{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T17:19:17Z","timestamp":1792084757632,"version":"4.4.0"},"reference-count":119,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2026,6,1]],"date-time":"2026-06-01T00:00:00Z","timestamp":1780272000000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"funder":[{"DOI":"10.13039\/501100004001","name":"Guizhou Province Science and Technology Foundation","doi-asserted-by":"publisher","award":["ZSYS(2025)040"],"award-info":[{"award-number":["ZSYS(2025)040"]}],"id":[{"id":"10.13039\/501100004001","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82560833"],"award-info":[{"award-number":["82560833"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100017957","name":"Health Commission of Guizhou Province","doi-asserted-by":"publisher","award":["2026\\u2013192"],"award-info":[{"award-number":["2026\\u2013192"]}],"id":[{"id":"10.13039\/100017957","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100017957","name":"Health Commission of Guizhou Province","doi-asserted-by":"publisher","award":["2026\\u2013187"],"award-info":[{"award-number":["2026\\u2013187"]}],"id":[{"id":"10.13039\/100017957","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100010891","name":"Guizhou Provincial Health Commission","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100010891","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Ageing Research Reviews"],"published-print":{"date-parts":[[2026,6]]},"DOI":"10.1016\/j.arr.2026.103133","type":"journal-article","created":{"date-parts":[[2026,4,8]],"date-time":"2026-04-08T07:42:53Z","timestamp":1775634173000},"page":"103133","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":20,"special_numbering":"C","title":["Mechanistic research and therapeutic prospects of alternative splicing in neurodegenerative diseases"],"prefix":"10.1016","volume":"118","author":[{"given":"Xiaolan","family":"Ran","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mei","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Juan","family":"Huang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nanyu","family":"Kuang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peng","family":"Tian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jingjing","family":"Wu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fei","family":"Feng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yong","family":"Luo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1910-6660","authenticated-orcid":false,"given":"Nanqu","family":"Huang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.arr.2026.103133_bib1","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2023.106050","article-title":"Immunotherapy targeting the C-terminal domain of TDP-43 decreases neuropathology and confers neuroprotection in mouse models of ALS\/FTD","volume":"179","author":"Afroz","year":"2023","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.arr.2026.103133_bib2","doi-asserted-by":"crossref","first-page":"661","DOI":"10.1261\/rna.325107","article-title":"Global analysis of exon creation versus loss and the role of alternative splicing in 17 vertebrate genomes","volume":"13","author":"Alekseyenko","year":"2007","journal-title":"RNA"},{"key":"10.1016\/j.arr.2026.103133_bib3","doi-asserted-by":"crossref","first-page":"1647","DOI":"10.1016\/j.ymthe.2023.03.004","article-title":"Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor","volume":"31","author":"Amistadi","year":"2023","journal-title":"Mol. Ther."},{"key":"10.1016\/j.arr.2026.103133_bib4","doi-asserted-by":"crossref","DOI":"10.1172\/JCI182088","article-title":"TDP-43 dysregulation of polyadenylation site selection is a defining feature of RNA misprocessing in amyotrophic lateral sclerosis and frontotemporal dementia","volume":"135","author":"Arnold","year":"2025","journal-title":"J. Clin. Invest"},{"key":"10.1016\/j.arr.2026.103133_bib5","article-title":"Huntington disease","volume":"1","author":"Bates","year":"2015","journal-title":"Nat. Rev. Dis. Prim."},{"key":"10.1016\/j.arr.2026.103133_bib6","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1146\/annurev-neuro-070918-050501","article-title":"Antisense oligonucleotide therapies for neurodegenerative diseases","volume":"42","author":"Bennett","year":"2019","journal-title":"Annu. Rev. Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib7","doi-asserted-by":"crossref","first-page":"237","DOI":"10.1007\/s00401-006-0104-6","article-title":"Alpha-synuclein structure, posttranslational modification and alternative splicing as aggregation enhancers","volume":"112","author":"Beyer","year":"2006","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.arr.2026.103133_bib8","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aau3960","article-title":"A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson\u2019s disease","volume":"12","author":"Boussaad","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.arr.2026.103133_bib9","doi-asserted-by":"crossref","first-page":"5789","DOI":"10.3390\/molecules25245789","article-title":"Comprehensive review on Alzheimer\u2019s disease: causes and treatment","volume":"25","author":"Breijyeh","year":"2020","journal-title":"Molecules"},{"key":"10.1016\/j.arr.2026.103133_bib10","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1038\/s41586-022-04436-3","article-title":"TDP-43 loss and ALS-risk SNPs drive mis-splicing and depletion of UNC13A","volume":"603","author":"Brown","year":"2022","journal-title":"Nature"},{"key":"10.1016\/j.arr.2026.103133_bib11","doi-asserted-by":"crossref","first-page":"3606","DOI":"10.1002\/alz.13784","article-title":"The six brain-specific TAU isoforms and their role in Alzheimer\u2019s disease and related neurodegenerative dementia syndromes","volume":"20","author":"Buchholz","year":"2024","journal-title":"Alzheimer\u2019S. Dement"},{"key":"10.1016\/j.arr.2026.103133_bib12","doi-asserted-by":"crossref","first-page":"10505","DOI":"10.1128\/MCB.24.24.10505-10514.2004","article-title":"Influence of RNA secondary structure on the pre-mRNA splicing process","volume":"24","author":"Buratti","year":"2004","journal-title":"Mol. Cell. Biol."},{"key":"10.1016\/j.arr.2026.103133_bib13","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1002\/wrna.100","article-title":"Evolution of SR protein and hnRNA splicing regulatory factors","volume":"3","author":"Busch","year":"2012","journal-title":"Wiley Interdiscip. Rev. RNA"},{"key":"10.1016\/j.arr.2026.103133_bib14","doi-asserted-by":"crossref","DOI":"10.3390\/ijms22157896","article-title":"Genomic variability in the survival motor neuron genes (SMN1 and SMN2): implications for spinal muscular atrophy phenotype and therapeutics development","volume":"22","author":"Butchbach","year":"2021","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib15","doi-asserted-by":"crossref","first-page":"712","DOI":"10.3389\/fgene.2018.00712","article-title":"RNA dysregulation in amyotrophic lateral sclerosis","volume":"9","author":"Butti","year":"2018","journal-title":"Front. Genet"},{"key":"10.1016\/j.arr.2026.103133_bib16","doi-asserted-by":"crossref","first-page":"522","DOI":"10.1016\/j.omtn.2023.04.009","article-title":"Single-swap editing for the correction of common Duchenne muscular dystrophy mutations","volume":"32","author":"Chai","year":"2023","journal-title":"Mol. Ther. Nucleic Acids"},{"key":"10.1016\/j.arr.2026.103133_bib17","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1016\/S1474-4422(09)70068-7","article-title":"Non-motor symptoms of Parkinson\u2019s disease: dopaminergic pathophysiology and treatment","volume":"8","author":"Chaudhuri","year":"2009","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.arr.2026.103133_bib18","article-title":"Spinal muscular atrophy: from approved therapies to future therapeutic targets for personalized medicine","volume":"2","author":"Chaytow","year":"2021","journal-title":"Cell Rep. Med."},{"key":"10.1016\/j.arr.2026.103133_bib19","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abg4910","article-title":"Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing","volume":"7","author":"Chemello","year":"2021","journal-title":"Sci. Adv."},{"key":"10.1016\/j.arr.2026.103133_bib20","doi-asserted-by":"crossref","first-page":"741","DOI":"10.1038\/nrm2777","article-title":"Mechanisms of alternative splicing regulation: insights from molecular and genomics approaches","volume":"10","author":"Chen","year":"2009","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.arr.2026.103133_bib21","doi-asserted-by":"crossref","first-page":"708","DOI":"10.1016\/j.biopha.2017.01.104","article-title":"Securinine enhances SMN2 exon 7 inclusion in spinal muscular atrophy cells","volume":"88","author":"Chen","year":"2017","journal-title":"Biomed. Pharm."},{"key":"10.1016\/j.arr.2026.103133_bib22","doi-asserted-by":"crossref","first-page":"2257","DOI":"10.1007\/s11427-021-2060-x","article-title":"Modification of alternative splicing in bovine somatic cell nuclear transfer embryos using engineered CRISPR-Cas13d","volume":"65","author":"Cheng","year":"2022","journal-title":"Sci. China Life Sci."},{"key":"10.1016\/j.arr.2026.103133_bib23","doi-asserted-by":"crossref","first-page":"972","DOI":"10.1016\/j.molcel.2012.12.025","article-title":"HnRNP L and HnRNP A1 induce extended U1 snRNA interactions with an exon to repress spliceosome assembly","volume":"49","author":"Chiou","year":"2013","journal-title":"Mol. Cell"},{"key":"10.1016\/j.arr.2026.103133_bib24","doi-asserted-by":"crossref","DOI":"10.3390\/cells9122647","article-title":"U2AF65-dependent SF3B1 function in SMN alternative splicing","volume":"9","author":"Choi","year":"2020","journal-title":"Cells"},{"key":"10.1016\/j.arr.2026.103133_bib25","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1038\/s12276-023-00981-7","article-title":"The implications of alternative pre-mRNA splicing in cell signal transduction","volume":"55","author":"Choi","year":"2023","journal-title":"Exp. Mol. Med."},{"key":"10.1016\/j.arr.2026.103133_bib26","doi-asserted-by":"crossref","first-page":"15383","DOI":"10.3390\/ijms232315383","article-title":"Tau isoforms: gaining insight into MAPT alternative splicing","volume":"23","author":"Corsi","year":"2022","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib27","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1016\/j.lfs.2017.12.025","article-title":"Mechanism of intranasal drug delivery directly to the brain","volume":"195","author":"Crowe","year":"2018","journal-title":"Life Sci."},{"key":"10.1016\/j.arr.2026.103133_bib28","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1007\/s10877-014-9607-1","article-title":"Intrathecal drug delivery for chronic pain management-scope, limitations and future","volume":"29","author":"Czernicki","year":"2015","journal-title":"J. Clin. Monit. Comput."},{"key":"10.1016\/j.arr.2026.103133_bib29","doi-asserted-by":"crossref","first-page":"1853","DOI":"10.1007\/s40265-020-01410-z","article-title":"Risdiplam: first approval","volume":"80","author":"Dhillon","year":"2020","journal-title":"Drugs"},{"key":"10.1016\/j.arr.2026.103133_bib30","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1016\/j.neurobiolaging.2010.11.021","article-title":"Regulation of alternative splicing of tau exon 10 by 9G8 and Dyrk1A","volume":"33","author":"Ding","year":"2012","journal-title":"Neurobiol. Aging"},{"key":"10.1016\/j.arr.2026.103133_bib31","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1177\/1087057107302676","article-title":"Identification of tau stem loop RNA stabilizers","volume":"12","author":"Donahue","year":"2007","journal-title":"J. Biomol. Screen."},{"key":"10.1016\/j.arr.2026.103133_bib32","doi-asserted-by":"crossref","DOI":"10.1016\/j.ejphar.2025.177644","article-title":"Antisense oligonucleotides: a promising advancement in neurodegenerative disease treatment","volume":"999","author":"Dudzisz","year":"2025","journal-title":"Eur. J. Pharm."},{"key":"10.1016\/j.arr.2026.103133_bib33","doi-asserted-by":"crossref","first-page":"2009","DOI":"10.1093\/brain\/awab087","article-title":"Huntington\u2019s disease-specific mis-splicing unveils key effector genes and altered splicing factors","volume":"144","author":"Elorza","year":"2021","journal-title":"Brain"},{"key":"10.1016\/j.arr.2026.103133_bib34","doi-asserted-by":"crossref","first-page":"193","DOI":"10.1186\/s40478-024-01901-0","article-title":"Paradoxical attenuation of early amyloid-induced cognitive impairment and synaptic plasticity in an aged APP\/tau bigenic rat model","volume":"12","author":"Emmerson","year":"2024","journal-title":"Acta Neuropathol. Commun."},{"key":"10.1016\/j.arr.2026.103133_bib35","doi-asserted-by":"crossref","first-page":"881","DOI":"10.1038\/nm.3617","article-title":"Huntington\u2019s disease is a four-repeat tauopathy with tau nuclear rods","volume":"20","author":"Fern\u00e1ndez-Nogales","year":"2014","journal-title":"Nat. Med."},{"key":"10.1016\/j.arr.2026.103133_bib36","doi-asserted-by":"crossref","first-page":"653","DOI":"10.3727\/096368912X655154","article-title":"Aberrant alternative splicing events in Parkinson\u2019s disease","volume":"22","author":"Fu","year":"2013","journal-title":"Cell Transpl."},{"key":"10.1016\/j.arr.2026.103133_bib37","doi-asserted-by":"crossref","first-page":"1725","DOI":"10.1073\/pnas.89.5.1725","article-title":"The 35-kDa mammalian splicing factor SC35 mediates specific interactions between U1 and U2 small nuclear ribonucleoprotein particles at the 3\u2019 splice site","volume":"89","author":"Fu","year":"1992","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.arr.2026.103133_bib38","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2024.1432659","article-title":"The role of the gut microbiota in neurodegenerative diseases targeting metabolism","volume":"18","author":"Fu","year":"2024","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib39","doi-asserted-by":"crossref","first-page":"1068","DOI":"10.1038\/s41467-024-55607-x","article-title":"Splicing accuracy varies across human introns, tissues, age and disease","volume":"16","author":"Garc\u00eda-Ruiz","year":"2025","journal-title":"Nat. Commun."},{"key":"10.1016\/j.arr.2026.103133_bib40","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1016\/0092-8674(90)90236-8","article-title":"A protein factor, ASF, controls cell-specific alternative splicing of SV40 early pre-mRNA in vitro","volume":"62","author":"Ge","year":"1990","journal-title":"Cell"},{"key":"10.1016\/j.arr.2026.103133_bib41","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1111\/j.1471-4159.2005.03552.x","article-title":"The alternative splicing of tau exon 10 and its regulatory proteins CLK2 and TRA2-BETA1 changes in sporadic Alzheimer\u2019s disease","volume":"96","author":"Glatz","year":"2006","journal-title":"J. Neurochem."},{"key":"10.1016\/j.arr.2026.103133_bib42","doi-asserted-by":"crossref","first-page":"474","DOI":"10.1261\/rna.041376.113","article-title":"Phosphorylation of SRSF1 by SRPK1 regulates alternative splicing of tumor-related Rac1b in colorectal cells","volume":"20","author":"Gon\u00e7alves","year":"2014","journal-title":"RNA"},{"key":"10.1016\/j.arr.2026.103133_bib43","doi-asserted-by":"crossref","DOI":"10.1016\/j.ebiom.2021.103691","article-title":"Antisense oligonucleotides for Alzheimer\u2019s disease therapy: from the mRNA to miRNA paradigm","volume":"74","author":"Grabowska-Pyrzewicz","year":"2021","journal-title":"eBioMedicine"},{"key":"10.1016\/j.arr.2026.103133_bib44","doi-asserted-by":"crossref","first-page":"2239","DOI":"10.1016\/j.febslet.2012.05.046","article-title":"Cyclic AMP-dependent protein kinase regulates 9G8-mediated alternative splicing of Tau exon 10","volume":"586","author":"Gu","year":"2012","journal-title":"FEBS Lett."},{"key":"10.1016\/j.arr.2026.103133_bib45","doi-asserted-by":"crossref","first-page":"1760","DOI":"10.1261\/rna.2142810","article-title":"Functional implications of the emergence of alternative splicing in hnRNP A\/B transcripts","volume":"16","author":"Han","year":"2010","journal-title":"RNA"},{"key":"10.1016\/j.arr.2026.103133_bib46","doi-asserted-by":"crossref","first-page":"1048","DOI":"10.1080\/15476286.2021.1874161","article-title":"Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing","volume":"18","author":"Hanson","year":"2021","journal-title":"RNA Biol."},{"key":"10.1016\/j.arr.2026.103133_bib47","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1126\/science.1072994","article-title":"The amyloid hypothesis of Alzheimer\u2019s disease: progress and problems on the road to therapeutics","volume":"297","author":"Hardy","year":"2002","journal-title":"Science"},{"key":"10.1016\/j.arr.2026.103133_bib48","doi-asserted-by":"crossref","first-page":"6549","DOI":"10.1093\/nar\/gkw533","article-title":"Splice-switching antisense oligonucleotides as therapeutic drugs","volume":"44","author":"Havens","year":"2016","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.arr.2026.103133_bib49","doi-asserted-by":"crossref","DOI":"10.1016\/j.biopha.2024.117332","article-title":"APE1: a critical focus in neurodegenerative conditions","volume":"179","author":"He","year":"2024","journal-title":"Biomed. Pharm."},{"key":"10.1016\/j.arr.2026.103133_bib50","doi-asserted-by":"crossref","first-page":"1289","DOI":"10.1126\/science.1198830","article-title":"Ordered and dynamic assembly of single spliceosomes","volume":"331","author":"Hoskins","year":"2011","journal-title":"Science"},{"key":"10.1016\/j.arr.2026.103133_bib51","doi-asserted-by":"crossref","first-page":"937","DOI":"10.1038\/nsmb1149","article-title":"An exonic splicing silencer represses spliceosome assembly after ATP-dependent exon recognition","volume":"13","author":"House","year":"2006","journal-title":"Nat. Struct. Mol. Biol."},{"key":"10.1016\/j.arr.2026.103133_bib52","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1007\/s00401-006-0095-3","article-title":"No alteration in tau exon 10 alternative splicing in tangle-bearing neurons of the Alzheimer\u2019s disease brain","volume":"112","author":"Ingelsson","year":"2006","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.arr.2026.103133_bib53","doi-asserted-by":"crossref","first-page":"4857","DOI":"10.1038\/s41598-020-61676-x","article-title":"Extensive splicing changes in an ALS\/FTD transgenic mouse model overexpressing cytoplasmic fused in sarcoma","volume":"10","author":"Ito","year":"2020","journal-title":"Sci. Rep."},{"key":"10.1016\/j.arr.2026.103133_bib54","doi-asserted-by":"crossref","first-page":"2983","DOI":"10.1093\/brain\/aww224","article-title":"TDP-43 stage, mixed pathologies, and clinical Alzheimer\u2019s-type dementia","volume":"139","author":"James","year":"2016","journal-title":"Brain"},{"key":"10.1016\/j.arr.2026.103133_bib55","doi-asserted-by":"crossref","first-page":"2112","DOI":"10.1002\/med.22036","article-title":"The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution","volume":"44","author":"Kachanov","year":"2024","journal-title":"Med. Res. Rev."},{"key":"10.1016\/j.arr.2026.103133_bib56","doi-asserted-by":"crossref","first-page":"125","DOI":"10.1093\/nar\/gkl924","article-title":"Different levels of alternative splicing among eukaryotes","volume":"35","author":"Kim","year":"2007","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.arr.2026.103133_bib57","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1016\/j.ultrasmedbio.2015.10.009","article-title":"Safety validation of repeated blood-brain barrier disruption using focused ultrasound","volume":"42","author":"Kobus","year":"2016","journal-title":"Ultrasound Med. Biol."},{"key":"10.1016\/j.arr.2026.103133_bib58","doi-asserted-by":"crossref","first-page":"313","DOI":"10.31662\/jmaj.2024-0038","article-title":"Abnormal splicing events due to loss of nuclear function of TDP-43: pathophysiology and perspectives","volume":"7","author":"Koike","year":"2024","journal-title":"JMA J."},{"key":"10.1016\/j.arr.2026.103133_bib59","doi-asserted-by":"crossref","first-page":"3259","DOI":"10.1093\/hmg\/ddt180","article-title":"Altered splicing of ATP6AP2 causes X-linked Parkinsonism with spasticity (XPDS)","volume":"22","author":"Korvatska","year":"2013","journal-title":"Hum. Mol. Genet"},{"key":"10.1016\/j.arr.2026.103133_bib60","doi-asserted-by":"crossref","DOI":"10.1016\/j.isci.2025.112380","article-title":"RNA splicing modulator for Huntington\u2019s disease treatment induces peripheral neuropathy","volume":"28","author":"Krach","year":"2025","journal-title":"iScience"},{"key":"10.1016\/j.arr.2026.103133_bib61","doi-asserted-by":"crossref","first-page":"1158","DOI":"10.1101\/gad.4.7.1158","article-title":"Purification and characterization of pre-mRNA splicing factor SF2 from HeLa cells","volume":"4","author":"Krainer","year":"1990","journal-title":"Genes Dev."},{"key":"10.1016\/j.arr.2026.103133_bib62","doi-asserted-by":"crossref","first-page":"10558","DOI":"10.1093\/nar\/gkab788","article-title":"Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors","volume":"49","author":"Krishnamurthy","year":"2021","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.arr.2026.103133_bib63","doi-asserted-by":"crossref","first-page":"378","DOI":"10.1016\/j.tibs.2013.05.003","article-title":"Huntington\u2019s disease: underlying molecular mechanisms and emerging concepts","volume":"38","author":"Labbadia","year":"2013","journal-title":"Trends Biochem. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib64","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1523\/JNEUROSCI.2305-16.2016","article-title":"Tau isoforms imbalance impairs the axonal transport of the amyloid precursor protein in human neurons","volume":"37","author":"Lacovich","year":"2017","journal-title":"J. Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib65","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1074\/jbc.M107045200","article-title":"Membrane-bound alpha-synuclein has a high aggregation propensity and the ability to seed the aggregation of the cytosolic form","volume":"277","author":"Lee","year":"2002","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.arr.2026.103133_bib66","doi-asserted-by":"crossref","DOI":"10.3390\/genes12091346","article-title":"Alternative splicing role in new therapies of spinal muscular atrophy","volume":"12","author":"Lejman","year":"2021","journal-title":"Genes"},{"key":"10.1016\/j.arr.2026.103133_bib67","doi-asserted-by":"crossref","first-page":"9367","DOI":"10.1523\/JNEUROSCI.0849-05.2005","article-title":"NMDA receptor activation inhibits \u03b1-secretase and promotes neuronal amyloid-\u03b2 production","volume":"25","author":"Lesn\u00e9","year":"2005","journal-title":"J. Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib68","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1186\/s40035-021-00240-7","article-title":"Neurodegenerative diseases: a hotbed for splicing defects and the potential therapies","volume":"10","author":"Li","year":"2021","journal-title":"Transl. Neurodegener."},{"key":"10.1016\/j.arr.2026.103133_bib69","doi-asserted-by":"crossref","first-page":"3454","DOI":"10.1093\/hmg\/ddw187","article-title":"Transcriptome sequencing reveals aberrant alternative splicing in Huntington\u2019s disease","volume":"25","author":"Lin","year":"2016","journal-title":"Hum. Mol. Genet"},{"key":"10.1016\/j.arr.2026.103133_bib70","doi-asserted-by":"crossref","first-page":"650","DOI":"10.1126\/science.aab0983","article-title":"TDP-43 repression of nonconserved cryptic exons is compromised in ALS-FTD","volume":"349","author":"Ling","year":"2015","journal-title":"Science"},{"key":"10.1016\/j.arr.2026.103133_bib71","doi-asserted-by":"crossref","first-page":"2616","DOI":"10.7150\/thno.83920","article-title":"The interplay between non-coding RNAs and alternative splicing: from regulatory mechanism to therapeutic implications in cancer","volume":"13","author":"Liu","year":"2023","journal-title":"Theranostics"},{"key":"10.1016\/j.arr.2026.103133_bib72","doi-asserted-by":"crossref","first-page":"1427","DOI":"10.3324\/haematol.2020.278238","article-title":"Correction of RNA splicing defect in \u03b2654-thalassemia mice using CRISPR\/Cas9 gene-editing technology","volume":"107","author":"Lu","year":"2022","journal-title":"Haematologica"},{"key":"10.1016\/j.arr.2026.103133_bib73","doi-asserted-by":"crossref","first-page":"825","DOI":"10.1089\/hum.2023.202","article-title":"Gene editing of the endogenous cryptic 3\u2019 splice site corrects the RNA splicing defect in the \u03b2654-thalassemia mouse model","volume":"35","author":"Lu","year":"2024","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.arr.2026.103133_bib74","doi-asserted-by":"crossref","first-page":"3113","DOI":"10.3390\/ijms26073113","article-title":"Cannabinerol restores mRNA splicing defects induced by \u03b2-amyloid in an in vitro model of Alzheimer\u2019s disease: a transcriptomic study","volume":"26","author":"Lui","year":"2025","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib75","doi-asserted-by":"crossref","first-page":"124","DOI":"10.1038\/s41586-022-04424-7","article-title":"TDP-43 represses cryptic exon inclusion in the FTD-ALS gene UNC13A","volume":"603","author":"Ma","year":"2022","journal-title":"Nature"},{"key":"10.1016\/j.arr.2026.103133_bib76","doi-asserted-by":"crossref","first-page":"1070","DOI":"10.3390\/ph17081070","article-title":"Advances in intrathecal nanoparticle delivery: targeting the blood-cerebrospinal fluid barrier for enhanced CNS drug delivery","volume":"17","author":"Madadi","year":"2024","journal-title":"Pharmaceuticals"},{"key":"10.1016\/j.arr.2026.103133_bib77","doi-asserted-by":"crossref","first-page":"1073","DOI":"10.1101\/gad.1934910","article-title":"A rational nomenclature for serine\/arginine-rich protein splicing factors (SR proteins)","volume":"24","author":"Manley","year":"2010","journal-title":"Genes Dev."},{"key":"10.1016\/j.arr.2026.103133_bib78","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pbio.0040021","article-title":"Intronic binding sites for hnRNP A\/B and hnRNP F\/H proteins stimulate pre-mRNA splicing","volume":"4","author":"Martinez-Contreras","year":"2006","journal-title":"PLOS Biol."},{"key":"10.1016\/j.arr.2026.103133_bib79","doi-asserted-by":"crossref","first-page":"713","DOI":"10.1086\/301768","article-title":"A BRCA1 nonsense mutation causes exon skipping","volume":"62","author":"Mazoyer","year":"1998","journal-title":"Am. J. Hum. Genet"},{"key":"10.1016\/j.arr.2026.103133_bib80","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1186\/s13024-023-00608-5","article-title":"The era of cryptic exons: implications for ALS-FTD","volume":"18","author":"Mehta","year":"2023","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.arr.2026.103133_bib81","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1038\/s41593-018-0293-z","article-title":"Premature polyadenylation-mediated loss of stathmin-2 is a hallmark of TDP-43-dependent neurodegeneration","volume":"22","author":"Melamed","year":"2019","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib82","doi-asserted-by":"crossref","first-page":"1307","DOI":"10.1038\/s41598-017-01510-z","article-title":"The pathogenic exon 1 HTT protein is produced by incomplete splicing in Huntington\u2019s disease patients","volume":"7","author":"Neueder","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.arr.2026.103133_bib83","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1038\/s41593-024-01850-w","article-title":"Aberrant splicing in Huntington\u2019s disease accompanies disrupted TDP-43 activity and altered m6A RNA modification","volume":"28","author":"Nguyen","year":"2025","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib84","doi-asserted-by":"crossref","first-page":"457","DOI":"10.1038\/s41583-023-00717-6","article-title":"Alternative splicing in neurodegenerative disease and the promise of RNA therapies","volume":"24","author":"Nikom","year":"2023","journal-title":"Nat. Rev. Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib85","doi-asserted-by":"crossref","first-page":"1147","DOI":"10.1002\/bies.10394","article-title":"The spliceosome: the most complex macromolecular machine in the cell?","volume":"25","author":"Nilsen","year":"2003","journal-title":"BioEssays"},{"key":"10.1016\/j.arr.2026.103133_bib86","doi-asserted-by":"crossref","first-page":"14794","DOI":"10.3390\/ijms241914794","article-title":"Amyloid precursor protein and Alzheimer\u2019s disease","volume":"24","author":"Orobets","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib87","doi-asserted-by":"crossref","first-page":"898","DOI":"10.1126\/science.6206566","article-title":"Lariat RNA\u2019s as intermediates and products in the splicing of messenger RNA precursors","volume":"225","author":"Padgett","year":"1984","journal-title":"Science"},{"key":"10.1016\/j.arr.2026.103133_bib88","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1177\/13872877251359633","article-title":"Alternative splicing in Alzheimer\u2019s disease: mechanisms, therapeutic implications, and 3D modeling approaches","volume":"107","author":"Pasteris","year":"2025","journal-title":"J. Alzheimers Dis."},{"key":"10.1016\/j.arr.2026.103133_bib89","doi-asserted-by":"crossref","DOI":"10.1016\/j.bbagrm.2022.194875","article-title":"Position-dependent effects of hnRNP A1\/A2 in SMN1\/2 exon7 splicing","volume":"1865","author":"Qiu","year":"2022","journal-title":"Biochim. Biophys. Acta Gene Regul. Mech."},{"key":"10.1016\/j.arr.2026.103133_bib90","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2024.106456","article-title":"Blood and cerebellar abundance of ATXN3 splice variants in spinocerebellar ataxia type 3\/Machado-Joseph disease","volume":"193","author":"Raposo","year":"2024","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.arr.2026.103133_bib91","doi-asserted-by":"crossref","first-page":"1655","DOI":"10.1038\/s41388-024-03036-x","article-title":"Alternative splicing in prostate cancer progression and therapeutic resistance","volume":"43","author":"Rawat","year":"2024","journal-title":"Oncogene"},{"key":"10.1016\/j.arr.2026.103133_bib92","first-page":"1081","article-title":"Cytoplasmic inclusions of TDP-43 in neurodegenerative diseases: a potential role for caspases","volume":"24","author":"Rohn","year":"2009","journal-title":"Histol. Histopathol."},{"key":"10.1016\/j.arr.2026.103133_bib93","doi-asserted-by":"crossref","first-page":"1262","DOI":"10.1136\/jnnp-2022-329675","article-title":"Neurodegenerative disease risk among former international rugby union players","volume":"93","author":"Russell","year":"2022","journal-title":"J. Neurol. Neurosurg. Psychiatry"},{"key":"10.1016\/j.arr.2026.103133_bib94","doi-asserted-by":"crossref","first-page":"2366","DOI":"10.1073\/pnas.1221891110","article-title":"Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington\u2019s disease","volume":"110","author":"Sathasivam","year":"2013","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.arr.2026.103133_bib95","doi-asserted-by":"crossref","first-page":"5300","DOI":"10.1007\/s12325-025-03337-x","article-title":"Emerging concepts in diagnosis, pathologic features, and treatment of limbic-predominant amnestic neurodegenerative syndrome (LANS): a narrative review","volume":"42","author":"Serio","year":"2025","journal-title":"Adv. Ther."},{"key":"10.1016\/j.arr.2026.103133_bib96","doi-asserted-by":"crossref","first-page":"6171","DOI":"10.1038\/ncomms7171","article-title":"ALS-causative mutations in FUS\/TLS confer gain and loss of function by altered association with SMN and U1-snRNP","volume":"6","author":"Sun","year":"2015","journal-title":"Nat. Commun."},{"key":"10.1016\/j.arr.2026.103133_bib97","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1007\/s10048-005-0001-5","article-title":"Differential expression of splice variant and wild-type parkin in sporadic Parkinson\u2019s disease","volume":"6","author":"Tan","year":"2005","journal-title":"Neurogenetics"},{"key":"10.1016\/j.arr.2026.103133_bib98","doi-asserted-by":"crossref","DOI":"10.1016\/j.ebiom.2023.104720","article-title":"Widespread dysregulation of mRNA splicing implicates RNA processing in the development and progression of Huntington\u2019s disease","volume":"94","author":"Tano","year":"2023","journal-title":"eBioMedicine"},{"key":"10.1016\/j.arr.2026.103133_bib99","doi-asserted-by":"crossref","first-page":"45","DOI":"10.1007\/s00401-024-02780-4","article-title":"TDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhibition","volume":"148","author":"Torres","year":"2024","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.arr.2026.103133_bib100","doi-asserted-by":"crossref","first-page":"925","DOI":"10.1016\/j.molcel.2010.08.011","article-title":"The nuclear-retained noncoding RNA MALAT1 regulates alternative splicing by modulating SR splicing factor phosphorylation","volume":"39","author":"Tripathi","year":"2010","journal-title":"Mol. Cell"},{"key":"10.1016\/j.arr.2026.103133_bib101","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1016\/j.molcel.2019.09.017","article-title":"Alternative splicing regulatory networks: functions, mechanisms, and evolution","volume":"76","author":"Ule","year":"2019","journal-title":"Mol. Cell"},{"key":"10.1016\/j.arr.2026.103133_bib102","doi-asserted-by":"crossref","first-page":"1045","DOI":"10.1101\/gad.316059.118","article-title":"Dysregulation of Mdm2 and Mdm4 alternative splicing underlies motor neuron death in spinal muscular atrophy","volume":"32","author":"Van Alstyne","year":"2018","journal-title":"Genes Dev."},{"key":"10.1016\/j.arr.2026.103133_bib103","doi-asserted-by":"crossref","first-page":"421","DOI":"10.1038\/gim.2015.117","article-title":"The genetic landscape of Alzheimer disease: clinical implications and perspectives","volume":"18","author":"Van Cauwenberghe","year":"2016","journal-title":"Genet. Med."},{"key":"10.1016\/j.arr.2026.103133_bib104","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1097\/00005072-198511000-00003","article-title":"Neuropathological classification of Huntington\u2019s disease","volume":"44","author":"Vonsattel","year":"1985","journal-title":"J. Neuropathol. Exp. Neurol."},{"key":"10.1016\/j.arr.2026.103133_bib105","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1038\/s41531-025-01046-4","article-title":"Impact of motor symptom asymmetry on non-motor outcomes in Parkinson\u2019s disease: a systematic review","volume":"11","author":"Voruz","year":"2025","journal-title":"npj Park. \u2019S. Dis."},{"key":"10.1016\/j.arr.2026.103133_bib106","doi-asserted-by":"crossref","first-page":"701","DOI":"10.1016\/j.cell.2009.02.009","article-title":"The spliceosome: design principles of a dynamic RNP machine","volume":"136","author":"Wahl","year":"2009","journal-title":"Cell"},{"key":"10.1016\/j.arr.2026.103133_bib107","doi-asserted-by":"crossref","first-page":"16259","DOI":"10.3390\/ijms242216259","article-title":"Potential implications of miRNAs in the pathogenesis, diagnosis, and therapeutics of Alzheimer\u2019s disease","volume":"24","author":"Wang","year":"2023","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib108","doi-asserted-by":"crossref","first-page":"a003707","DOI":"10.1101\/cshperspect.a003707","article-title":"Spliceosome structure and function","volume":"3","author":"Will","year":"2011","journal-title":"Cold Spring Harb. Perspect. Biol."},{"key":"10.1016\/j.arr.2026.103133_bib109","doi-asserted-by":"crossref","DOI":"10.1177\/1756286418776932","article-title":"Antisense oligonucleotides in neurological disorders","volume":"11","author":"Wurster","year":"2018","journal-title":"Ther. Adv. Neurol. Disord."},{"key":"10.1016\/j.arr.2026.103133_bib110","doi-asserted-by":"crossref","DOI":"10.1093\/nar\/gkaf347","article-title":"PRMT5 promotes full-length HTT expression by repressing multiple proximal intronic polyadenylation sites","volume":"53","author":"Yadav","year":"2025","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.arr.2026.103133_bib111","article-title":"Cryptic splicing of GAP43 mRNA is a novel hallmark of TDP-43-associated ALS and AD","volume":"12","author":"Yang","year":"2025","journal-title":"Adv. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib112","doi-asserted-by":"crossref","first-page":"2634","DOI":"10.1002\/med.21705","article-title":"Modern approaches in gene therapy of motor neuron diseases","volume":"41","author":"Zakharova","year":"2021","journal-title":"Med. Res. Rev."},{"key":"10.1016\/j.arr.2026.103133_bib113","doi-asserted-by":"crossref","first-page":"5948","DOI":"10.3390\/ijms23115948","article-title":"Correction of beta-thalassemia IVS-II-654 mutation in a mouse model using prime editing","volume":"23","author":"Zhang","year":"2022","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.arr.2026.103133_bib114","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1186\/1750-1326-1-5","article-title":"The amyloid precursor protein: beyond amyloid","volume":"1","author":"Zheng","year":"2006","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.arr.2026.103133_bib115","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1186\/1750-1326-6-27","article-title":"Biology and pathophysiology of the amyloid precursor protein","volume":"6","author":"Zheng","year":"2011","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.arr.2026.103133_bib116","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1007\/BF02254432","article-title":"Regulation of alternative RNA splicing by exon definition and exon sequences in viral and mammalian gene expression","volume":"11","author":"Zheng","year":"2004","journal-title":"J. Biomed. Sci."},{"issue":"2","key":"10.1016\/j.arr.2026.103133_bib117","article-title":"Alternative splicing of exon 10 in the Tau gene as a target for treatment of tauopathies","volume":"9","author":"Zhou","year":"2008","journal-title":"BMC Neurosci."},{"key":"10.1016\/j.arr.2026.103133_bib118","doi-asserted-by":"crossref","DOI":"10.1016\/j.phrs.2025.107977","article-title":"TDP-43 in Alzheimer\u2019s disease: pathophysiology and therapeutic strategies","volume":"221","author":"Zhou","year":"2025","journal-title":"Pharmacol. Res."},{"key":"10.1016\/j.arr.2026.103133_bib119","doi-asserted-by":"crossref","first-page":"1351","DOI":"10.1016\/S1097-2765(01)00409-9","article-title":"Exon identity established through differential antagonism between exonic splicing silencer-bound hnRNP A1 and enhancer-bound SR proteins","volume":"8","author":"Zhu","year":"2001","journal-title":"Mol. Cell"}],"container-title":["Ageing Research Reviews"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S156816372600125X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S156816372600125X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T05:02:24Z","timestamp":1778821344000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S156816372600125X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,6]]},"references-count":119,"alternative-id":["S156816372600125X"],"URL":"https:\/\/doi.org\/10.1016\/j.arr.2026.103133","relation":{},"ISSN":["1568-1637"],"issn-type":[{"value":"1568-1637","type":"print"}],"subject":[],"published":{"date-parts":[[2026,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Mechanistic research and therapeutic prospects of alternative splicing in neurodegenerative diseases","name":"articletitle","label":"Article Title"},{"value":"Ageing Research Reviews","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.arr.2026.103133","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2026 Elsevier B.V. All rights are reserved, including those for text and data mining, AI training, and similar technologies.","name":"copyright","label":"Copyright"}],"article-number":"103133"}}