{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T15:14:06Z","timestamp":1788880446979,"version":"build-2803163510"},"reference-count":67,"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\/100020797","name":"NanoMedicines Innovation Network","doi-asserted-by":"publisher","award":["2019-T2-03"],"award-info":[{"award-number":["2019-T2-03"]}],"id":[{"id":"10.13039\/100020797","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.2026.103025","type":"journal-article","created":{"date-parts":[[2026,7,20]],"date-time":"2026-07-20T23:12:50Z","timestamp":1784589170000},"page":"103025","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":0,"title":["Design and development of lipid nanoparticle formulations for brain gene therapy"],"prefix":"10.1016","volume":"37","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-9407-1245","authenticated-orcid":false,"given":"Sarah B.","family":"Thomson","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alissandra","family":"de Moura Gomes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pardis","family":"Kazemian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel Z.","family":"Kurek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jayesh A.","family":"Kulkarni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fariba","family":"Sadaati","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pamela K.","family":"Wagner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terri L.","family":"Petkau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marco A.","family":"Ciufolini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pieter R.","family":"Cullis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4532-766X","authenticated-orcid":false,"given":"Blair R.","family":"Leavitt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtn.2026.103025_bib1","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1016\/S1474-4422(18)30499-X","article-title":"Global, regional, and national burden of neurological disorders, 1990-2016: a systematic analysis for the Global Burden of Disease Study 2016","volume":"18","author":"Feigin","year":"2019","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.omtn.2026.103025_bib2","doi-asserted-by":"crossref","first-page":"971","DOI":"10.1016\/0092-8674(93)90585-E","article-title":"A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes","volume":"72","author":"MacDonald","year":"1993","journal-title":"Cell"},{"key":"10.1016\/j.omtn.2026.103025_bib3","doi-asserted-by":"crossref","first-page":"170","DOI":"10.1016\/S1474-4422(19)30287-X","article-title":"The genetic architecture of Parkinson's disease","volume":"19","author":"Blauwendraat","year":"2020","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.omtn.2026.103025_bib4","doi-asserted-by":"crossref","first-page":"414","DOI":"10.1038\/s41588-019-0358-2","article-title":"Genetic meta-analysis of diagnosed Alzheimer's disease identifies new risk loci and implicates Abeta, tau, immunity and lipid processing","volume":"51","author":"Kunkle","year":"2019","journal-title":"Nat. Genet."},{"key":"10.1016\/j.omtn.2026.103025_bib5","doi-asserted-by":"crossref","first-page":"11926","DOI":"10.1073\/pnas.182272699","article-title":"RNAi functions in cultured mammalian neurons","volume":"99","author":"Krichevsky","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtn.2026.103025_bib6","doi-asserted-by":"crossref","first-page":"862","DOI":"10.1016\/S0021-9258(19)85022-6","article-title":"Effects of apolipoproteins A-IV and A-I on the uptake of phospholipid liposomes by hepatocytes","volume":"264","author":"Bisgaier","year":"1989","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.omtn.2026.103025_bib7","doi-asserted-by":"crossref","first-page":"1357","DOI":"10.1038\/mt.2010.85","article-title":"Targeted delivery of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms","volume":"18","author":"Akinc","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.103025_bib8","doi-asserted-by":"crossref","first-page":"555","DOI":"10.2217\/clp.10.37","article-title":"Apolipoproteins in the brain: implications for neurological and psychiatric disorders","volume":"51","author":"Elliott","year":"2010","journal-title":"Clin. Lipidol."},{"key":"10.1016\/j.omtn.2026.103025_bib9","doi-asserted-by":"crossref","first-page":"190","DOI":"10.1097\/MOL.0b013e32832d3a10","article-title":"Apolipoprotein E receptors in the nervous system","volume":"20","author":"Herz","year":"2009","journal-title":"Curr. Opin. Lipidol."},{"key":"10.1016\/j.omtn.2026.103025_bib10","doi-asserted-by":"crossref","first-page":"2060","DOI":"10.1021\/acs.molpharmaceut.7b01084","article-title":"In Vivo Introduction of mRNA Encapsulated in Lipid Nanoparticles to Brain Neuronal Cells and Astrocytes via Intracerebroventricular Administration","volume":"15","author":"Tanaka","year":"2018","journal-title":"Mol. Pharm."},{"key":"10.1016\/j.omtn.2026.103025_bib11","doi-asserted-by":"crossref","DOI":"10.1038\/mtna.2013.65","article-title":"Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain","volume":"2","author":"Rungta","year":"2013","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2026.103025_bib12","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1016\/j.trci.2015.06.007","article-title":"Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: A phase 1 clinical trial","volume":"1","author":"Kim","year":"2015","journal-title":"Alzheimer's Dement."},{"key":"10.1016\/j.omtn.2026.103025_bib13","doi-asserted-by":"crossref","first-page":"2427","DOI":"10.1056\/NEJMoa2028436","article-title":"Safety and Immunogenicity of SARS-CoV-2 mRNA-1273 Vaccine in Older Adults","volume":"383","author":"Anderson","year":"2020","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.omtn.2026.103025_bib14","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1038\/s41586-020-2814-7","article-title":"COVID-19 vaccine BNT162b1 elicits human antibody and T(H)1 T cell responses","volume":"586","author":"Sahin","year":"2020","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2026.103025_bib15","doi-asserted-by":"crossref","first-page":"8529","DOI":"10.1002\/anie.201203263","article-title":"Maximizing the potency of siRNA lipid nanoparticles for hepatic gene silencing in vivo","volume":"51","author":"Jayaraman","year":"2012","journal-title":"Angew. Chem. Int. Ed. Engl."},{"key":"10.1016\/j.omtn.2026.103025_bib16","doi-asserted-by":"crossref","first-page":"1377","DOI":"10.1016\/j.nano.2016.12.014","article-title":"Design of lipid nanoparticles for in vitro and in vivo delivery of plasmid DNA","volume":"13","author":"Kulkarni","year":"2017","journal-title":"Nanomedicine."},{"key":"10.1016\/j.omtn.2026.103025_bib17","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1016\/j.ijpharm.2015.05.043","article-title":"Effect of hydrophobic scaffold on the cellular uptake and gene transfection activities of DNA-encapsulating liposomal nanoparticles via intracerebroventricular administration","volume":"490","author":"Akita","year":"2015","journal-title":"Int. J. Pharm."},{"key":"10.1016\/j.omtn.2026.103025_bib18","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1016\/S0014-5793(97)00313-X","article-title":"'Green mice' as a source of ubiquitous green cells","volume":"407","author":"Okabe","year":"1997","journal-title":"FEBS Lett."},{"key":"10.1016\/j.omtn.2026.103025_bib19","doi-asserted-by":"crossref","first-page":"8698","DOI":"10.1021\/acs.jpcb.5b02891","article-title":"Microfluidic Mixing: A General Method for Encapsulating Macromolecules in Lipid Nanoparticle Systems","volume":"119","author":"Leung","year":"2015","journal-title":"J. Phys. Chem. B"},{"key":"10.1016\/j.omtn.2026.103025_bib20","doi-asserted-by":"crossref","first-page":"362","DOI":"10.1007\/s11095-004-1873-z","article-title":"A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA","volume":"22","author":"Jeffs","year":"2005","journal-title":"Pharm. Res."},{"key":"10.1016\/j.omtn.2026.103025_bib21","doi-asserted-by":"crossref","first-page":"23959","DOI":"10.1039\/D0NR06816K","article-title":"Spontaneous, solvent-free entrapment of siRNA within lipid nanoparticles","volume":"12","author":"Kulkarni","year":"2020","journal-title":"Nanoscale"},{"key":"10.1016\/j.omtn.2026.103025_bib22","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1016\/j.jconrel.2022.08.042","article-title":"LNP-mediated delivery of CRISPR RNP for wide-spread in vivo genome editing in mouse cornea","volume":"350","author":"Mirjalili Mohanna","year":"2022","journal-title":"J. Control. Release"},{"key":"10.1016\/j.omtn.2026.103025_bib23","doi-asserted-by":"crossref","first-page":"2049","DOI":"10.1007\/s40123-023-00729-6","article-title":"ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons","volume":"12","author":"Adair","year":"2023","journal-title":"Ophthalmol. Ther."},{"key":"10.1016\/j.omtn.2026.103025_bib24","doi-asserted-by":"crossref","DOI":"10.1002\/btm2.10213","article-title":"Cytosolic delivery of nucleic acids: The case of ionizable lipid nanoparticles","volume":"6","author":"Schlich","year":"2021","journal-title":"Bioeng. Transl. Med."},{"key":"10.1016\/j.omtn.2026.103025_bib25","doi-asserted-by":"crossref","first-page":"1084","DOI":"10.1038\/s41565-019-0591-y","article-title":"The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs","volume":"14","author":"Akinc","year":"2019","journal-title":"Nat. Nanotechnol."},{"key":"10.1016\/j.omtn.2026.103025_bib26","series-title":"Sulfur-containing lipids","author":"Ciufolini","year":"2022"},{"key":"10.1016\/j.omtn.2026.103025_bib27","doi-asserted-by":"crossref","first-page":"6171","DOI":"10.1523\/JNEUROSCI.0183-10.2010","article-title":"Transfection techniques for neuronal cells","volume":"30","author":"Karra","year":"2010","journal-title":"J. Neurosci."},{"key":"10.1016\/j.omtn.2026.103025_bib28","doi-asserted-by":"crossref","first-page":"7300","DOI":"10.1021\/acs.nanolett.5b02497","article-title":"Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs","volume":"15","author":"Kauffman","year":"2015","journal-title":"Nano Lett."},{"key":"10.1016\/j.omtn.2026.103025_bib29","doi-asserted-by":"crossref","first-page":"2148","DOI":"10.1021\/acs.nanolett.8b00432","article-title":"A Direct Comparison of in Vitro and in Vivo Nucleic Acid Delivery Mediated by Hundreds of Nanoparticles Reveals a Weak Correlation","volume":"18","author":"Paunovska","year":"2018","journal-title":"Nano Lett."},{"key":"10.1016\/j.omtn.2026.103025_bib30","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1097\/00005072-198511000-00003","article-title":"Neuropathological classification of Huntington's disease","volume":"44","author":"Vonsattel","year":"1985","journal-title":"J. Neuropathol. Exp. Neurol."},{"key":"10.1016\/j.omtn.2026.103025_bib31","doi-asserted-by":"crossref","DOI":"10.1016\/j.parkreldis.2023.105405","article-title":"GNAO1-related movement disorder: An update on phenomenology, clinical course, and response to treatments","volume":"111","author":"Novelli","year":"2023","journal-title":"Parkinsonism Relat. Disord."},{"key":"10.1016\/j.omtn.2026.103025_bib32","doi-asserted-by":"crossref","first-page":"1833","DOI":"10.1038\/mt.2008.200","article-title":"Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability","volume":"16","author":"Karik\u00f3","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtn.2026.103025_bib33","doi-asserted-by":"crossref","DOI":"10.1002\/smtd.201700375","article-title":"State-of-the-Art Design and Rapid-Mixing Production Techniques of Lipid Nanoparticles for Nucleic Acid Delivery","volume":"2","author":"Evers","year":"2018","journal-title":"Small Methods"},{"key":"10.1016\/j.omtn.2026.103025_bib34","doi-asserted-by":"crossref","first-page":"3609","DOI":"10.1039\/C9OB00473D","article-title":"Substitution of unsaturated lipid chains by thioether-containing lipid chains in cationic amphiphiles: physicochemical consequences and application for gene delivery","volume":"17","author":"Bouraoui","year":"2019","journal-title":"Org. Biomol. Chem."},{"key":"10.1016\/j.omtn.2026.103025_bib35","doi-asserted-by":"crossref","first-page":"1749","DOI":"10.1016\/j.addr.2012.05.009","article-title":"Cationic lipids activate intracellular signaling pathways","volume":"64","author":"Lonez","year":"2012","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.omtn.2026.103025_bib36","doi-asserted-by":"crossref","first-page":"2086","DOI":"10.3390\/pharmaceutics14102086","article-title":"Development of a Combined Lipid-Based Nanoparticle Formulation for Enhanced siRNA Delivery to Vascular Endothelial Cells","volume":"14","author":"He","year":"2022","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.omtn.2026.103025_bib37","doi-asserted-by":"crossref","first-page":"1755","DOI":"10.1007\/s13346-024-01709-4","article-title":"Oral delivery of stabilized lipid nanoparticles for nucleic acid therapeutics","volume":"15","author":"Suri","year":"2025","journal-title":"Drug Deliv. Transl. Res."},{"key":"10.1016\/j.omtn.2026.103025_bib38","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.jconrel.2006.04.014","article-title":"Toxicity of cationic lipids and cationic polymers in gene delivery","volume":"114","author":"Lv","year":"2006","journal-title":"J. Control. Release"},{"key":"10.1016\/j.omtn.2026.103025_bib39","doi-asserted-by":"crossref","first-page":"956","DOI":"10.1038\/s42003-021-02441-2","article-title":"Ionization and structural properties of mRNA lipid nanoparticles influence expression in intramuscular and intravascular administration","volume":"4","author":"Carrasco","year":"2021","journal-title":"Commun. Biol."},{"key":"10.1016\/j.omtn.2026.103025_bib40","doi-asserted-by":"crossref","first-page":"276","DOI":"10.1016\/j.jconrel.2005.06.014","article-title":"Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids","volume":"107","author":"Heyes","year":"2005","journal-title":"J. Control. Release"},{"key":"10.1016\/j.omtn.2026.103025_bib41","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1016\/0009-3084(86)90067-8","article-title":"Lipid polymorphism and the roles of lipids in membranes","volume":"40","author":"Cullis","year":"1986","journal-title":"Chem. Phys. Lipids"},{"key":"10.1016\/j.omtn.2026.103025_bib42","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1016\/0005-2736(95)80016-9","article-title":"The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer","volume":"1235","author":"Farhood","year":"1995","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.omtn.2026.103025_bib43","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1007\/s13346-013-0161-z","article-title":"Lipid nanoparticle delivery systems for siRNA-based therapeutics","volume":"4","author":"Wan","year":"2014","journal-title":"Drug Deliv. Transl. Res."},{"key":"10.1016\/j.omtn.2026.103025_bib44","doi-asserted-by":"crossref","first-page":"1188","DOI":"10.1038\/sj.gt.3301506","article-title":"On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids","volume":"8","author":"Hafez","year":"2001","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtn.2026.103025_bib45","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1016\/0304-4157(79)90012-1","article-title":"Lipid polymorphism and the functional roles of lipids in biological membranes","volume":"559","author":"Cullis","year":"1979","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.omtn.2026.103025_bib46","doi-asserted-by":"crossref","first-page":"9190","DOI":"10.1016\/j.biomaterials.2013.07.081","article-title":"Multifunctional receptor-targeted nanocomplexes for the delivery of therapeutic nucleic acids to the brain","volume":"34","author":"Kenny","year":"2013","journal-title":"Biomaterials"},{"key":"10.1016\/j.omtn.2026.103025_bib47","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1016\/j.jconrel.2013.11.014","article-title":"PEGylation improves the receptor-mediated transfection efficiency of peptide-targeted, self-assembling, anionic nanocomplexes","volume":"174","author":"Tagalakis","year":"2014","journal-title":"J. Control. Release"},{"key":"10.1016\/j.omtn.2026.103025_bib48","doi-asserted-by":"crossref","first-page":"8406","DOI":"10.1016\/j.biomaterials.2014.06.003","article-title":"Multifunctional, self-assembling anionic peptide-lipid nanocomplexes for targeted siRNA delivery","volume":"35","author":"Tagalakis","year":"2014","journal-title":"Biomaterials"},{"key":"10.1016\/j.omtn.2026.103025_bib49","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S0005-2736(01)00399-6","article-title":"Characterization of the inhibitory effect of PEG-lipid conjugates on the intracellular delivery of plasmid and antisense DNA mediated by cationic lipid liposomes","volume":"1558","author":"Song","year":"2002","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.omtn.2026.103025_bib50","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1038\/nrd3010","article-title":"Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application","volume":"9","author":"Jackson","year":"2010","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtn.2026.103025_bib51","doi-asserted-by":"crossref","first-page":"760","DOI":"10.1046\/j.1471-4159.1997.68020760.x","article-title":"Effect of apolipoprotein E on neurite outgrowth and beta-amyloid-induced toxicity in developing rat primary hippocampal cultures","volume":"68","author":"Puttfarcken","year":"1997","journal-title":"J. Neurochem."},{"key":"10.1016\/j.omtn.2026.103025_bib52","doi-asserted-by":"crossref","first-page":"96","DOI":"10.1016\/S0006-8993(01)03367-4","article-title":"Apolipoprotein E4 inhibits, and apolipoprotein E3 promotes neurite outgrowth in cultured adult mouse cortical neurons through the low-density lipoprotein receptor-related protein","volume":"928","author":"Nathan","year":"2002","journal-title":"Brain Res."},{"key":"10.1016\/j.omtn.2026.103025_bib53","doi-asserted-by":"crossref","first-page":"2278","DOI":"10.1046\/j.1471-4159.1999.0722278.x","article-title":"Inhibition of cholesterol production but not of nonsterol isoprenoid products induces neuronal cell death","volume":"72","author":"Michikawa","year":"1999","journal-title":"J. Neurochem."},{"key":"10.1016\/j.omtn.2026.103025_bib54","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.neuron.2011.09.027","article-title":"How inhibition shapes cortical activity","volume":"72","author":"Isaacson","year":"2011","journal-title":"Neuron"},{"key":"10.1016\/j.omtn.2026.103025_bib55","doi-asserted-by":"crossref","first-page":"150","DOI":"10.3389\/fnana.2010.00150","article-title":"Heterogeneity and diversity of striatal GABAergic interneurons","volume":"4","author":"Tepper","year":"2010","journal-title":"Front. Neuroanat."},{"key":"10.1016\/j.omtn.2026.103025_bib56","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1038\/nature13992","article-title":"A comparative encyclopedia of DNA elements in the mouse genome","volume":"515","author":"Yue","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.omtn.2026.103025_bib57","doi-asserted-by":"crossref","first-page":"344","DOI":"10.1016\/j.addr.2020.06.026","article-title":"Lipid nanoparticle technology for therapeutic gene regulation in the liver","volume":"159","author":"Witzigmann","year":"2020","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.omtn.2026.103025_bib58","doi-asserted-by":"crossref","first-page":"2550","DOI":"10.1074\/mcp.M114.041533","article-title":"Label-free Quantitative Proteomics of Mouse Cerebrospinal Fluid Detects beta-Site APP Cleaving Enzyme (BACE1) Protease Substrates In Vivo","volume":"14","author":"Dislich","year":"2015","journal-title":"Mol. Cell. Proteomics"},{"key":"10.1016\/j.omtn.2026.103025_bib59","article-title":"Imaging and recording subventricular zone progenitor cells in live tissue of postnatal mice","volume":"4","author":"Lacar","year":"2010","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.omtn.2026.103025_bib60","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1038\/nn.2536","article-title":"Chordin-induced lineage plasticity of adult SVZ neuroblasts after demyelination","volume":"13","author":"Jablonska","year":"2010","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.omtn.2026.103025_bib61","first-page":"468","article-title":"Roles of Ependymal Cells in the Physiology and Pathology of the Central Nervous System","volume":"14","author":"Deng","year":"2023","journal-title":"Aging Dis."},{"key":"10.1016\/j.omtn.2026.103025_bib62","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102380","article-title":"A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy","volume":"35","author":"Sawamura","year":"2024","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.omtn.2026.103025_bib63","doi-asserted-by":"crossref","first-page":"561","DOI":"10.1038\/nbt1402","article-title":"A combinatorial library of lipid-like materials for delivery of RNAi therapeutics","volume":"26","author":"Akinc","year":"2008","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtn.2026.103025_bib64","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1016\/j.expneurol.2003.12.013","article-title":"Improving lipoplex-mediated gene transfer into C6 glioma cells and primary neurons","volume":"187","author":"da Cruz","year":"2004","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.omtn.2026.103025_bib65","doi-asserted-by":"crossref","first-page":"4787","DOI":"10.1021\/acsnano.8b01516","article-title":"On the Formation and Morphology of Lipid Nanoparticles Containing Ionizable Cationic Lipids and siRNA","volume":"12","author":"Kulkarni","year":"2018","journal-title":"ACS Nano"},{"key":"10.1016\/j.omtn.2026.103025_bib66","doi-asserted-by":"crossref","DOI":"10.1186\/gb-2002-3-7-research0034","article-title":"Accurate normalization of real-time quantitative RT-PCR data by geometric averaging of multiple internal control genes","volume":"3","author":"Vandesompele","year":"2002","journal-title":"Genome Biol."},{"key":"10.1016\/j.omtn.2026.103025_bib67","series-title":"Paxinos and Franklin's the Mouse Brain in Stereotaxic Coordinates","author":"Paxinos","year":"2019"}],"container-title":["Molecular Therapy Nucleic Acids"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126001964?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2162253126001964?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,9,8]],"date-time":"2026-09-08T15:03:39Z","timestamp":1788879819000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2162253126001964"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,9]]},"references-count":67,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2026,9]]}},"alternative-id":["S2162253126001964"],"URL":"https:\/\/doi.org\/10.1016\/j.omtn.2026.103025","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":"Design and development of lipid nanoparticle formulations for brain gene therapy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Nucleic Acids","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtn.2026.103025","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 The Author(s). Published by Elsevier Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"103025"}}