in autosomal dominant radial drusen and age-related macular degeneration","volume":"35","author":"Sohn","year":"2015","journal-title":"Retina"},{"key":"10.1016\/j.omtn.2026.103024_bib14","doi-asserted-by":"crossref","first-page":"AMD160","DOI":"10.1167\/iovs.18-24882","article-title":"Soft Drusen in Age-Related Macular Degeneration: Biology and Targeting Via the Oil Spill Strategies","volume":"59","author":"Curcio","year":"2018","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.omtn.2026.103024_bib15","first-page":"466","article-title":"Structure, function, and pathology of Bruch\u2019s membrane","volume":"1","author":"Curcio","year":"2013","journal-title":"Retina"},{"key":"10.1016\/j.omtn.2026.103024_bib16","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1016\/j.exer.2005.04.012","article-title":"Esterified and unesterified cholesterol in drusen and basal deposits of eyes with age-related maculopathy","volume":"81","author":"Curcio","year":"2005","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.omtn.2026.103024_bib17","doi-asserted-by":"crossref","first-page":"134","DOI":"10.1038\/ng.3448","article-title":"A large genome-wide association study of age-related macular degeneration highlights contributions of rare and common variants","volume":"48","author":"Fritsche","year":"2016","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib18","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1167\/iovs.61.13.19","article-title":"Measuring the Contributions of Basal Laminar Deposit and Bruch's Membrane in Age-Related Macular Degeneration","volume":"61","author":"Sura","year":"2020","journal-title":"Investig. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.omtn.2026.103024_bib19","doi-asserted-by":"crossref","first-page":"1434","DOI":"10.1016\/S0140-6736(23)01520-9","article-title":"Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials","volume":"402","author":"Heier","year":"2023","journal-title":"Lancet"},{"key":"10.1016\/j.omtn.2026.103024_bib20","doi-asserted-by":"crossref","first-page":"1449","DOI":"10.1016\/S0140-6736(23)01583-0","article-title":"Efficacy and safety of avacincaptad pegol in patients with geographic atrophy (GATHER2): 12-month results from a randomised, double-masked, phase 3 trial","volume":"402","author":"Khanani","year":"2023","journal-title":"Lancet"},{"key":"10.1016\/j.omtn.2026.103024_bib21","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1177\/24741264231220224","article-title":"Retinal Vasculitis After Intravitreal Pegcetacoplan: Report From the ASRS Research and Safety in Therapeutics (ReST) Committee","volume":"8","author":"Witkin","year":"2024","journal-title":"J. Vitreoretin. Dis."},{"key":"10.1016\/j.omtn.2026.103024_bib22","doi-asserted-by":"crossref","first-page":"3","DOI":"10.1007\/978-1-0716-2010-6_1","article-title":"Introduction and History of the Chemistry of Nucleic Acids Therapeutics","volume":"2434","author":"Gait","year":"2022","journal-title":"Methods Mol. Biol."},{"key":"10.1016\/j.omtn.2026.103024_bib23","first-page":"14","article-title":"Fomivirsen approved for CMV retinitis","volume":"4","author":"Roehr","year":"1998","journal-title":"J. Int. Assoc. Physicians AIDS Care"},{"key":"10.1016\/j.omtn.2026.103024_bib24","doi-asserted-by":"crossref","first-page":"1061","DOI":"10.1016\/j.molmed.2020.08.007","article-title":"RNA Therapeutics Are Stepping Out of the Maze","volume":"26","author":"Agrawal","year":"2020","journal-title":"Trends Mol. Med."},{"key":"10.1016\/j.omtn.2026.103024_bib25","doi-asserted-by":"crossref","first-page":"369","DOI":"10.1080\/17460441.2026.2652440","article-title":"The impact of antisense oligonucleotide (ASO) therapeutics on the future of rare disease drug discovery","volume":"21","author":"Ruchi","year":"2026","journal-title":"Expert Opin. Drug Discov."},{"key":"10.1016\/j.omtn.2026.103024_bib26","doi-asserted-by":"crossref","first-page":"6486","DOI":"10.1002\/alz.14142","article-title":"Proteomic analyses reveal plasma EFEMP1 and CXCL12 as biomarkers and determinants of neurodegeneration","volume":"20","author":"Duggan","year":"2024","journal-title":"Alzheimer's Dement."},{"key":"10.1016\/j.omtn.2026.103024_bib27","article-title":"EFEMP1 Overexpression Contributes to Neovascularization in Age-Related Macular Degeneration","volume":"11","author":"Cheng","year":"2020","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtn.2026.103024_bib28","doi-asserted-by":"crossref","DOI":"10.1371\/annotation\/c6b85ec4-996d-4daf-863a-44260a888470","article-title":"Choroid sprouting assay: an ex vivo model of microvascular angiogenesis","volume":"8","author":"Shao","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.omtn.2026.103024_bib29","article-title":"Recent advances of anti-angiogenic inhibitors targeting VEGF\/VEGFR axis","volume":"14","author":"Wang","year":"2023","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtn.2026.103024_bib30","doi-asserted-by":"crossref","first-page":"2468","DOI":"10.1182\/bloodadvances.2022009129","article-title":"Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria","volume":"7","author":"Wong","year":"2023","journal-title":"Blood Adv."},{"key":"10.1016\/j.omtn.2026.103024_bib31","doi-asserted-by":"crossref","first-page":"395","DOI":"10.1016\/S1474-4422(23)00080-7","article-title":"Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study","volume":"22","author":"Howard","year":"2023","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.omtn.2026.103024_bib32","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1093\/hmg\/ddac187","article-title":"Complement factor B is critical for sub-RPE deposit accumulation in a model of Doyne honeycomb retinal dystrophy with features of age-related macular degeneration","volume":"32","author":"Crowley","year":"2023","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib33","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-021-89978-8","article-title":"Complement C5 is not critical for the formation of sub-RPE deposits in Efemp1 mutant mice","volume":"11","author":"Garland","year":"2021","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2026.103024_bib34","doi-asserted-by":"crossref","DOI":"10.1111\/acel.13011","article-title":"Late-onset retinal degeneration pathology due to mutations in CTRP5 is mediated through HTRA1","volume":"18","author":"Chekuri","year":"2019","journal-title":"Aging Cell"},{"key":"10.1016\/j.omtn.2026.103024_bib35","doi-asserted-by":"crossref","DOI":"10.1111\/acel.12710","article-title":"HTRA1, an age-related macular degeneration protease, processes extracellular matrix proteins EFEMP1 and TSP1","volume":"17","author":"Lin","year":"2018","journal-title":"Aging Cell"},{"key":"10.1016\/j.omtn.2026.103024_bib36","doi-asserted-by":"crossref","first-page":"3290","DOI":"10.1016\/j.devcel.2024.09.006","article-title":"Human iPSC-based disease modeling studies identify a common mechanistic defect and potential therapies for AMD and related macular dystrophies","volume":"59","author":"Dalvi","year":"2024","journal-title":"Dev. Cell"},{"key":"10.1016\/j.omtn.2026.103024_bib37","doi-asserted-by":"crossref","first-page":"363","DOI":"10.18632\/oncotarget.1620","article-title":"EFEMP1 induces \u03b3-secretase\/Notch-mediated temozolomide resistance in glioblastoma","volume":"5","author":"Hiddingh","year":"2014","journal-title":"Oncotarget"},{"key":"10.1016\/j.omtn.2026.103024_bib38","doi-asserted-by":"crossref","first-page":"43518","DOI":"10.18632\/oncotarget.9846","article-title":"HIF2\u03b1\/EFEMP1 cascade mediates hypoxic effects on breast cancer stem cell hierarchy","volume":"7","author":"Kwak","year":"2016","journal-title":"Oncotarget"},{"key":"10.1016\/j.omtn.2026.103024_bib39","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1016\/j.biocel.2016.09.026","article-title":"Prolyl hydroxylase domain enzymes and their role in cell signaling and cancer metabolism","volume":"80","author":"Nguyen","year":"2016","journal-title":"Int. J. Biochem. Cell Biol."},{"key":"10.1016\/j.omtn.2026.103024_bib40","doi-asserted-by":"crossref","first-page":"31277","DOI":"10.1074\/jbc.M110.158485","article-title":"Stabilization of hypoxia-inducible factor-1alpha protein in hypoxia occurs independently of mitochondrial reactive oxygen species production","volume":"285","author":"Chua","year":"2010","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2026.103024_bib41","article-title":"Action Sites and Clinical Application of HIF-1\u03b1 Inhibitors","volume":"27","author":"Xu","year":"2022","journal-title":"Molecules"},{"key":"10.1016\/j.omtn.2026.103024_bib42","doi-asserted-by":"crossref","first-page":"628","DOI":"10.1038\/cdd.2008.17","article-title":"Biology of hypoxia-inducible factor-2alpha in development and disease","volume":"15","author":"Patel","year":"2008","journal-title":"Cell Death Differ."},{"key":"10.1016\/j.omtn.2026.103024_bib43","doi-asserted-by":"crossref","first-page":"435","DOI":"10.1007\/s10059-010-0067-2","article-title":"HIF-1 and HIF-2 Transcription Factors - Similar but Not Identical","volume":"29","author":"Loboda","year":"2010","journal-title":"Mol. Cells"},{"key":"10.1016\/j.omtn.2026.103024_bib44","doi-asserted-by":"crossref","DOI":"10.3389\/fphar.2023.1304342","article-title":"Antisense oligonucleotides: a novel Frontier in pharmacological strategy","volume":"14","author":"Collotta","year":"2023","journal-title":"Front. Pharmacol."},{"key":"10.1016\/j.omtn.2026.103024_bib45","article-title":"Durable vision improvement after a single intravitreal treatment with antisense oligonucleotide in CEP290-LCA: Replication in two eyes","volume":"32","author":"Cideciyan","year":"2023","journal-title":"Am. J. Ophthalmol. Case Rep."},{"key":"10.1016\/j.omtn.2026.103024_bib46","doi-asserted-by":"crossref","first-page":"1014","DOI":"10.1038\/s41591-022-01755-w","article-title":"Intravitreal antisense oligonucleotide sepofarsen in Leber congenital amaurosis type 10: a phase 1b\/2 trial","volume":"28","author":"Russell","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.omtn.2026.103024_bib47","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1038\/s41431-019-0546-7","article-title":"Biallelic variants in EFEMP1 in a man with a pronounced connective tissue phenotype","volume":"28","author":"Driver","year":"2020","journal-title":"Eur. J. Hum. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib48","doi-asserted-by":"crossref","first-page":"9679","DOI":"10.1038\/s41598-018-28143-0","article-title":"C3a triggers formation of sub-retinal pigment epithelium deposits via the ubiquitin proteasome pathway","volume":"8","author":"Fernandez-Godino","year":"2018","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtn.2026.103024_bib49","doi-asserted-by":"crossref","first-page":"469","DOI":"10.3389\/fcell.2020.00469","article-title":"Expression of R345W-Fibulin-3 Induces Epithelial-Mesenchymal Transition in Retinal Pigment Epithelial Cells","volume":"8","author":"Zhou","year":"2020","journal-title":"Front. Cell Dev. Biol."},{"key":"10.1016\/j.omtn.2026.103024_bib50","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1016\/j.ymeth.2013.06.027","article-title":"Kraken: A set of tools for quality control and analysis of high-throughput sequence data","volume":"63","author":"Davis","year":"2013","journal-title":"Methods"},{"key":"10.1016\/j.omtn.2026.103024_bib51","doi-asserted-by":"crossref","first-page":"3047","DOI":"10.1093\/bioinformatics\/btw354","article-title":"MultiQC: summarize analysis results for multiple tools and samples in a single report","volume":"32","author":"Ewels","year":"2016","journal-title":"Bioinformatics"},{"key":"10.1016\/j.omtn.2026.103024_bib52","doi-asserted-by":"crossref","first-page":"907","DOI":"10.1038\/s41587-019-0201-4","article-title":"Graph-based genome alignment and genotyping with HISAT2 and HISAT-genotype","volume":"37","author":"Kim","year":"2019","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2026.103024_bib53","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1038\/nbt.3122","article-title":"StringTie enables improved reconstruction of a transcriptome from RNA-seq reads","volume":"33","author":"Pertea","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2026.103024_bib54","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1186\/s13059-014-0550-8","article-title":"Moderated estimation of fold change and dispersion for RNA-seq data with DESeq2","volume":"15","author":"Love","year":"2014","journal-title":"Genome Biol."},{"key":"10.1016\/j.omtn.2026.103024_bib55","doi-asserted-by":"crossref","first-page":"15545","DOI":"10.1073\/pnas.0506580102","article-title":"Gene set enrichment analysis: a knowledge-based approach for interpreting genome-wide expression profiles","volume":"102","author":"Subramanian","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2026.103024_bib56","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1038\/ng1180","article-title":"PGC-1alpha-responsive genes involved in oxidative phosphorylation are coordinately downregulated in human diabetes","volume":"34","author":"Mootha","year":"2003","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2026.103024_bib57","doi-asserted-by":"crossref","first-page":"1523","DOI":"10.1038\/s41467-019-09234-6","article-title":"Metascape provides a biologist-oriented resource for the analysis of systems-level datasets","volume":"10","author":"Zhou","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtn.2026.103024_bib58","doi-asserted-by":"crossref","first-page":"D766","DOI":"10.1093\/nar\/gky955","article-title":"GENCODE reference annotation for the human and mouse genomes","volume":"47","author":"Frankish","year":"2019","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtn.2026.103024_bib59","doi-asserted-by":"crossref","DOI":"10.3791\/61677","article-title":"An Ex Vivo Choroid Sprouting Assay of Ocular Microvascular Angiogenesis","author":"Tomita","year":"2020","journal-title":"J. Vis. Exp."},{"key":"10.1016\/j.omtn.2026.103024_bib60","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1093\/nar\/30.1.207","article-title":"Gene Expression Omnibus: NCBI gene expression and hybridization array data repository","volume":"30","author":"Edgar","year":"2002","journal-title":"Nucleic Acids Res."}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126001952?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126001952?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T15:13:37Z","timestamp":1788880417000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253126001952"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,9]]},"references-count":60,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,9]]}},"alternative-id":["S2162253126001952"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2026.103024","relation":{},"ISSN":["2162-2531"],"issn-type":[{"value":"2162-2531","type":"print"}],"subject":[],"published":{"date-parts":[[2026,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Development of an antisense oligonucleotide targeting EFEMP1 in models relevant to macular degeneration","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2026.103024","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Authors. Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"103024"}}