{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,21]],"date-time":"2026-09-21T17:26:37Z","timestamp":1790011597602,"version":"4.0.1"},"reference-count":226,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,12,1]],"date-time":"2023-12-01T00:00:00Z","timestamp":1701388800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2023,10,30]],"date-time":"2023-10-30T00:00:00Z","timestamp":1698624000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Advanced Drug Delivery Reviews"],"published-print":{"date-parts":[[2023,12]]},"DOI":"10.1016\/j.addr.2023.115132","type":"journal-article","created":{"date-parts":[[2023,10,31]],"date-time":"2023-10-31T04:50:29Z","timestamp":1698727829000},"page":"115132","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":15,"special_numbering":"C","title":["Nanomedicines to treat rare neurological disorders: The case of Krabbe disease"],"prefix":"10.1016","volume":"203","author":[{"given":"Thomas Lee","family":"Moore","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanna","family":"Pannuzzo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gabriella","family":"Costabile","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anna Lisa","family":"Palange","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Raffaele","family":"Span\u00f2","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Miguel","family":"Ferreira","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Adriana Carol Eleonora","family":"Graziano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paolo","family":"Decuzzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Venera","family":"Cardile","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.addr.2023.115132_b0005","doi-asserted-by":"crossref","unstructured":"Nanomedicine and the COVID-19 vaccines, Nature Nanotechnology. 15 (2020) 963. https:\/\/doi.org\/10.1038\/s41565-020-00820-0.","DOI":"10.1038\/s41565-020-00820-0"},{"key":"10.1016\/j.addr.2023.115132_b0010","doi-asserted-by":"crossref","DOI":"10.1002\/btm2.10246","article-title":"Nanoparticles in the clinic: An update post COVID-19 vaccines","volume":"6","author":"Anselmo","year":"2021","journal-title":"Bioeng. Transl. Med."},{"key":"10.1016\/j.addr.2023.115132_b0015","doi-asserted-by":"crossref","first-page":"189","DOI":"10.1002\/wnan.73","article-title":"New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders","volume":"2","author":"Muro","year":"2010","journal-title":"Wiley Interdiscip. Rev. Nanomed. Nanobiotechnol."},{"key":"10.1016\/j.addr.2023.115132_b0020","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1016\/j.jconrel.2016.05.044","article-title":"Nanoparticle-mediated brain drug delivery: Overcoming blood-brain barrier to treat neurodegenerative diseases","volume":"235","author":"Saraiva","year":"2016","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0025","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1016\/j.jconrel.2017.07.028","article-title":"Strategies to enhance the distribution of nanotherapeutics in the brain","volume":"267","author":"Zhang","year":"2017","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0030","doi-asserted-by":"crossref","first-page":"1801362","DOI":"10.1002\/adma.201801362","article-title":"Overcoming the Blood-Brain Barrier: The Role of Nanomaterials in Treating Neurological Diseases","volume":"30","author":"Furtado","year":"2018","journal-title":"Adv. Mater."},{"key":"10.1016\/j.addr.2023.115132_b0035","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1016\/j.jconrel.2017.12.015","article-title":"Crossing the blood-brain barrier with nanoparticles","volume":"270","author":"Zhou","year":"2018","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0040","first-page":"1","article-title":"Delivery of Nanoparticles and Macromolecules Across the Blood Brain Barrier","volume":"1900073","author":"Nowak","year":"2020","journal-title":"Advanced Therapeutics."},{"key":"10.1016\/j.addr.2023.115132_b0045","doi-asserted-by":"crossref","first-page":"2967","DOI":"10.1039\/C8CS00805A","article-title":"Emerging blood-brain-barrier-crossing nanotechnology for brain cancer theranostics","volume":"48","author":"Tang","year":"2019","journal-title":"Chem. Soc. Rev."},{"key":"10.1016\/j.addr.2023.115132_b0050","doi-asserted-by":"crossref","first-page":"595","DOI":"10.1177\/08830738030180090201","article-title":"Globoid cell leukodystrophy (Krabbe\u2019s disease): Update","volume":"18","author":"Suzuki","year":"2003","journal-title":"J. Child Neurol."},{"key":"10.1016\/j.addr.2023.115132_b0055","doi-asserted-by":"crossref","first-page":"479","DOI":"10.1111\/j.1471-4159.1974.tb06882.x","article-title":"The biosynthesis and concentration of galactosyl diglyceride in glial and neuronal enriched fractions of actively myelinating rat brain","volume":"22","author":"Deshmukh","year":"1974","journal-title":"J. Neurochem."},{"key":"10.1016\/j.addr.2023.115132_b0060","doi-asserted-by":"crossref","first-page":"588","DOI":"10.1002\/jnr.10247","article-title":"Establishment and characterization of spontaneously immortalized schwann cells from murine model of globoid cell leukodystrophy (twitcher)","volume":"68","author":"Shen","year":"2002","journal-title":"J. Neurosci. Res."},{"issue":"Suppl 1","key":"10.1016\/j.addr.2023.115132_b0065","first-page":"689","article-title":"Insights into the pathogenesis and treatment of Krabbe disease","volume":"13","author":"Bongarzone","year":"2016","journal-title":"Pediatric Endocrinology Reviews: PER."},{"key":"10.1016\/j.addr.2023.115132_b0070","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/0165-3806(86)90009-X","article-title":"The twitcher mouse: Degeneration of oligodendrocytes in vitro","volume":"391","author":"Nagara","year":"1986","journal-title":"Brain Res."},{"key":"10.1016\/j.addr.2023.115132_b0075","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1126\/science.6719111","article-title":"Progressive accumulation of toxic metabolite in a genetic leukodystrophy","volume":"224","author":"Igisu","year":"1984","journal-title":"Science (New York, N.Y.)"},{"key":"10.1016\/j.addr.2023.115132_b0080","doi-asserted-by":"crossref","first-page":"874","DOI":"10.1016\/S0006-3495(00)76645-8","article-title":"Trans interactions between galactosylceramide and cerebroside sulfate across apposed bilayers","volume":"78","author":"Boggs","year":"2000","journal-title":"Biophys. J ."},{"key":"10.1016\/j.addr.2023.115132_b0085","doi-asserted-by":"crossref","first-page":"194","DOI":"10.1016\/j.bbamem.2008.11.010","article-title":"How the molecular features of glycosphingolipids affect domain formation in fluid membranes","volume":"1788","author":"Westerlund","year":"2009","journal-title":"BBA"},{"key":"10.1016\/j.addr.2023.115132_b0090","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.plipres.2015.12.004","article-title":"Recent progress on lipid lateral heterogeneity in plasma membranes: From rafts to submicrometric domains","volume":"62","author":"Carquin","year":"2016","journal-title":"Prog. Lipid Res."},{"key":"10.1016\/j.addr.2023.115132_b0095","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1023\/A:1022436928925","article-title":"Twenty five years of the \u201cpsychosine hypothesis\u201d: A personal perspective of its history and present status","volume":"23","author":"Suzuki","year":"1998","journal-title":"Neurochem. Res."},{"key":"10.1016\/j.addr.2023.115132_b0100","doi-asserted-by":"crossref","first-page":"3303","DOI":"10.1194\/jlr.M039610","article-title":"Psychosine, the cytotoxic sphingolipid that accumulates in globoid cell leukodystrophy, alters membrane architecture","volume":"54","author":"Hawkins-Salsbury","year":"2013","journal-title":"J. Lipid Res."},{"key":"10.1016\/j.addr.2023.115132_b0105","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1016\/S0304-3940(02)00655-9","article-title":"Apoptotic positive cells in Krabbe brain and induction of apoptosis in rat C6 glial cells by psychosine","volume":"330","author":"Jatana","year":"2002","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.addr.2023.115132_b0110","doi-asserted-by":"crossref","first-page":"1571","DOI":"10.1023\/B:NERE.0000029571.39497.56","article-title":"NF- Prevents TNF-\u2013Induced Apoptosis in an Oligodendrocyte Cell Line","volume":"29","author":"Hamanoue","year":"2004","journal-title":"Neurochem. Res."},{"key":"10.1016\/j.addr.2023.115132_b0115","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1016\/j.ymgme.2010.03.021","article-title":"A galactose-free diet enriched in soy isoflavones and antioxidants results in delayed onset of symptoms of Krabbe disease in twitcher mice","volume":"100","author":"Pannuzzo","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.addr.2023.115132_b0120","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1002\/glia.20249","article-title":"TRAIL-induced death of human adult oligodendrocytes is mediated by JNK pathway","volume":"53","author":"Jurewicz","year":"2006","journal-title":"Glia"},{"key":"10.1016\/j.addr.2023.115132_b0125","doi-asserted-by":"crossref","first-page":"758","DOI":"10.1002\/jnr.22256","article-title":"Astrocytes protect oligodendrocyte precursor cells via MEK\/ERK and PI3K\/Akt signaling","volume":"88","author":"Arai","year":"2010","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.addr.2023.115132_b0130","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1016\/j.brainres.2013.02.024","article-title":"Role of endogenous psychosine accumulation in oligodendrocyte differentiation and survival: Implication for Krabbe disease","volume":"1508","author":"Won","year":"2013","journal-title":"Brain Res."},{"key":"10.1016\/j.addr.2023.115132_b0135","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1007\/s10495-015-1183-4","article-title":"Krabbe disease: Involvement of connexin43 in the apoptotic effects of sphingolipid psychosine on mouse oligodendrocyte precursors","volume":"21","author":"Graziano","year":"2016","journal-title":"Apoptosis: an International Journal on Programmed Cell Death."},{"key":"10.1016\/j.addr.2023.115132_b0140","doi-asserted-by":"crossref","first-page":"3878","DOI":"10.1242\/jcs.169342","article-title":"Galactosylsphingosine (psychosine)-induced demyelination is attenuated by sphingosine 1-phosphate signalling","volume":"128","author":"O\u2019Sullivan","year":"2015","journal-title":"J. Cell Sci."},{"key":"10.1016\/j.addr.2023.115132_b0145","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1093\/brain\/39.1-2.74","article-title":"A New Familial, Infantile Form of Diffuse Brain-Sclerosis","volume":"39","author":"Krabbe","year":"1916","journal-title":"Brain"},{"key":"10.1016\/j.addr.2023.115132_b0150","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1016\/S0387-7604(02)00057-8","article-title":"Immunohistological study of globoid cell leukodystrophy","volume":"24","author":"Itoh","year":"2002","journal-title":"Brain & Development."},{"key":"10.1016\/j.addr.2023.115132_b0155","unstructured":"D. Wenger, K. Suzuki, Y. Suzuki, K. Suzuki, Galactosyl-ceramide lipidosis: Globoid cell leukodystrophy (Krabbe disease), in: The Metabolic and Molecular Bases of Inherited Disease, McGraw-Hill, New York, NY, 2001: pp. 2669\u20133694."},{"key":"10.1016\/j.addr.2023.115132_b0160","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1016\/j.gene.2014.09.046","article-title":"History, genetic, and recent advances on Krabbe disease","volume":"555","author":"Graziano","year":"2015","journal-title":"Gene"},{"key":"10.1016\/j.addr.2023.115132_b0165","doi-asserted-by":"crossref","first-page":"587","DOI":"10.1023\/A:1005315311165","article-title":"Prevalent mutations in the GALC gene of patients with Krabbe disease of Dutch and other European origin","volume":"20","author":"Kleijer","year":"1997","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.addr.2023.115132_b0170","doi-asserted-by":"crossref","first-page":"201","DOI":"10.1212\/01.wnl.0000231531.73713.a9","article-title":"Peripheral nerve involvement in Krabbe disease: A guide to therapy selection and evaluation","volume":"67","author":"Moser","year":"2006","journal-title":"Neurology"},{"key":"10.1016\/j.addr.2023.115132_b0175","doi-asserted-by":"crossref","first-page":"1235","DOI":"10.1038\/gim.2016.35","article-title":"Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York State","volume":"18","author":"Wasserstein","year":"2016","journal-title":"Genet. Med."},{"key":"10.1016\/j.addr.2023.115132_b0180","first-page":"239","article-title":"New York State Krabbe Disease Consortium, Newborn screening for Krabbe disease in New York State: The first eight years\u2019 experience, Genetics in Medicine: Official Journal of the American College of Medical","volume":"18","author":"Orsini","year":"2016","journal-title":"Genetics"},{"key":"10.1016\/j.addr.2023.115132_b0185","doi-asserted-by":"crossref","first-page":"2835","DOI":"10.1002\/ajmg.a.35624","article-title":"Death rates in the u.s. Due to krabbe disease and related leukodystrophy and lysosomal storage diseases","volume":"158A","author":"Barczykowski","year":"2012","journal-title":"Am. J. Med. Genet. A"},{"key":"10.1016\/j.addr.2023.115132_b0190","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1002\/ajmg.1320210420","article-title":"Krabbe disease: Increased incidence in a highly inbred community","volume":"21","author":"Zlotogora","year":"1985","journal-title":"Am. J. Med. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0195","doi-asserted-by":"crossref","first-page":"359","DOI":"10.1111\/ane.13153","article-title":"Screening for krabbe disease: The first 2 years\u2019 experience","volume":"140","author":"Pannuzzo","year":"2019","journal-title":"Acta Neurol. Scand."},{"key":"10.1016\/j.addr.2023.115132_b0200","first-page":"244","article-title":"Regional mapping of the human galactocerebrosidase gene (GALC) to 14q31 by in situ hybridization, Cytogenetics and Cell","volume":"66","author":"Cannizzaro","year":"1994","journal-title":"Genetics"},{"key":"10.1016\/j.addr.2023.115132_b0205","doi-asserted-by":"crossref","first-page":"2335","DOI":"10.1093\/hmg\/4.12.2335","article-title":"Characterization of the large deletion in the GALC gene found in patients with krabbe disease","volume":"4","author":"Luzi","year":"1995","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0210","doi-asserted-by":"crossref","first-page":"15169","DOI":"10.1073\/pnas.1105639108","article-title":"Insights into Krabbe disease from structures of galactocerebrosidase","volume":"108","author":"Deane","year":"2011","journal-title":"Proc. Natl. Acad. Sci."},{"key":"10.1016\/j.addr.2023.115132_b0215","first-page":"1233","article-title":"Molecular heterogeneity of late-onset forms of globoid-cell leukodystrophy","volume":"59","author":"De Gasperi","year":"1996","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0220","doi-asserted-by":"crossref","first-page":"548","DOI":"10.1007\/s10038-006-0396-3","article-title":"Six novel mutations detected in the GALC gene in 17 Japanese patients with Krabbe disease, and new genotype\u2013phenotype correlation","volume":"51","author":"Xu","year":"2006","journal-title":"J. Hum. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0225","doi-asserted-by":"crossref","first-page":"742","DOI":"10.1002\/humu.9500","article-title":"A single mutation in the GALC gene is responsible for the majority of late onset Krabbe disease patients in the Catania (Sicily, Italy) region","volume":"28","author":"Lissens","year":"2007","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.addr.2023.115132_b0230","doi-asserted-by":"crossref","first-page":"E1894","DOI":"10.1002\/humu.21367","article-title":"Identification and characterization of 15 novel GALC gene mutations causing Krabbe disease","volume":"31","author":"Tappino","year":"2010","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.addr.2023.115132_b0235","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1111\/cge.13071","article-title":"Large-scale study of clinical and biochemical characteristics of Chinese patients diagnosed with Krabbe disease","volume":"93","author":"Zhao","year":"2018","journal-title":"Clin. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0240","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1016\/j.ymgme.2013.12.009","article-title":"Krabbe disease: Are certain mutations disease-causing only when specific polymorphisms are present or when inherited in trans with specific second mutations?","volume":"111","author":"Wenger","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.addr.2023.115132_b0245","doi-asserted-by":"crossref","unstructured":"Y.-H. Shao, K. Choquet, R. La Piana, M. T\u00e9treault, M.-J. Dicaire, Care4Rare Canada Consortium, K.M. Boycott, J. Majewski, B. Brais, Mutations in GALC cause late-onset Krabbe disease with predominant cerebellar ataxia, Neurogenetics. 17 (2016) 137\u2013141. https:\/\/doi.org\/10.1007\/s10048-016-0476-2.","DOI":"10.1007\/s10048-016-0476-2"},{"key":"10.1016\/j.addr.2023.115132_b0250","series-title":"The Online Metabolic and Molecular Bases of Inherited Disease","article-title":"Krabbe disease (Globoid Cell Leukodystrophy)","author":"Wenger","year":"2019"},{"key":"10.1016\/j.addr.2023.115132_b0255","doi-asserted-by":"crossref","first-page":"172","DOI":"10.1016\/j.ymgme.2013.09.011","article-title":"Characterization and application of a disease-cell model for a neurodegenerative lysosomal disease","volume":"111","author":"Ribbens","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.addr.2023.115132_b0260","doi-asserted-by":"crossref","first-page":"908","DOI":"10.1111\/tra.12404","article-title":"Molecular Mechanisms of Disease Pathogenesis Differ in Krabbe Disease Variants","volume":"17","author":"Spratley","year":"2016","journal-title":"Traffic (copenhagen, Denmark)."},{"key":"10.1016\/j.addr.2023.115132_b0265","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1373\/clinchem.2008.115873","article-title":"Specific determination of beta-galactocerebrosidase activity via competitive inhibition of beta-galactosidase","volume":"55","author":"Martino","year":"2009","journal-title":"Clin. Chem."},{"key":"10.1016\/j.addr.2023.115132_b0270","doi-asserted-by":"crossref","first-page":"5489","DOI":"10.1523\/JNEUROSCI.6383-09.2010","article-title":"Molecular characterization of mutations that cause globoid cell leukodystrophy and pharmacological rescue using small molecule chemical chaperones, The Journal of Neuroscience: The Official Journal of the Society for","volume":"30","author":"Lee","year":"2010","journal-title":"Neuroscience"},{"key":"10.1016\/j.addr.2023.115132_b0275","doi-asserted-by":"crossref","first-page":"1858","DOI":"10.1523\/JNEUROSCI.3095-15.2016","article-title":"Altered Trafficking and Processing of GALC Mutants Correlates with Globoid Cell Leukodystrophy Severity, The Journal of Neuroscience: The Official Journal of the Society for","volume":"36","author":"Shin","year":"2016","journal-title":"Neuroscience"},{"key":"10.1016\/j.addr.2023.115132_b0280","doi-asserted-by":"crossref","first-page":"1284","DOI":"10.1002\/jnr.23846","article-title":"Krabbe\u2019s leukodystrophy: Approaches and models in vitro","volume":"94","author":"Avola","year":"2016","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.addr.2023.115132_b0285","doi-asserted-by":"crossref","first-page":"765","DOI":"10.1002\/glia.22471","article-title":"MMP-3 mediates psychosine-induced globoid cell formation: Implications for leukodystrophy pathology","volume":"61","author":"Ijichi","year":"2013","journal-title":"Glia"},{"key":"10.1016\/j.addr.2023.115132_b0290","first-page":"e51903","article-title":"An in vitro model for the study of cellular pathophysiology in globoid cell leukodystrophy","author":"Claycomb","year":"2014","journal-title":"Journal of Visualized Experiments: Jove."},{"key":"10.1016\/j.addr.2023.115132_b0295","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1038\/nrd.2016.245","article-title":"Induced pluripotent stem cell technology: A decade of progress","volume":"16","author":"Shi","year":"2017","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.addr.2023.115132_b0300","doi-asserted-by":"crossref","first-page":"1478","DOI":"10.1016\/j.stemcr.2021.04.011","article-title":"Human iPSC-based neurodevelopmental models of globoid cell leukodystrophy uncover patient- and cell type-specific disease phenotypes","volume":"16","author":"Mangiameli","year":"2021","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.addr.2023.115132_b0305","doi-asserted-by":"crossref","first-page":"874","DOI":"10.1016\/j.ymthe.2018.01.009","article-title":"others, Long-term improvement of neurological signs and metabolic dysfunction in a mouse model of krabbe\u2019s disease after global gene therapy","volume":"26","author":"Marshall","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2023.115132_b0310","doi-asserted-by":"crossref","first-page":"98","DOI":"10.2353\/ajpath.2008.070404","article-title":"Clinical and immunopathologic alterations in rhesus macaques affected with globoid cell leukodystrophy","volume":"172","author":"Borda","year":"2008","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.addr.2023.115132_b0315","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1006\/geno.1996.0220","article-title":"Cloning of the canine GALC cDNA and identification of the mutation causing globoid cell leukodystrophy in West Highland White and Cairn terriers","volume":"33","author":"Victoria","year":"1996","journal-title":"Genomics"},{"key":"10.1016\/j.addr.2023.115132_b0320","doi-asserted-by":"crossref","first-page":"319","DOI":"10.1006\/geno.1997.4744","article-title":"Characterization of the rhesus monkey galactocerebrosidase (GALC) cDNA and gene and identification of the mutation causing globoid cell leukodystrophy (Krabbe disease) in this primate","volume":"42","author":"Luzi","year":"1997","journal-title":"Genomics"},{"key":"10.1016\/j.addr.2023.115132_b0325","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1093\/brain\/103.3.695","article-title":"Hereditary Leucodystrophy in the Mouse: The New Mutant Twitcher","volume":"103","author":"Duchen","year":"1980","journal-title":"Brain"},{"key":"10.1016\/j.addr.2023.115132_b0330","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1111\/j.1750-3639.1995.tb00601.x","article-title":"The Twitcher Mouse: A Model for Krabbe Disease and for Experimental Therapies","volume":"5","author":"Suzuki","year":"1995","journal-title":"Brain Pathol."},{"key":"10.1016\/j.addr.2023.115132_b0335","doi-asserted-by":"crossref","first-page":"950","DOI":"10.1177\/0192623321991469","article-title":"Quantitative assessment of neuroinflammation, myelinogenesis, demyelination, and nerve fiber regeneration in immunostained sciatic nerves from twitcher mice with a tissue image analysis platform","volume":"49","author":"Wilson","year":"2021","journal-title":"Toxicol. Pathol."},{"key":"10.1016\/j.addr.2023.115132_b0340","doi-asserted-by":"crossref","unstructured":"A.M. Yeager, S. Brennan, C. Tiffany, H.W. Moser, G.W. Santos, Prolonged survival and remyelination after hematopoietic cell transplantation in the twitcher mouse, Science (New York, N.Y.). 225 (1984) 1052\u20131054. https:\/\/doi.org\/10.1126\/science.6382609.","DOI":"10.1126\/science.6382609"},{"key":"10.1016\/j.addr.2023.115132_b0345","doi-asserted-by":"crossref","unstructured":"P.M. Hoogerbrugge, K. Suzuki, K. Suzuki, B.J. Poorthuis, T. Kobayashi, G. Wagemaker, D.W. van Bekkum, Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation, Science (New York, N.Y.). 239 (1988) 1035\u20131038. https:\/\/doi.org\/10.1126\/science.3278379.","DOI":"10.1126\/science.3278379"},{"key":"10.1016\/j.addr.2023.115132_b0350","doi-asserted-by":"crossref","first-page":"845","DOI":"10.1016\/S0041-1345(96)00163-7","article-title":"Widespread engraftment of neural progenitor and stem-like cells throughout the mouse brain","volume":"29","author":"Taylor","year":"1997","journal-title":"Transpl. Proc."},{"key":"10.1016\/j.addr.2023.115132_b0355","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.ymgme.2009.01.005","article-title":"Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells","volume":"97","author":"Strazza","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.addr.2023.115132_b0360","doi-asserted-by":"crossref","unstructured":"M. Neri, A. Ricca, I. di Girolamo, B. Alcala\u2019-Franco, C. Cavazzin, A. Orlacchio, S. Martino, L. Naldini, A. Gritti, Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy, Stem Cells (Dayton, Ohio). 29 (2011) 1559\u20131571. https:\/\/doi.org\/.","DOI":"10.1002\/stem.701"},{"key":"10.1016\/j.addr.2023.115132_b0365","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1002\/stem.555","article-title":"Mesenchymal lineage stem cells have pronounced anti-inflammatory effects in the twitcher mouse model of Krabbe\u2019s disease","volume":"29","author":"Ripoll","year":"2011","journal-title":"Stem Cells (dayton, Ohio)."},{"key":"10.1016\/j.addr.2023.115132_b0370","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1002\/(SICI)1097-4547(20000415)60:2<231::AID-JNR12>3.0.CO;2-E","article-title":"L-cycloserine slows the clinical and pathological course in mice with globoid cell leukodystrophy (twitcher mice)","volume":"60","author":"LeVine","year":"2000","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.addr.2023.115132_b0375","doi-asserted-by":"crossref","first-page":"294","DOI":"10.1016\/j.ymgme.2014.05.009","article-title":"Pharmacological chaperones increase residual -galactocerebrosidase activity in fibroblasts from Krabbe patients","volume":"112","author":"Berardi","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.addr.2023.115132_b0380","doi-asserted-by":"crossref","first-page":"1549","DOI":"10.1096\/fj.05-3826fje","article-title":"Enzyme replacement therapy results in substantial improvements in early clinical phenotype in a mouse model of globoid cell leukodystrophy","volume":"19","author":"Lee","year":"2005","journal-title":"FASEB Journal: Official Publication of the Federation of American Societies for Experimental Biology."},{"key":"10.1016\/j.addr.2023.115132_b0385","doi-asserted-by":"crossref","first-page":"2520","DOI":"10.1096\/fj.06-6169com","article-title":"Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy","volume":"21","author":"Lee","year":"2007","journal-title":"FASEB Journal: Official Publication of the Federation of American Societies for Experimental Biology."},{"key":"10.1016\/j.addr.2023.115132_b0390","first-page":"422","article-title":"AAV2\/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2, Molecular Therapy: The Journal of the American Society of","volume":"12","author":"Lin","year":"2005","journal-title":"Gene Ther."},{"key":"10.1016\/j.addr.2023.115132_b0395","first-page":"734","article-title":"AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy, Molecular Therapy: The Journal of the American Society of","volume":"11","author":"Rafi","year":"2005","journal-title":"Gene Ther."},{"key":"10.1016\/j.addr.2023.115132_b0400","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1016\/j.ymgme.2014.12.300","article-title":"Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features","volume":"114","author":"Rafi","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.addr.2023.115132_b0405","first-page":"1681","article-title":"Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice, Molecular Therapy: The Journal of the American Society of","volume":"23","author":"Rafi","year":"2015","journal-title":"Gene Ther."},{"key":"10.1016\/j.addr.2023.115132_b0410","doi-asserted-by":"crossref","first-page":"1081","DOI":"10.1038\/sj.gt.3301495","article-title":"Intraventricular administration of recombinant adenovirus to neonatal twitcher mouse leads to clinicopathological improvements","volume":"8","author":"Shen","year":"2001","journal-title":"Gene Ther."},{"key":"10.1016\/j.addr.2023.115132_b0415","first-page":"44","article-title":"Central nervous system-directed AAV2\/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy, Molecular Therapy: The Journal of the American Society of","volume":"15","author":"Lin","year":"2007","journal-title":"Gene Ther."},{"key":"10.1016\/j.addr.2023.115132_b0420","doi-asserted-by":"crossref","first-page":"1748","DOI":"10.1002\/jnr.22006","article-title":"Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease","volume":"87","author":"Galbiati","year":"2009","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.addr.2023.115132_b0425","doi-asserted-by":"crossref","first-page":"9945","DOI":"10.1523\/JNEUROSCI.1802-11.2011","volume":"31","author":"Reddy","year":"2011","journal-title":"Neuroscience"},{"key":"10.1016\/j.addr.2023.115132_b0430","doi-asserted-by":"crossref","first-page":"186","DOI":"10.1016\/j.ymgme.2012.05.021","article-title":"Bone marrow transplantation increases efficacy of central nervous system-directed enzyme replacement therapy in the murine model of globoid cell leukodystrophy","volume":"107","author":"Qin","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.addr.2023.115132_b0435","doi-asserted-by":"crossref","first-page":"6495","DOI":"10.1523\/JNEUROSCI.4199-14.2015","article-title":"Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy, The Journal of Neuroscience: The Official Journal of the Society for","volume":"35","author":"Hawkins-Salsbury","year":"2015","journal-title":"Neuroscience"},{"key":"10.1016\/j.addr.2023.115132_b0440","doi-asserted-by":"crossref","first-page":"3372","DOI":"10.1093\/hmg\/ddv086","article-title":"Combined gene\/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy","volume":"24","author":"Ricca","year":"2015","journal-title":"Human Molecular Genetics."},{"key":"10.1016\/j.addr.2023.115132_b0445","doi-asserted-by":"crossref","first-page":"R54","DOI":"10.1093\/hmg\/ddr112","article-title":"Combination therapies for lysosomal storage disease: Is the whole greater than the sum of its parts?","volume":"20","author":"Hawkins-Salsbury","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0450","first-page":"111","article-title":"Recent gene therapy advancements for neurological diseases","volume":"15","author":"Nagabhushan Kalburgi","year":"2013","journal-title":"Discov. Med."},{"key":"10.1016\/j.addr.2023.115132_b0455","first-page":"3041","article-title":"Reduced-intensity single-unit unrelated cord blood transplant with optional immune boost for nonmalignant disorders, Blood","volume":"4","author":"Vander Lugt","year":"2020","journal-title":"Advances"},{"key":"10.1016\/j.addr.2023.115132_b0460","doi-asserted-by":"crossref","first-page":"1971","DOI":"10.1182\/blood-2011-01-329235","article-title":"Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: the largest single-institution cohort report","volume":"118","author":"Miller","year":"2011","journal-title":"Blood"},{"key":"10.1016\/j.addr.2023.115132_b0465","doi-asserted-by":"crossref","first-page":"298","DOI":"10.1016\/j.pediatrneurol.2012.02.023","article-title":"Later onset phenotypes of Krabbe disease: Results of the world-wide registry","volume":"46","author":"Duffner","year":"2012","journal-title":"Pediatr. Neurol."},{"key":"10.1016\/j.addr.2023.115132_b0470","doi-asserted-by":"crossref","first-page":"1481","DOI":"10.1016\/S0140-6736(00)02161-9","article-title":"Novel oral treatment of Gaucher\u2019s disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis","volume":"355","author":"Cox","year":"2000","journal-title":"Lancet (london, England)."},{"key":"10.1016\/j.addr.2023.115132_b0475","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1023\/A:1025902113005","article-title":"Advisory Council to the European Working Group on Gaucher Disease, The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: A position statement","volume":"26","author":"Cox","year":"2003","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.addr.2023.115132_b0480","doi-asserted-by":"crossref","first-page":"766","DOI":"10.1006\/bbrc.1996.0136","article-title":"Correction of the galactocerebrosidase deficiency in globoid cell leukodystrophy-cultured cells by SL3-3 retroviral-mediated gene transfer","volume":"218","author":"Sosa","year":"1996","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.addr.2023.115132_b0485","doi-asserted-by":"crossref","first-page":"142","DOI":"10.1006\/bmme.1996.0042","article-title":"others, Retroviral vector-mediated transfer of the galactocerebrosidase (GALC) cDNA leads to overexpression and transfer of GALC activity to neighboring cells","volume":"58","author":"Rafi","year":"1996","journal-title":"Biochem. Mol. Med."},{"key":"10.1016\/j.addr.2023.115132_b0490","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1023\/A:1022574323784","article-title":"Transduction of cultured oligodendrocytes from normal and twitcher mice by a retroviral vector containing human galactocerebrosidase (GALC) cDNA","volume":"24","author":"Costantino-Ceccarini","year":"1999","journal-title":"Neurochem. Res."},{"key":"10.1016\/j.addr.2023.115132_b0495","doi-asserted-by":"crossref","first-page":"600","DOI":"10.1006\/nbdi.2001.0407","article-title":"Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction","volume":"8","author":"Luddi","year":"2001","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.addr.2023.115132_b0500","doi-asserted-by":"crossref","first-page":"2031","DOI":"10.1038\/mt.2012.153","article-title":"Extended normal life after AAVrh10-mediated gene therapy in the mouse model of krabbe disease","volume":"20","author":"Rafi","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.addr.2023.115132_b0505","doi-asserted-by":"crossref","first-page":"3250","DOI":"10.1093\/hmg\/ddu034","article-title":"Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy","volume":"23","author":"Lattanzi","year":"2014","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0510","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1089\/hum.2021.245","article-title":"others, Efficacy and safety of a Krabbe disease gene therapy","volume":"33","author":"Hordeaux","year":"2022","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.addr.2023.115132_b0515","doi-asserted-by":"crossref","first-page":"785","DOI":"10.1089\/hum.2017.151","article-title":"AAVrh10 gene therapy ameliorates central and peripheral nervous system disease in canine globoid cell leukodystrophy (Krabbe disease)","volume":"29","author":"Bradbury","year":"2018","journal-title":"Human Gene Therapy"},{"key":"10.1016\/j.addr.2023.115132_b0520","doi-asserted-by":"crossref","first-page":"4906","DOI":"10.1172\/JCI133953","article-title":"others, Krabbe disease successfully treated via monotherapy of intrathecal gene therapy","volume":"130","author":"Bradbury","year":"2020","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.addr.2023.115132_b0525","doi-asserted-by":"crossref","first-page":"2208","DOI":"10.1093\/hmg\/ddq099","article-title":"Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models","volume":"19","author":"Lattanzi","year":"2010","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0530","doi-asserted-by":"crossref","first-page":"489","DOI":"10.15252\/emmm.201505850","article-title":"Pervasive supply of therapeutic lysosomal enzymes in the CNS of normal and krabbe-affected non-human primates by intracerebral lentiviral gene therapy","volume":"8","author":"Meneghini","year":"2016","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.addr.2023.115132_b0535","doi-asserted-by":"crossref","unstructured":"L.C. Parr-Brownlie, C. Bosch-Bouju, L. Schoderboeck, R.J. Sizemore, W.C. Abraham, S.M. Hughes, Lentiviral vectors as tools to understand central nervous system biology in mammalian model organisms, front. Mol. Neurosci. 8 (2015) 14, Frontiers in Molecular Neuroscience. 8 (2015) 14, doi: 10.3389\/fnmol.2015.00014.","DOI":"10.3389\/fnmol.2015.00014"},{"key":"10.1016\/j.addr.2023.115132_b0540","doi-asserted-by":"crossref","first-page":"757","DOI":"10.1517\/14712598.2012.681463","article-title":"The advent of AAV9 expands applications for brain and spinal cord gene delivery","volume":"12","author":"Dayton","year":"2012","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.addr.2023.115132_b0545","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s11689-018-9234-0","article-title":"Recent progress and considerations for AAV gene therapies targeting the central nervous system","volume":"10","author":"Lykken","year":"2018","journal-title":"J. Neurodev. Disord."},{"key":"10.1016\/j.addr.2023.115132_b0550","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1038\/s41593-021-00969-4","article-title":"others, AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset","volume":"25","author":"Goertsen","year":"2022","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.addr.2023.115132_b0555","doi-asserted-by":"crossref","first-page":"328","DOI":"10.1016\/j.brainresrev.2010.05.003","article-title":"Looking at the blood-brain barrier: Molecular anatomy and possible investigation approaches","volume":"64","author":"Cardoso","year":"2010","journal-title":"Brain Res. Rev."},{"key":"10.1016\/j.addr.2023.115132_b0560","doi-asserted-by":"crossref","first-page":"1584","DOI":"10.1038\/nm.3407","article-title":"Development, maintenance and disruption of the blood-brain barrier","volume":"19","author":"Obermeier","year":"2013","journal-title":"Nat. Med."},{"key":"10.1016\/j.addr.2023.115132_b0565","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1007\/s00401-018-1815-1","article-title":"Functional morphology of the blood\u2013brain barrier in health and disease","volume":"135","author":"Liebner","year":"2018","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.addr.2023.115132_b0570","doi-asserted-by":"crossref","DOI":"10.1016\/j.neulet.2018.06.033","article-title":"Blood-brain barrier regulation in psychiatric disorders","volume":"726","author":"Kealy","year":"2020","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.addr.2023.115132_b0575","doi-asserted-by":"crossref","first-page":"1203","DOI":"10.1002\/jnr.23762","article-title":"New therapeutic approaches for krabbe disease: The potential of pharmacological chaperones","volume":"94","author":"Spratley","year":"2016","journal-title":"Journal of Neuroscience Research."},{"key":"10.1016\/j.addr.2023.115132_b0580","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1007\/s10456-015-9481-6","article-title":"Brain angioarchitecture and intussusceptive microvascular growth in a murine model of krabbe disease","volume":"18","author":"Giacomini","year":"2015","journal-title":"Angiogenesis"},{"key":"10.1016\/j.addr.2023.115132_b0585","doi-asserted-by":"crossref","first-page":"20097","DOI":"10.1073\/pnas.1912108116","article-title":"others, Genetic ablation of acid ceramidase in krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target","volume":"116","author":"Li","year":"2019","journal-title":"Proc. Natl. Acad. Sci."},{"key":"10.1016\/j.addr.2023.115132_b0590","doi-asserted-by":"crossref","first-page":"1359","DOI":"10.1002\/jnr.23744","article-title":"Endothelial cell dysfunction in globoid cell leukodystrophy","volume":"94","author":"Belleri","year":"2016","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.addr.2023.115132_b0595","doi-asserted-by":"crossref","first-page":"186","DOI":"10.1016\/0006-8993(87)90499-9","article-title":"Blood-brain barrier permeability to horseradish peroxidase in twitcher and cuprizone-intoxicated mice","volume":"425","author":"Kondo","year":"1987","journal-title":"Brain Res."},{"key":"10.1016\/j.addr.2023.115132_b0600","doi-asserted-by":"crossref","first-page":"2859","DOI":"10.1093\/brain\/awt215","article-title":"Inhibition of angiogenesis by -galactosylceramidase deficiency in globoid cell leukodystrophy","volume":"136","author":"Belleri","year":"2013","journal-title":"Brain"},{"key":"10.1016\/j.addr.2023.115132_b0605","doi-asserted-by":"crossref","first-page":"1341","DOI":"10.1002\/jnr.23804","article-title":"Neuroimmune mechanisms in Krabbe\u2019s disease","volume":"94","author":"Potter","year":"2016","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.addr.2023.115132_b0610","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1111\/j.1365-2990.2008.01005.x","article-title":"Review: Role of developmental inflammation and blood-brain barrier dysfunction in neurodevelopmental and neurodegenerative diseases","volume":"35","author":"Stolp","year":"2009","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"10.1016\/j.addr.2023.115132_b0615","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.addr.2019.11.009","article-title":"The blood-brain barrier: Physiology and strategies for drug delivery","volume":"165\u2013166","author":"Pandit","year":"2020","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.addr.2023.115132_b0620","doi-asserted-by":"crossref","first-page":"332","DOI":"10.1016\/j.addr.2021.01.015","article-title":"Targeting the blood-brain barrier for the delivery of stroke therapies","volume":"171","author":"D\u2019Souza","year":"2021","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.addr.2023.115132_b0625","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1517\/17425247.2.2.299","article-title":"Targeted delivery across the blood-brain barrier","volume":"2","author":"Gaillard","year":"2005","journal-title":"Expert Opin. Drug Deliv."},{"key":"10.1016\/j.addr.2023.115132_b0630","doi-asserted-by":"crossref","first-page":"4690","DOI":"10.1039\/C6CS00076B","article-title":"Blood\u2013brain barrier shuttle peptides: An emerging paradigm for brain delivery","volume":"45","author":"Oller-Salvia","year":"2016","journal-title":"Chem. Soc. Rev."},{"key":"10.1016\/j.addr.2023.115132_b0635","doi-asserted-by":"crossref","first-page":"542","DOI":"10.1080\/1061186X.2018.1533556","article-title":"Targeting central nervous system pathologies with nanomedicines","volume":"27","author":"Mizrahy","year":"2019","journal-title":"J. Drug Target."},{"key":"10.1016\/j.addr.2023.115132_b0640","doi-asserted-by":"crossref","first-page":"2101090","DOI":"10.1002\/advs.202101090","article-title":"Development of novel therapeutics targeting the blood\u2013brain barrier: From barrier to carrier","volume":"8","author":"Li","year":"2021","journal-title":"Adv. Sci."},{"key":"10.1016\/j.addr.2023.115132_b0645","doi-asserted-by":"crossref","first-page":"584","DOI":"10.1182\/blood-2004-12-4942","article-title":"ICAM-1 regulates neutrophil adhesion and transcellular migration of TNF-\u03b1-activated vascular endothelium under flow","volume":"106","author":"Yang","year":"2005","journal-title":"Blood"},{"key":"10.1016\/j.addr.2023.115132_b0650","doi-asserted-by":"crossref","first-page":"1031","DOI":"10.1016\/j.jconrel.2022.07.022","article-title":"Altered blood-brain barrier transport of nanotherapeutics in lysosomal storage diseases","volume":"349","author":"Solomon","year":"2022","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0655","doi-asserted-by":"crossref","first-page":"520","DOI":"10.1152\/physrev.1987.67.2.520","article-title":"The physiology of transferrin and transferrin receptors","volume":"67","author":"Huebers","year":"1987","journal-title":"Physiol. Rev."},{"key":"10.1016\/j.addr.2023.115132_b0660","doi-asserted-by":"crossref","first-page":"561","DOI":"10.1124\/pr.54.4.561","article-title":"Targeted Drug Delivery via the Transferrin Receptor-Mediated Endocytosis Pathway","volume":"54","author":"Qian","year":"2002","journal-title":"Pharmacol. Rev."},{"key":"10.1016\/j.addr.2023.115132_b0665","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1016\/j.clim.2006.06.010","article-title":"The transferrin receptor part I: Biology and targeting with cytotoxic antibodies for the treatment of cancer","volume":"121","author":"Daniels","year":"2006","journal-title":"Clin. Immunol."},{"key":"10.1016\/j.addr.2023.115132_b0670","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1016\/j.jconrel.2015.11.032","article-title":"Revisiting nanoparticle technology for blood\u2013brain barrier transport: Unfolding at the endothelial gate improves the fate of transferrin receptor-targeted liposomes","volume":"222","author":"Johnsen","year":"2016","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0675","doi-asserted-by":"crossref","first-page":"12486","DOI":"10.1073\/pnas.1517048112","article-title":"Increased brain uptake of targeted nanoparticles by adding an acid-cleavable linkage between transferrin and the nanoparticle core","volume":"112","author":"Clark","year":"2015","journal-title":"Proceedings of the National Academy of Sciences of the United States of America."},{"key":"10.1016\/j.addr.2023.115132_b0680","doi-asserted-by":"crossref","first-page":"3416","DOI":"10.7150\/thno.25228","article-title":"Antibody affinity and valency impact brain uptake of transferrin receptor-targeted gold nanoparticles","volume":"8","author":"Johnsen","year":"2018","journal-title":"Theranostics."},{"key":"10.1016\/j.addr.2023.115132_b0685","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1016\/j.jconrel.2019.01.005","article-title":"Modulating the antibody density changes the uptake and transport at the blood-brain barrier of both transferrin receptor-targeted gold nanoparticles and liposomal cargo","volume":"295","author":"Johnsen","year":"2019","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0690","doi-asserted-by":"crossref","first-page":"125","DOI":"10.3109\/10611861003734001","article-title":"Targeting the insulin receptor: Nanoparticles for drug delivery across the blood-brain barrier (BBB)","volume":"19","author":"Ulbrich","year":"2011","journal-title":"J. Drug Target."},{"key":"10.1016\/j.addr.2023.115132_b0695","doi-asserted-by":"crossref","first-page":"730","DOI":"10.3109\/1061186X.2013.812094","article-title":"Solid lipid nanoparticles carrying chemotherapeutic drug across the blood-brain barrier through insulin receptor-mediated pathway","volume":"21","author":"Kuo","year":"2013","journal-title":"J. Drug Target."},{"key":"10.1016\/j.addr.2023.115132_b0700","doi-asserted-by":"crossref","first-page":"2146","DOI":"10.1039\/C3NR04878K","article-title":"Transport of nanoparticles through the blood-brain barrier for imaging and therapeutic applications","volume":"6","author":"Shilo","year":"2014","journal-title":"Nanoscale"},{"key":"10.1016\/j.addr.2023.115132_b0705","doi-asserted-by":"crossref","first-page":"1533","DOI":"10.2217\/nnm-2017-0022","article-title":"The effect of nanoparticle size on the ability to cross the blood-brain barrier: An in vivo study","volume":"12","author":"Betzer","year":"2017","journal-title":"Nanomedicine"},{"key":"10.1016\/j.addr.2023.115132_b0710","doi-asserted-by":"crossref","unstructured":"W.H. Wunner, K.-K. Conzelmann, Rabies virus, in: Rabies, Elsevier, 2020: pp. 43\u201381.","DOI":"10.1016\/B978-0-12-818705-0.00002-9"},{"key":"10.1016\/j.addr.2023.115132_b0715","doi-asserted-by":"crossref","first-page":"4123","DOI":"10.1021\/acsnano.7b08172","article-title":"Targeted Brain Delivery of Rabies Virus Glycoprotein 29-Modified Deferoxamine-Loaded Nanoparticles Reverses Functional Deficits in Parkinsonian Mice","volume":"12","author":"You","year":"2018","journal-title":"ACS Nano"},{"key":"10.1016\/j.addr.2023.115132_b0720","doi-asserted-by":"crossref","first-page":"2109","DOI":"10.1093\/hmg\/10.19.2109","article-title":"Rabies virus glycoprotein pseudotyping of lentiviral vectors enables retrograde axonal transport and access to the nervous system after peripheral delivery","volume":"10","author":"Mazarakis","year":"2001","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.addr.2023.115132_b0725","doi-asserted-by":"crossref","DOI":"10.1016\/j.ijpharm.2019.118449","article-title":"Rabies virus glycoprotein (RVG29)-linked microRNA-124-loaded polymeric nanoparticles inhibit neuroinflammation in a Parkinson\u2019s disease model","volume":"567","author":"Gan","year":"2019","journal-title":"Int. J. Pharm."},{"key":"10.1016\/j.addr.2023.115132_b0730","doi-asserted-by":"crossref","first-page":"4014","DOI":"10.1021\/acsnano.1c09601","article-title":"Rabies Virus Glycoprotein-Mediated Transportation and T Cell Infiltration to Brain Tumor by Magnetoelectric Gold Yarnballs","volume":"16","author":"Cheng","year":"2022","journal-title":"ACS Nano"},{"key":"10.1016\/j.addr.2023.115132_b0735","doi-asserted-by":"crossref","DOI":"10.1016\/j.msec.2021.112615","article-title":"Enhanced activity of AZD5582 and SM-164 in rabies virus glycoprotein-lactoferrin-liposomes to downregulate inhibitors of apoptosis proteins in glioblastoma","volume":"133","author":"Kuo","year":"2022","journal-title":"Biomaterials Advances."},{"key":"10.1016\/j.addr.2023.115132_b0740","doi-asserted-by":"crossref","first-page":"1170","DOI":"10.1016\/j.biomaterials.2012.09.047","article-title":"Brain-targeted delivery of protein using chitosan- and RVG peptide-conjugated, pluronic-based nano-carrier","volume":"34","author":"Kim","year":"2013","journal-title":"Biomaterials"},{"key":"10.1016\/j.addr.2023.115132_b0745","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1083\/jcb.126.2.465","article-title":"Upregulation of the low density lipoprotein receptor at the blood-brain barrier: Intercommunications between brain capillary endothelial cells and astrocytes","volume":"126","author":"Dehouck","year":"1994","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.addr.2023.115132_b0750","doi-asserted-by":"crossref","first-page":"877","DOI":"10.1083\/jcb.138.4.877","article-title":"A new function for the LDL receptor: Transcytosis of LDL across the blood-brain barrier","volume":"138","author":"Dehouck","year":"1997","journal-title":"Journal of Cell Biology."},{"key":"10.1016\/j.addr.2023.115132_b0755","doi-asserted-by":"crossref","first-page":"1884","DOI":"10.1111\/j.1538-7836.2005.01371.x","article-title":"Beyond endocytosis: LRP function in cell migration, proliferation and vascular permeability","volume":"3","author":"Lillis","year":"2005","journal-title":"J. Thromb. Haemost."},{"key":"10.1016\/j.addr.2023.115132_b0760","doi-asserted-by":"crossref","DOI":"10.1088\/0957-4484\/26\/49\/495103","article-title":"Solid lipid nanoparticles as a vehicle for brain-targeted drug delivery: Two new strategies of functionalization with Apolipoprotein E","volume":"26","author":"Neves","year":"2015","journal-title":"Nanotechnology"},{"key":"10.1016\/j.addr.2023.115132_b0765","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1186\/s12951-016-0177-x","article-title":"Brain-targeted delivery of resveratrol using solid lipid nanoparticles functionalized with Apolipoprotein E","volume":"14","author":"Neves","year":"2016","journal-title":"J. Nanobiotechnol."},{"key":"10.1016\/j.addr.2023.115132_b0770","doi-asserted-by":"crossref","first-page":"995","DOI":"10.1021\/acs.bioconjchem.6b00705","article-title":"Apo E-Functionalization of Solid Lipid Nanoparticles Enhances Brain Drug Delivery: Uptake Mechanism and Transport Pathways","volume":"28","author":"Neves","year":"2017","journal-title":"Bioconjug. Chem."},{"key":"10.1016\/j.addr.2023.115132_b0775","first-page":"429","article-title":"Artificial apolipoprotein corona enables nanoparticle brain targeting, Nanomedicine: Nanotechnology","volume":"14","author":"Dal Magro","year":"2018","journal-title":"Biology, and Medicine."},{"key":"10.1016\/j.addr.2023.115132_b0780","first-page":"552","article-title":"Cell membrane biomimetic nanoparticles for inflammation and cancer targeting in drug delivery, Biomaterials","volume":"8","author":"Wang","year":"2020","journal-title":"Science"},{"key":"10.1016\/j.addr.2023.115132_b0785","first-page":"1272","article-title":"Neutrophil Membrane-Derived Nanovesicles Alleviate Inflammation to Protect Mouse Brain Injury from Ischemic Stroke","volume":"13","author":"Dong","year":"2019","journal-title":"ACS Nano"},{"key":"10.1016\/j.addr.2023.115132_b0790","doi-asserted-by":"crossref","first-page":"1902011","DOI":"10.1002\/smll.201902011","article-title":"Targeted Drug Delivery to Stroke via Chemotactic Recruitment of Nanoparticles Coated with Membrane of Engineered Neural Stem Cells","volume":"15","author":"Ma","year":"2019","journal-title":"Small"},{"key":"10.1016\/j.addr.2023.115132_b0795","doi-asserted-by":"crossref","first-page":"543","DOI":"10.1038\/nmat2442","article-title":"Understanding biophysicochemical interactions at the nano-bio interface","volume":"8","author":"Nel","year":"2009","journal-title":"Nat. Mater."},{"key":"10.1016\/j.addr.2023.115132_b0800","doi-asserted-by":"crossref","first-page":"11476","DOI":"10.1021\/acs.chemrev.7b00194","article-title":"Form Follows Function: Nanoparticle Shape and Its Implications for Nanomedicine","volume":"117","author":"Kinnear","year":"2017","journal-title":"Chemical Reviews."},{"key":"10.1016\/j.addr.2023.115132_b0805","first-page":"aay1344","article-title":"Augmentation of Brain Tumor Interstitial Flow via Focused Ultrasound Promotes Brain-Penetrating Nanoparticle Dispersion and Transfection, Science","volume":"6","author":"Curley","year":"2020","journal-title":"Advances"},{"key":"10.1016\/j.addr.2023.115132_b0810","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/j.jconrel.2015.02.027","article-title":"Remote control of the permeability of the blood\u2013brain barrier by magnetic heating of nanoparticles: A proof of concept for brain drug delivery","volume":"206","author":"Tabatabaei","year":"2015","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0815","doi-asserted-by":"crossref","unstructured":"H. Baghirov, D. Karaman, T. Viitala, A. Duchanoy, Y.-R. Lou, V. Mamaeva, E. Pryazhnikov, L. Khiroug, C. de Lange Davies, C. Sahlgren, J.M. Rosenholm, Feasibility study of the permeability and uptake of mesoporous silica nanoparticles across the blood\u2013brain barrier, PLoS One 11 (2016) e0160705, doi: 10.1371\/journal.pone.0160705.","DOI":"10.1371\/journal.pone.0160705"},{"key":"10.1016\/j.addr.2023.115132_b0820","doi-asserted-by":"crossref","first-page":"10753","DOI":"10.1073\/pnas.1308345110","article-title":"Using shape effects to target antibody-coated nanoparticles to lung and brain endothelium","volume":"110","author":"Kolhar","year":"2013","journal-title":"Proc. Natl. Acad. Sci."},{"key":"10.1016\/j.addr.2023.115132_b0825","doi-asserted-by":"crossref","first-page":"149ra119","DOI":"10.1126\/scitranslmed.3003594","article-title":"A dense poly(ethylene glycol) coating improves penetration of large polymeric nanoparticles within brain tissue","volume":"4","author":"Nance","year":"2012","journal-title":"Science Translational Medicine"},{"key":"10.1016\/j.addr.2023.115132_b0830","first-page":"1669","article-title":"Nanoparticle size and chemical modification play a crucial role in the interaction of nano gold with the brain: Extent of accumulation and toxicity, Biomaterials","volume":"8","author":"Mahmoud","year":"2020","journal-title":"Science"},{"key":"10.1016\/j.addr.2023.115132_b0835","doi-asserted-by":"crossref","first-page":"711","DOI":"10.1517\/17425247.2014.897693","article-title":"Drug delivery across the blood\u2013brain barrier using focused ultrasound","volume":"11","author":"Burgess","year":"2014","journal-title":"Expert Opin. Drug Deliv."},{"key":"10.1016\/j.addr.2023.115132_b0840","first-page":"7","article-title":"Applications of focused ultrasound in the brain: From thermoablation to drug delivery, Nature Reviews","volume":"17","author":"Meng","year":"2021","journal-title":"Neurology"},{"key":"10.1016\/j.addr.2023.115132_b0845","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/j.jconrel.2014.06.031","article-title":"Non-invasive delivery of stealth, brain-penetrating nanoparticles across the blood\u2013brain barrier using MRI-guided focused ultrasound","volume":"189","author":"Nance","year":"2014","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0850","doi-asserted-by":"crossref","first-page":"18220","DOI":"10.1038\/s41598-020-75253-9","article-title":"Investigating the optimum size of nanoparticles for their delivery into the brain assisted by focused ultrasound-induced blood\u2013brain barrier opening","volume":"10","author":"Ohta","year":"2020","journal-title":"Sci. Rep."},{"key":"10.1016\/j.addr.2023.115132_b0855","series-title":"Progress in Brain Research","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1016\/S0079-6123(08)62044-0","article-title":"Blood-brain barrier permeability during hyperthermia","author":"Shivers","year":"1998"},{"key":"10.1016\/j.addr.2023.115132_b0860","doi-asserted-by":"crossref","first-page":"393","DOI":"10.1016\/j.tins.2020.12.002","article-title":"Blood-Brain Barrier Dynamics to Maintain Brain Homeostasis","volume":"44","author":"Segarra","year":"2021","journal-title":"Trends Neurosci."},{"key":"10.1016\/j.addr.2023.115132_b0865","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1016\/0006-8993(77)90726-0","article-title":"Microwave alteration of the blood-brain barrier system of rats","volume":"126","author":"Oscar","year":"1977","journal-title":"Brain Res."},{"key":"10.1016\/j.addr.2023.115132_b0870","doi-asserted-by":"crossref","first-page":"17589","DOI":"10.1039\/D2NR02210A","article-title":"Transmigration of magnetite nanoparticles across the blood\u00e2\u20ac\u201cbrain barrier in a rodent model: Influence of external and alternating magnetic fields","volume":"14","author":"Gupta","year":"2022","journal-title":"Nanoscale"},{"key":"10.1016\/j.addr.2023.115132_b0875","first-page":"1","article-title":"Nanomaterials for convection-enhanced delivery of agents to treat brain tumors, Current Opinion in Biomedical","volume":"4","author":"Seo","year":"2017","journal-title":"Engineering"},{"key":"10.1016\/j.addr.2023.115132_b0880","first-page":"381","article-title":"Convection-enhanced delivery of nanodiamond drug delivery platforms for intracranial tumor treatment, Nanomedicine: Nanotechnology","volume":"10","author":"Xi","year":"2014","journal-title":"Biology and Medicine."},{"key":"10.1016\/j.addr.2023.115132_b0885","doi-asserted-by":"crossref","first-page":"3305","DOI":"10.1021\/nl071521o","article-title":"Active Nanodiamond Hydrogels for Chemotherapeutic Delivery","volume":"7","author":"Huang","year":"2007","journal-title":"Nano Lett."},{"key":"10.1016\/j.addr.2023.115132_b0890","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3001713","article-title":"Nanodiamond Therapeutic Delivery Agents Mediate Enhanced Chemoresistant Tumor Treatment","volume":"3","author":"Chow","year":"2011","journal-title":"Science Translational Medicine."},{"key":"10.1016\/j.addr.2023.115132_b0895","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/j.jconrel.2016.04.006","article-title":"Distribution of polymer nanoparticles by convection-enhanced delivery to brain tumors","volume":"232","author":"Saucier-Sawyer","year":"2016","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0900","doi-asserted-by":"crossref","first-page":"138","DOI":"10.1016\/j.jconrel.2017.06.028","article-title":"Liposomal temozolomide drug delivery using convection enhanced delivery","volume":"261","author":"Nordling-David","year":"2017","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0905","doi-asserted-by":"crossref","first-page":"1184","DOI":"10.1016\/S1359-6446(02)02529-1","article-title":"Nasal drug delivery: New developments and strategies","volume":"7","author":"Illum","year":"2002","journal-title":"Drug Discov. Today"},{"key":"10.1016\/j.addr.2023.115132_b0910","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1016\/S0168-3659(02)00363-2","article-title":"Nasal drug delivery\u2014possibilities, problems and solutions","volume":"87","author":"Illum","year":"2003","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0915","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1038\/nrd.2018.183","article-title":"Non-invasive delivery strategies for biologics","volume":"18","author":"Anselmo","year":"2018","journal-title":"Nature Reviews Drug Discovery."},{"key":"10.1016\/j.addr.2023.115132_b0920","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1016\/j.jconrel.2018.05.011","article-title":"Nose-to-brain drug delivery: An update on clinical challenges and progress towards approval of anti-Alzheimer drugs","volume":"281","author":"Agrawal","year":"2018","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0925","doi-asserted-by":"crossref","first-page":"2000076","DOI":"10.1002\/adtp.202000076","article-title":"Polymeric Nanoparticles for Nasal Drug Delivery to the Brain: Relevance to Alzheimer\u2019s Disease","volume":"4","author":"Rabiee","year":"2021","journal-title":"Advanced Therapeutics."},{"key":"10.1016\/j.addr.2023.115132_b0930","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/j.ijpharm.2009.06.019","article-title":"Nanoparticles for direct nose-to-brain delivery of drugs","volume":"379","author":"Mistry","year":"2009","journal-title":"Int. J. Pharm."},{"key":"10.1016\/j.addr.2023.115132_b0935","doi-asserted-by":"crossref","first-page":"7176","DOI":"10.1021\/acs.nanolett.6b03515","article-title":"Rotation-Facilitated Rapid Transport of Nanorods in Mucosal Tissues","volume":"16","author":"Yu","year":"2016","journal-title":"Nano Lett."},{"key":"10.1016\/j.addr.2023.115132_b0940","doi-asserted-by":"crossref","first-page":"202","DOI":"10.1016\/j.jconrel.2014.07.045","article-title":"Nanoparticle penetration of human cervicovaginal mucus: The effect of polyvinyl alcohol","volume":"192","author":"Yang","year":"2014","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b0945","doi-asserted-by":"crossref","first-page":"9217","DOI":"10.1021\/acsnano.5b03876","article-title":"Impact of Surface Polyethylene Glycol (PEG) Density on Biodegradable Nanoparticle Transport in Mucus ex Vivo and Distribution in Vivo","volume":"9","author":"Xu","year":"2015","journal-title":"ACS Nano"},{"key":"10.1016\/j.addr.2023.115132_b0950","first-page":"1688","article-title":"Nose-to-brain drug delivery mediated by polymeric nanoparticles: Influence of PEG surface coating, Drug Delivery and Translational","volume":"10","author":"de Oliveira Junior","year":"2020","journal-title":"Research"},{"key":"10.1016\/j.addr.2023.115132_b0955","doi-asserted-by":"crossref","first-page":"7565","DOI":"10.1021\/acsami.1c14975","article-title":"Hybrid Lipid\/Polymer Nanoparticles to Tackle the Cystic Fibrosis Mucus Barrier in siRNA Delivery to the Lungs: Does PEGylation Make the Difference?","volume":"14","author":"Conte","year":"2022","journal-title":"ACS Appl. Mater. Interfaces"},{"key":"10.1016\/j.addr.2023.115132_b0960","first-page":"eaax7462","article-title":"Brain-targeted enzyme-loaded nanoparticles: A breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease, Science","volume":"5","author":"Del Grosso","year":"2019","journal-title":"Advances"},{"key":"10.1016\/j.addr.2023.115132_b0965","doi-asserted-by":"crossref","first-page":"5820","DOI":"10.1038\/s41467-020-19505-2","article-title":"The once and future gene therapy","volume":"11","author":"Bulaklak","year":"2020","journal-title":"Nature Communications."},{"key":"10.1016\/j.addr.2023.115132_b0970","doi-asserted-by":"crossref","first-page":"824","DOI":"10.1038\/s41587-020-0561-9","article-title":"Genome editing with CRISPR\u2013cas nucleases, base editors, transposases and prime editors","volume":"38","author":"Anzalone","year":"2020","journal-title":"Nature Biotechnology."},{"key":"10.1016\/j.addr.2023.115132_b0975","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1038\/s41580-019-0131-5","article-title":"The next generation of CRISPR\u2013cas technologies and applications","volume":"20","author":"Pickar-Oliver","year":"2019","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.addr.2023.115132_b0980","doi-asserted-by":"crossref","first-page":"400","DOI":"10.1007\/s11095-007-9357-6","article-title":"Lysosomal Enzyme Replacement of the Brain with Intravenous Non-Viral Gene Transfer","volume":"25","author":"Zhang","year":"2008","journal-title":"Pharm. Res."},{"key":"10.1016\/j.addr.2023.115132_b0985","doi-asserted-by":"crossref","first-page":"7567","DOI":"10.1073\/pnas.130187497","article-title":"Noninvasive gene targeting to the brain","volume":"97","author":"Shi","year":"2000","journal-title":"Proc. Natl. Acad. Sci."},{"key":"10.1016\/j.addr.2023.115132_b0990","doi-asserted-by":"crossref","first-page":"2107534","DOI":"10.1002\/smll.202107534","article-title":"Brain-Penetration and Neuron-Targeting DNA Nanoflowers Co-Delivering miR-124 and Rutin for Synergistic Therapy of Alzheimer\u2019s Disease","volume":"18","author":"Ouyang","year":"2022","journal-title":"Small"},{"key":"10.1016\/j.addr.2023.115132_b0995","first-page":"889","article-title":"Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair, Nature","volume":"1","author":"Lee","year":"2017","journal-title":"Biomed. Eng."},{"key":"10.1016\/j.addr.2023.115132_b1000","first-page":"497","article-title":"Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours, Nature","volume":"2","author":"Lee","year":"2018","journal-title":"Biomed. Eng."},{"key":"10.1016\/j.addr.2023.115132_b1005","doi-asserted-by":"crossref","DOI":"10.1016\/j.addr.2022.114464","article-title":"Current treatment options and novel nanotechnology-driven enzyme replacement strategies for lysosomal storage disorders","volume":"188","author":"Del Grosso","year":"2022","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.addr.2023.115132_b1010","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1038\/nbt.3330","article-title":"Principles of nanoparticle design for overcoming biological barriers to drug delivery","volume":"33","author":"Blanco","year":"2015","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.addr.2023.115132_b1015","doi-asserted-by":"crossref","unstructured":"M. Salvalaio, L. Rigon, D. Belletti, F. D\u2019Avanzo, F. Pederzoli, B. Ruozi, O. Marin, M.A. Vandelli, F. Forni, M. Scarpa, R. Tomanin, G. Tosi, Targeted Polymeric Nanoparticles for Brain Delivery of High Molecular Weight Molecules in Lysosomal Storage Disorders, PLoS One 11 (2016) e0156452, doi: 10.1371\/journal.pone.0156452.","DOI":"10.1371\/journal.pone.0156452"},{"key":"10.1016\/j.addr.2023.115132_b1020","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jconrel.2017.02.016","article-title":"Potential of surfactant-coated nanoparticles to improve brain delivery of arylsulfatase A","volume":"253","author":"Schuster","year":"2017","journal-title":"J. Control. Release"},{"key":"10.1016\/j.addr.2023.115132_b1025","doi-asserted-by":"crossref","first-page":"663","DOI":"10.1016\/S0006-291X(85)80212-6","article-title":"Enzyme replacement with liposomes containing beta-galactosidase from charonia lumpas in murine globoid cell leukodystrophy (Twitcher)","volume":"127","author":"Umezawa","year":"1985","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.addr.2023.115132_b1030","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1038\/nrn.2017.136","article-title":"Regenerating CNS myelin \u2014 from mechanisms to experimental medicines","volume":"18","author":"Franklin","year":"2017","journal-title":"Nat. Rev. Neurosci."},{"key":"10.1016\/j.addr.2023.115132_b1035","doi-asserted-by":"crossref","first-page":"872","DOI":"10.1021\/acschemneuro.8b00402","article-title":"Targeting Neuroinflammation as a Therapeutic Strategy for Alzheimer\u2019s Disease: Mechanisms, Drug Candidates, and New Opportunities","volume":"10","author":"Fu","year":"2018","journal-title":"ACS Chem. Nerosci."},{"key":"10.1016\/j.addr.2023.115132_b1040","doi-asserted-by":"crossref","first-page":"3104","DOI":"10.1523\/JNEUROSCI.2346-19.2020","article-title":"Fingolimod rescues demyelination in a mouse model of Krabbe\u2019s disease","volume":"40","author":"B\u00e9chet","year":"2020","journal-title":"J. Neurosci."},{"key":"10.1016\/j.addr.2023.115132_b1045","doi-asserted-by":"crossref","first-page":"5620","DOI":"10.1111\/ejn.15391","article-title":"Local delivery of fingolimod through PLGA nanoparticles and PuraMatrix-embedded neural precursor cells promote motor function recovery and tissue repair in spinal cord injury","volume":"54","author":"Zeraatpisheh","year":"2021","journal-title":"Eur. J. Neurosci."},{"key":"10.1016\/j.addr.2023.115132_b1050","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1016\/j.xphs.2016.07.026","article-title":"Application of Artificial neural networks in the design and optimization of a nanoparticulate fingolimod delivery system based on biodegradable poly(3-hydroxybutyrate-co-3-hydroxyvalerate)","volume":"106","author":"Shahsavari","year":"2017","journal-title":"J. Pharm. Sci."},{"key":"10.1016\/j.addr.2023.115132_b1055","doi-asserted-by":"crossref","first-page":"216","DOI":"10.1038\/nature14335","article-title":"Drug-based modulation of endogenous stem cells promotes functional remyelination in vivo","volume":"522","author":"Najm","year":"2015","journal-title":"Nature"},{"key":"10.1016\/j.addr.2023.115132_b1060","doi-asserted-by":"crossref","first-page":"954","DOI":"10.1038\/nm.3618","article-title":"Micropillar arrays as a high-throughput screening platform for therapeutics in multiple sclerosis","volume":"20","author":"Mei","year":"2014","journal-title":"Nat. Med."},{"key":"10.1016\/j.addr.2023.115132_b1065","doi-asserted-by":"crossref","first-page":"7925","DOI":"10.1523\/JNEUROSCI.1493-16.2016","article-title":"Identification of the kappa-opioid receptor as a therapeutic target for oligodendrocyte remyelination","volume":"36","author":"Mei","year":"2016","journal-title":"J. Neurosci."},{"key":"10.1016\/j.addr.2023.115132_b1070","doi-asserted-by":"crossref","first-page":"2481","DOI":"10.1016\/S0140-6736(17)32346-2","article-title":"Clemastine fumarate as a remyelinating therapy for multiple sclerosis (ReBUILD): A randomised, controlled, double-blind, crossover trial","volume":"390","author":"Green","year":"2017","journal-title":"Lancet"},{"key":"10.1016\/j.addr.2023.115132_b1075","doi-asserted-by":"crossref","first-page":"300","DOI":"10.1016\/j.supflu.2010.06.001","article-title":"Organic nanoparticles recovery in supercritical antisolvent precipitation","volume":"55","author":"Torino","year":"2010","journal-title":"J. Supercrit. Fluids"},{"key":"10.1016\/j.addr.2023.115132_b1080","doi-asserted-by":"crossref","DOI":"10.3389\/fncel.2020.576037","article-title":"Neuroinflammation Treatment via Targeted Delivery of Nanoparticles","volume":"14","author":"Cerqueira","year":"2020","journal-title":"Front. Cell. Neurosci."},{"key":"10.1016\/j.addr.2023.115132_b1085","doi-asserted-by":"crossref","first-page":"2608","DOI":"10.1007\/s13311-021-01109-3","article-title":"Hybrid Nanoparticles as a Novel Tool for Regulating Psychosine-Induced Neuroinflammation and Demyelination In Vitro and Ex vivo","volume":"18","author":"Clementino","year":"2021","journal-title":"Neurotherapeutics"},{"key":"10.1016\/j.addr.2023.115132_b1090","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/j.brainres.2009.09.017","article-title":"Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy","volume":"1300","author":"Luzi","year":"2009","journal-title":"Brain Res."},{"key":"10.1016\/j.addr.2023.115132_b1095","doi-asserted-by":"crossref","first-page":"247","DOI":"10.1007\/s00401-015-1426-z","article-title":"Non-steroidal anti-inflammatory drug indometacin enhances endogenous remyelination","volume":"130","author":"Preisner","year":"2015","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.addr.2023.115132_b1100","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.ijpharm.2005.08.011","article-title":"Characterization of indomethacin-loaded lipid nanoparticles by differential scanning calorimetry","volume":"304","author":"Castelli","year":"2005","journal-title":"Int. J. Pharm."},{"key":"10.1016\/j.addr.2023.115132_b1105","first-page":"231","article-title":"Anti-inflammatory effect from indomethacin nanoparticles inhaled by male mice, Journal of Aerosol Medicine and Pulmonary","volume":"21","author":"Onischuk","year":"2008","journal-title":"Drug Deliv."},{"key":"10.1016\/j.addr.2023.115132_b1110","doi-asserted-by":"crossref","first-page":"1213","DOI":"10.2147\/IJN.S196681","article-title":"Energy-dependent endocytosis is responsible for drug transcorneal penetration following the instillation of ophthalmic formulations containing indomethacin nanoparticles","volume":"14","author":"Nagai","year":"2019","journal-title":"Int. J. Nanomed."},{"key":"10.1016\/j.addr.2023.115132_b1115","doi-asserted-by":"crossref","first-page":"2591","DOI":"10.2217\/nnm-2017-0221","article-title":"Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy","volume":"12","author":"Kelly","year":"2017","journal-title":"Nanomedicine"},{"key":"10.1016\/j.addr.2023.115132_b1120","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/0003-9861(82)90290-9","article-title":"Effects of phospholipids on substrate specificity of \u03b2-galactosidase purified from Aspergillus oryzae","volume":"215","author":"Naoi","year":"1982","journal-title":"Archives of Biochem. Biophysics."},{"key":"10.1016\/j.addr.2023.115132_b1125","doi-asserted-by":"crossref","first-page":"4510","DOI":"10.1021\/acs.molpharmaceut.0c00615","article-title":"Protein Delivery by Peptide-Based Stealth Liposomes: A Biomolecular Insight into Enzyme Replacement Therapy","volume":"17","author":"Santi","year":"2020","journal-title":"Mol. Pharm."},{"key":"10.1016\/j.addr.2023.115132_b1130","doi-asserted-by":"crossref","first-page":"771","DOI":"10.1016\/j.msec.2019.03.004","article-title":"Magnetic brain targeting of naproxen-loaded polymeric micelles: Pharmacokinetics and biodistribution study","volume":"100","author":"Karami","year":"2019","journal-title":"Mater. Sci. Eng. C"}],"container-title":["Advanced Drug Delivery Reviews"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0169409X23004477?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0169409X23004477?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,9,30]],"date-time":"2025-09-30T05:41:09Z","timestamp":1759210869000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0169409X23004477"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,12]]},"references-count":226,"alternative-id":["S0169409X23004477"],"URL":"https:\/\/doi.org\/10.1016\/j.addr.2023.115132","relation":{},"ISSN":["0169-409X"],"issn-type":[{"value":"0169-409X","type":"print"}],"subject":[],"published":{"date-parts":[[2023,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Nanomedicines to treat rare neurological disorders: The case of Krabbe disease","name":"articletitle","label":"Article Title"},{"value":"Advanced Drug Delivery Reviews","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.addr.2023.115132","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2023 The Author(s). Published by Elsevier B.V.","name":"copyright","label":"Copyright"}],"article-number":"115132"}}