{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T14:55:03Z","timestamp":1790952903358,"version":"4.1.0"},"reference-count":131,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,12,10]],"date-time":"2025-12-10T00:00:00Z","timestamp":1765324800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006108","name":"National Center for Advancing Translational Sciences","doi-asserted-by":"publisher","award":["UL1TR001878"],"award-info":[{"award-number":["UL1TR001878"]}],"id":[{"id":"10.13039\/100006108","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000049","name":"NIA","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000049","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006438","name":"Office of the Assistant Secretary for Health","doi-asserted-by":"publisher","award":["W81XWH-20-1-0242"],"award-info":[{"award-number":["W81XWH-20-1-0242"]}],"id":[{"id":"10.13039\/100006438","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002565","name":"Alzheimer's Drug Discovery Foundation","doi-asserted-by":"publisher","award":["R01NS12718"],"award-info":[{"award-number":["R01NS12718"]}],"id":[{"id":"10.13039\/100002565","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002565","name":"Alzheimer's Drug Discovery Foundation","doi-asserted-by":"publisher","award":["R21NS13367"],"award-info":[{"award-number":["R21NS13367"]}],"id":[{"id":"10.13039\/100002565","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002565","name":"Alzheimer's Drug Discovery Foundation","doi-asserted-by":"publisher","award":["R21AG064940"],"award-info":[{"award-number":["R21AG064940"]}],"id":[{"id":"10.13039\/100002565","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002565","name":"Alzheimer's Drug Discovery Foundation","doi-asserted-by":"publisher","award":["RF1AG09091"],"award-info":[{"award-number":["RF1AG09091"]}],"id":[{"id":"10.13039\/100002565","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002565","name":"Alzheimer's Drug Discovery Foundation","doi-asserted-by":"publisher","award":["R01NS105756"],"award-info":[{"award-number":["R01NS105756"]}],"id":[{"id":"10.13039\/100002565","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100002163","name":"Johnson Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100002163","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000971","name":"ALS Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000971","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100003607","name":"Association for Frontotemporal Degeneration","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100003607","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100008590","name":"Dr. Ralph and Marian Falk Medical Research Trust","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100008590","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000957","name":"Alzheimer's Association","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000957","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["RF1NS121143"],"award-info":[{"award-number":["RF1NS121143"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R35GM138109"],"award-info":[{"award-number":["R35GM138109"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000001","name":"National Science Foundation","doi-asserted-by":"publisher","award":["R01NS116176"],"award-info":[{"award-number":["R01NS116176"]}],"id":[{"id":"10.13039\/100000001","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000001","name":"National Science Foundation","doi-asserted-by":"publisher","award":["R01GM147677"],"award-info":[{"award-number":["R01GM147677"]}],"id":[{"id":"10.13039\/100000001","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007929","name":"Institute for Translational Medicine and Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100007929","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004325","name":"AstraZeneca","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004325","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Cell"],"published-print":{"date-parts":[[2026,1]]},"DOI":"10.1016\/j.molcel.2025.12.009","type":"journal-article","created":{"date-parts":[[2026,1,8]],"date-time":"2026-01-08T16:24:39Z","timestamp":1767889479000},"page":"114-134.e10","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":13,"title":["Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains"],"prefix":"10.1016","volume":"86","author":[{"given":"Lin","family":"Guo","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jacob R.","family":"Mann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jocelyn C.","family":"Mauna","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katie E.","family":"Copley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hejia","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jack D.","family":"Rubien","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cristian A.","family":"Bergmann","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jenny L.","family":"Carey","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Merjane","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marilyn","family":"Ngo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jiazhen","family":"Xu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hana M.","family":"Odeh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"JiaBei","family":"Lin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bo Lim","family":"Lee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Ganser","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emma","family":"Robinson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin M.","family":"Kim","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anastasia C.","family":"Murthy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tapas","family":"Paul","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bede","family":"Portz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amanda M.","family":"Gleixner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zamia","family":"Diaz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ashleigh","family":"Smirnov","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"George","family":"Padilla","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ellen","family":"Lavorando","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carolann","family":"Espy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yulei","family":"Shang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric J.","family":"Huang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandra","family":"Chesi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolas L.","family":"Fawzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sua","family":"Myong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher J.","family":"Donnelly","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5269-8533","authenticated-orcid":false,"given":"James","family":"Shorter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.molcel.2025.12.009_bib1","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1016\/j.tibs.2020.12.005","article-title":"FUS and TDP-43 Phases in Health and Disease","volume":"46","author":"Portz","year":"2021","journal-title":"Trends Biochem. Sci."},{"key":"10.1016\/j.molcel.2025.12.009_bib2","doi-asserted-by":"crossref","first-page":"1069","DOI":"10.1111\/j.1750-3639.2010.00413.x","article-title":"Extensive FUS-immunoreactive pathology in juvenile amyotrophic lateral sclerosis with basophilic inclusions","volume":"20","author":"Huang","year":"2010","journal-title":"Brain Pathol."},{"key":"10.1016\/j.molcel.2025.12.009_bib3","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1038\/s41586-021-04199-3","article-title":"Structure of pathological TDP-43 filaments from ALS with FTLD","volume":"601","author":"Arseni","year":"2022","journal-title":"Nature"},{"key":"10.1016\/j.molcel.2025.12.009_bib4","doi-asserted-by":"crossref","first-page":"130","DOI":"10.1126\/science.1134108","article-title":"Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis","volume":"314","author":"Neumann","year":"2006","journal-title":"Science"},{"key":"10.1016\/j.molcel.2025.12.009_bib5","doi-asserted-by":"crossref","first-page":"1208","DOI":"10.1126\/science.1165942","article-title":"Mutations in FUS, an RNA processing protein, cause familial amyotrophic lateral sclerosis type 6","volume":"323","author":"Vance","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.molcel.2025.12.009_bib6","doi-asserted-by":"crossref","first-page":"1205","DOI":"10.1126\/science.1166066","article-title":"Mutations in the FUS\/TLS gene on chromosome 16 cause familial amyotrophic lateral sclerosis","volume":"323","author":"Kwiatkowski","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.molcel.2025.12.009_bib7","doi-asserted-by":"crossref","first-page":"898","DOI":"10.1038\/s41586-023-06405-w","article-title":"TDP-43 forms amyloid filaments with a distinct fold in type A FTLD-TDP","volume":"620","author":"Arseni","year":"2023","journal-title":"Nature"},{"key":"10.1016\/j.molcel.2025.12.009_bib8","article-title":"FUS Mislocalization Rewires a Cortical Gene Network to Drive Cognitive and Behavioral Impairment in ALS","author":"Cassel","year":"2025","journal-title":"medRxiv"},{"key":"10.1016\/j.molcel.2025.12.009_bib9","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1016\/j.neuron.2019.02.038","article-title":"Cytoplasmic TDP-43 De-mixing Independent of Stress Granules Drives Inhibition of Nuclear Import, Loss of Nuclear TDP-43, and Cell Death","volume":"102","author":"Gasset-Rosa","year":"2019","journal-title":"Neuron"},{"key":"10.1016\/j.molcel.2025.12.009_bib10","doi-asserted-by":"crossref","DOI":"10.1186\/s13024-024-00737-5","article-title":"Frontotemporal dementia-like disease progression elicited by seeded aggregation and spread of FUS","volume":"19","author":"Vazquez-Sanchez","year":"2024","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.molcel.2025.12.009_bib11","doi-asserted-by":"crossref","first-page":"1614","DOI":"10.1016\/j.neuron.2025.03.008","article-title":"Seeded aggregation of TDP-43 induces its loss of function and reveals early pathological signatures","volume":"113","author":"Scial\u00f2","year":"2025","journal-title":"Neuron"},{"key":"10.1016\/j.molcel.2025.12.009_bib12","doi-asserted-by":"crossref","first-page":"1597","DOI":"10.1016\/j.neuron.2025.03.004","article-title":"TDP-43 seeding induces cytoplasmic aggregation heterogeneity and nuclear loss of function of TDP-43","volume":"113","author":"Rummens","year":"2025","journal-title":"Neuron"},{"key":"10.1016\/j.molcel.2025.12.009_bib13","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a024463","article-title":"TDP-43 Prions","volume":"8","author":"Nonaka","year":"2018","journal-title":"Cold Spring Harb. Perspect. Med."},{"key":"10.1016\/j.molcel.2025.12.009_bib14","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1016\/j.cell.2018.03.002","article-title":"Nuclear-Import Receptors Reverse Aberrant Phase Transitions of RNA-Binding Proteins with Prion-like Domains","volume":"173","author":"Guo","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib15","doi-asserted-by":"crossref","first-page":"20329","DOI":"10.1074\/jbc.M109.010264","article-title":"TDP-43 is intrinsically aggregation-prone, and amyotrophic lateral sclerosis-linked mutations accelerate aggregation and increase toxicity","volume":"284","author":"Johnson","year":"2009","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.molcel.2025.12.009_bib16","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pbio.1000614","article-title":"Molecular determinants and genetic modifiers of aggregation and toxicity for the ALS disease protein FUS\/TLS","volume":"9","author":"Sun","year":"2011","journal-title":"PLoS Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib17","doi-asserted-by":"crossref","first-page":"1066","DOI":"10.1016\/j.cell.2015.07.047","article-title":"A Liquid-to-Solid Phase Transition of the ALS Protein FUS Accelerated by Disease Mutation","volume":"162","author":"Patel","year":"2015","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib18","doi-asserted-by":"crossref","first-page":"4680","DOI":"10.1016\/j.cell.2021.07.018","article-title":"TDP-43 condensation properties specify its RNA-binding and regulatory repertoire","volume":"184","author":"Hallegger","year":"2021","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib19","doi-asserted-by":"crossref","DOI":"10.1083\/jcb.202008030","article-title":"FUS-dependent liquid-liquid phase separation is important for DNA repair initiation","volume":"220","author":"Levone","year":"2021","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib20","doi-asserted-by":"crossref","first-page":"945","DOI":"10.1016\/j.cell.2024.01.015","article-title":"PARP1-DNA co-condensation drives DNA repair site assembly to prevent disjunction of broken DNA ends","volume":"187","author":"Chappidi","year":"2024","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib21","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1016\/j.neuron.2019.01.048","article-title":"RNA Binding Antagonizes Neurotoxic Phase Transitions of TDP-43","volume":"102","author":"Mann","year":"2019","journal-title":"Neuron"},{"key":"10.1016\/j.molcel.2025.12.009_bib22","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.39578","article-title":"Chronic optogenetic induction of stress granules is cytotoxic and reveals the evolution of ALS-FTD pathology","volume":"8","author":"Zhang","year":"2019","journal-title":"eLife"},{"key":"10.1016\/j.molcel.2025.12.009_bib23","doi-asserted-by":"crossref","first-page":"4123","DOI":"10.1016\/j.cell.2025.04.039","article-title":"Intra-condensate demixing of TDP-43 inside stress granules generates pathological aggregates","volume":"188","author":"Yan","year":"2025","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib24","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.gde.2017.01.008","article-title":"Designer protein disaggregases to counter neurodegenerative disease","volume":"44","author":"Shorter","year":"2017","journal-title":"Curr. Opin. Genet. Dev."},{"key":"10.1016\/j.molcel.2025.12.009_bib25","doi-asserted-by":"crossref","first-page":"170","DOI":"10.1016\/j.cell.2013.11.047","article-title":"Potentiated Hsp104 variants antagonize diverse proteotoxic misfolding events","volume":"156","author":"Jackrel","year":"2014","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib26","doi-asserted-by":"crossref","DOI":"10.1186\/s13024-022-00585-1","article-title":"Nuclear import receptors are recruited by FG-nucleoporins to rescue hallmarks of TDP-43 proteinopathy","volume":"17","author":"Khalil","year":"2022","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.molcel.2025.12.009_bib27","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2024.115005","article-title":"Design principles to tailor Hsp104 therapeutics","volume":"43","author":"Lin","year":"2024","journal-title":"Cell Rep."},{"key":"10.1016\/j.molcel.2025.12.009_bib28","doi-asserted-by":"crossref","first-page":"3314","DOI":"10.1016\/j.molcel.2023.07.029","article-title":"Tuning Hsp104 specificity to selectively detoxify alpha-synuclein","volume":"83","author":"Mack","year":"2023","journal-title":"Mol. Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib29","doi-asserted-by":"crossref","first-page":"2080","DOI":"10.1016\/j.celrep.2019.07.069","article-title":"Mining Disaggregase Sequence Space to Safely Counter TDP-43, FUS, and alpha-Synuclein Proteotoxicity","volume":"28","author":"Tariq","year":"2019","journal-title":"Cell Rep."},{"key":"10.1016\/j.molcel.2025.12.009_bib30","doi-asserted-by":"crossref","DOI":"10.1093\/femsyr\/foy042","article-title":"Potentiating Hsp104 activity via phosphomimetic mutations in the middle domain","volume":"18","author":"Tariq","year":"2018","journal-title":"FEMS Yeast Res."},{"key":"10.1016\/j.molcel.2025.12.009_bib31","doi-asserted-by":"crossref","DOI":"10.1016\/j.jbc.2022.102806","article-title":"A minimal construct of nuclear-import receptor Karyopherin-beta2 defines the regions critical for chaperone and disaggregation activity","volume":"299","author":"Fare","year":"2023","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.molcel.2025.12.009_bib32","doi-asserted-by":"crossref","first-page":"1015","DOI":"10.1083\/jcb.201608022","article-title":"FUS inclusions disrupt RNA localization by sequestering kinesin-1 and inhibiting microtubule detyrosination","volume":"216","author":"Yasuda","year":"2017","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib33","first-page":"1175","article-title":"Potentiated Hsp104 variants suppress toxicity of diverse neurodegenerative disease-linked proteins","volume":"7","author":"Jackrel","year":"2014","journal-title":"Dis. Model. Mech."},{"key":"10.1016\/j.molcel.2025.12.009_bib34","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1038\/s41593-021-00969-4","article-title":"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.molcel.2025.12.009_bib35","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1038\/s41573-023-00766-7","article-title":"AAV-based in vivo gene therapy for neurological disorders","volume":"22","author":"Ling","year":"2023","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.molcel.2025.12.009_bib36","doi-asserted-by":"crossref","first-page":"327","DOI":"10.1038\/s41593-020-0589-7","article-title":"Gut-seeded \u03b1-synuclein fibrils promote gut dysfunction and brain pathology specifically in aged mice","volume":"23","author":"Challis","year":"2020","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.molcel.2025.12.009_bib37","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2025.116419","article-title":"AAVs targeting human carbonic anhydrase IV enhance gene delivery to the brain","volume":"44","author":"Lin","year":"2025","journal-title":"Cell Rep."},{"key":"10.1016\/j.molcel.2025.12.009_bib38","doi-asserted-by":"crossref","first-page":"1988","DOI":"10.1016\/j.ymthe.2025.03.057","article-title":"Current trends in gene therapy to treat inherited disorders of the brain","volume":"33","author":"Matuszek","year":"2025","journal-title":"Mol. Ther."},{"key":"10.1016\/j.molcel.2025.12.009_bib39","doi-asserted-by":"crossref","first-page":"648","DOI":"10.1097\/WCO.0000000000000594","article-title":"Emerging antisense oligonucleotide and viral therapies for amyotrophic lateral sclerosis","volume":"31","author":"Ly","year":"2018","journal-title":"Curr. Opin. Neurol."},{"key":"10.1016\/j.molcel.2025.12.009_bib40","doi-asserted-by":"crossref","DOI":"10.1172\/JCI186116","article-title":"The expanding application of antisense oligonucleotides to neurodegenerative diseases","volume":"134","author":"Sumner","year":"2024","journal-title":"J. Clin. Investig."},{"key":"10.1016\/j.molcel.2025.12.009_bib41","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1038\/s41573-020-0075-7","article-title":"Advances in oligonucleotide drug delivery","volume":"19","author":"Roberts","year":"2020","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.molcel.2025.12.009_bib42","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.molcel.2025.12.009_bib43","doi-asserted-by":"crossref","first-page":"6807","DOI":"10.1074\/jbc.M008304200","article-title":"Identification of an RNA binding specificity for the potential splicing factor TLS","volume":"276","author":"Lerga","year":"2001","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.molcel.2025.12.009_bib44","doi-asserted-by":"crossref","first-page":"1488","DOI":"10.1038\/nn.3230","article-title":"Divergent roles of ALS-linked proteins FUS\/TLS and TDP-43 intersect in processing long pre-mRNAs","volume":"15","author":"Lagier-Tourenne","year":"2012","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.molcel.2025.12.009_bib45","doi-asserted-by":"crossref","first-page":"981","DOI":"10.1172\/JCI72723","article-title":"ALS-associated mutation FUS-R521C causes DNA damage and RNA splicing defects","volume":"124","author":"Qiu","year":"2014","journal-title":"J. Clin. Investig."},{"key":"10.1016\/j.molcel.2025.12.009_bib46","doi-asserted-by":"crossref","first-page":"918","DOI":"10.1016\/j.celrep.2013.11.017","article-title":"RNA seeds higher-order assembly of FUS protein","volume":"5","author":"Schwartz","year":"2013","journal-title":"Cell Rep."},{"key":"10.1016\/j.molcel.2025.12.009_bib47","doi-asserted-by":"crossref","first-page":"36337","DOI":"10.1074\/jbc.M104236200","article-title":"Characterization and functional implications of the RNA binding properties of nuclear factor TDP-43, a novel splicing regulator of CFTR exon 9","volume":"276","author":"Buratti","year":"2001","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.molcel.2025.12.009_bib48","doi-asserted-by":"crossref","first-page":"678","DOI":"10.1016\/j.neuron.2015.10.030","article-title":"ALS\/FTD Mutation-Induced Phase Transition of FUS Liquid Droplets and Reversible Hydrogels into Irreversible Hydrogels Impairs RNP Granule Function","volume":"88","author":"Murakami","year":"2015","journal-title":"Neuron"},{"key":"10.1016\/j.molcel.2025.12.009_bib49","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1016\/j.cell.2012.04.017","article-title":"Cell-free formation of RNA granules: low complexity sequence domains form dynamic fibers within hydrogels","volume":"149","author":"Kato","year":"2012","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib50","doi-asserted-by":"crossref","first-page":"1417","DOI":"10.1042\/BCJ20160499","article-title":"RNA-binding proteins with prion-like domains in health and disease","volume":"474","author":"Harrison","year":"2017","journal-title":"Biochem. J."},{"key":"10.1016\/j.molcel.2025.12.009_bib51","doi-asserted-by":"crossref","first-page":"W70","DOI":"10.1093\/nar\/gkn188","article-title":"The Vienna RNA websuite","volume":"36","author":"Gruber","year":"2008","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.molcel.2025.12.009_bib52","article-title":"RNA G-Quadruplexes Function as a Tunable Switch of FUS Phase Separation","author":"Carey","year":"2025","journal-title":"bioRxiv"},{"key":"10.1016\/j.molcel.2025.12.009_bib53","doi-asserted-by":"crossref","first-page":"82","DOI":"10.1016\/j.molcel.2019.09.022","article-title":"Loss of Dynamic RNA Interaction and Aberrant Phase Separation Induced by Two Distinct Types of ALS\/FTD-Linked FUS Mutations","volume":"77","author":"Niaki","year":"2020","journal-title":"Mol. Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib54","doi-asserted-by":"crossref","first-page":"507","DOI":"10.1038\/nmeth.1208","article-title":"A practical guide to single-molecule FRET","volume":"5","author":"Roy","year":"2008","journal-title":"Nat. Methods"},{"key":"10.1016\/j.molcel.2025.12.009_bib55","doi-asserted-by":"crossref","first-page":"3406","DOI":"10.1093\/nar\/gkg595","article-title":"Mfold web server for nucleic acid folding and hybridization prediction","volume":"31","author":"Zuker","year":"2003","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.molcel.2025.12.009_bib56","doi-asserted-by":"crossref","first-page":"490","DOI":"10.1016\/j.molcel.2018.11.012","article-title":"The Solution Structure of FUS Bound to RNA Reveals a Bipartite Mode of RNA Recognition with Both Sequence and Shape Specificity","volume":"73","author":"Loughlin","year":"2019","journal-title":"Mol. Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib57","doi-asserted-by":"crossref","first-page":"1193","DOI":"10.1093\/hmg\/dds526","article-title":"RNA-binding ability of FUS regulates neurodegeneration, cytoplasmic mislocalization and incorporation into stress granules associated with FUS carrying ALS-linked mutations","volume":"22","author":"Daigle","year":"2013","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.molcel.2025.12.009_bib58","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.molcel.2015.09.006","article-title":"Residue-by-Residue View of In Vitro FUS Granules that Bind the C-Terminal Domain of RNA Polymerase II","volume":"60","author":"Burke","year":"2015","journal-title":"Mol. Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib59","doi-asserted-by":"crossref","first-page":"44834","DOI":"10.1074\/jbc.M408552200","article-title":"Domain architectures and characterization of an RNA-binding protein, TLS","volume":"279","author":"Iko","year":"2004","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.molcel.2025.12.009_bib60","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1016\/j.celrep.2018.06.070","article-title":"Molecular Dissection of FUS Points at Synergistic Effect of Low-Complexity Domains in Toxicity","volume":"24","author":"Bogaert","year":"2018","journal-title":"Cell Rep."},{"key":"10.1016\/j.molcel.2025.12.009_bib61","doi-asserted-by":"crossref","first-page":"918","DOI":"10.1126\/science.aar7366","article-title":"RNA buffers the phase separation behavior of prion-like RNA binding proteins","volume":"360","author":"Maharana","year":"2018","journal-title":"Science"},{"key":"10.1016\/j.molcel.2025.12.009_bib62","doi-asserted-by":"crossref","first-page":"1467","DOI":"10.1016\/j.cell.2018.10.048","article-title":"Mapping Local and Global Liquid Phase Behavior in Living Cells Using Photo-Oligomerizable Seeds","volume":"175","author":"Bracha","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib63","doi-asserted-by":"crossref","first-page":"706","DOI":"10.1016\/j.cell.2018.03.004","article-title":"Phase Separation of FUS Is Suppressed by Its Nuclear Import Receptor and Arginine Methylation","volume":"173","author":"Hofweber","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib64","doi-asserted-by":"crossref","first-page":"720","DOI":"10.1016\/j.cell.2018.03.056","article-title":"FUS Phase Separation Is Modulated by a Molecular Chaperone and Methylation of Arginine Cation-pi Interactions","volume":"173","author":"Qamar","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib65","doi-asserted-by":"crossref","first-page":"693","DOI":"10.1016\/j.cell.2018.03.003","article-title":"Nuclear Import Receptor Inhibits Phase Separation of FUS through Binding to Multiple Sites","volume":"173","author":"Yoshizawa","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib66","article-title":"Nuclear-import receptors remodel the dilute phase to suppress phase transitions of RNA-binding proteins with prion-like domains","author":"Linsenmeier","year":"2025","journal-title":"bioRxiv"},{"key":"10.1016\/j.molcel.2025.12.009_bib67","doi-asserted-by":"crossref","DOI":"10.1038\/ncomms5925","article-title":"An optimized optogenetic clustering tool for probing protein interaction and function","volume":"5","author":"Taslimi","year":"2014","journal-title":"Nat. Commun."},{"key":"10.1016\/j.molcel.2025.12.009_bib68","doi-asserted-by":"crossref","first-page":"2595","DOI":"10.1093\/brain\/awr201","article-title":"FET proteins TAF15 and EWS are selective markers that distinguish FTLD with FUS pathology from amyotrophic lateral sclerosis with FUS mutations","volume":"134","author":"Neumann","year":"2011","journal-title":"Brain"},{"key":"10.1016\/j.molcel.2025.12.009_bib69","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1007\/s00401-012-1020-6","article-title":"Transportin 1 accumulates specifically with FET proteins but no other transportin cargos in FTLD-FUS and is absent in FUS inclusions in ALS with FUS mutations","volume":"124","author":"Neumann","year":"2012","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.molcel.2025.12.009_bib70","doi-asserted-by":"crossref","first-page":"995","DOI":"10.1016\/S1474-4422(10)70195-2","article-title":"TDP-43 and FUS in amyotrophic lateral sclerosis and frontotemporal dementia","volume":"9","author":"Mackenzie","year":"2010","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.molcel.2025.12.009_bib71","doi-asserted-by":"crossref","first-page":"4258","DOI":"10.1038\/emboj.2012.261","article-title":"Arginine methylation next to the PY-NLS modulates Transportin binding and nuclear import of FUS","volume":"31","author":"Dormann","year":"2012","journal-title":"EMBO J."},{"key":"10.1016\/j.molcel.2025.12.009_bib72","doi-asserted-by":"crossref","first-page":"238","DOI":"10.1038\/nbt.3765","article-title":"The chemical evolution of oligonucleotide therapies of clinical utility","volume":"35","author":"Khvorova","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.molcel.2025.12.009_bib73","doi-asserted-by":"crossref","first-page":"2019","DOI":"10.1093\/nar\/23.11.2019","article-title":"Characterization of fully 2\u2032-modified oligoribonucleotide hetero- and homoduplex hybridization and nuclease sensitivity","volume":"23","author":"Cummins","year":"1995","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.molcel.2025.12.009_bib74","doi-asserted-by":"crossref","first-page":"14533","DOI":"10.1074\/jbc.271.24.14533","article-title":"Nuclease resistance and antisense activity of modified oligonucleotides targeted to Ha-ras","volume":"271","author":"Monia","year":"1996","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.molcel.2025.12.009_bib75","doi-asserted-by":"crossref","first-page":"919","DOI":"10.1016\/j.bbrc.2006.02.049","article-title":"Chemical modification of siRNAs to improve serum stability without loss of efficacy","volume":"342","author":"Choung","year":"2006","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.molcel.2025.12.009_bib76","doi-asserted-by":"crossref","first-page":"1663","DOI":"10.1038\/sj.mt.6300240","article-title":"2\u2032-O-methyl-modified RNAs act as TLR7 antagonists","volume":"15","author":"Robbins","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.molcel.2025.12.009_bib77","doi-asserted-by":"crossref","first-page":"2841","DOI":"10.1038\/emboj.2010.143","article-title":"ALS-associated fused in sarcoma (FUS) mutations disrupt Transportin-mediated nuclear import","volume":"29","author":"Dormann","year":"2010","journal-title":"EMBO J."},{"key":"10.1016\/j.molcel.2025.12.009_bib78","doi-asserted-by":"crossref","first-page":"1431","DOI":"10.1083\/jcb.147.7.1431","article-title":"RNA-binding proteins TIA-1 and TIAR link the phosphorylation of eIF-2 alpha to the assembly of mammalian stress granules","volume":"147","author":"Kedersha","year":"1999","journal-title":"J. Cell Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib79","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1038\/ncb1004","article-title":"Histone H2AX phosphorylation is dispensable for the initial recognition of DNA breaks","volume":"5","author":"Celeste","year":"2003","journal-title":"Nat. Cell Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib80","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1139\/o03-042","article-title":"Characteristics of gamma-H2AX foci at DNA double-strand breaks sites","volume":"81","author":"Pilch","year":"2003","journal-title":"Biochem. Cell Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib81","doi-asserted-by":"crossref","first-page":"2899","DOI":"10.1093\/hmg\/dds116","article-title":"Evaluating the role of the FUS\/TLS-related gene EWSR1 in amyotrophic lateral sclerosis","volume":"21","author":"Couthouis","year":"2012","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.molcel.2025.12.009_bib82","doi-asserted-by":"crossref","first-page":"20881","DOI":"10.1073\/pnas.1109434108","article-title":"A yeast functional screen predicts new candidate ALS disease genes","volume":"108","author":"Couthouis","year":"2011","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.molcel.2025.12.009_bib83","doi-asserted-by":"crossref","first-page":"467","DOI":"10.1038\/nature11922","article-title":"Mutations in prion-like domains in hnRNPA2B1 and hnRNPA1 cause multisystem proteinopathy and ALS","volume":"495","author":"Kim","year":"2013","journal-title":"Nature"},{"key":"10.1016\/j.molcel.2025.12.009_bib84","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.148363","article-title":"Characterization of HNRNPA1 mutations defines diversity in pathogenic mechanisms and clinical presentation","volume":"6","author":"Beijer","year":"2021","journal-title":"JCI Insight"},{"key":"10.1016\/j.molcel.2025.12.009_bib85","article-title":"Heterozygous frameshift variants in HNRNPA2B1 cause early-onset oculopharyngeal muscular dystrophy","volume":"13","author":"Kim","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.molcel.2025.12.009_bib86","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1038\/emboj.2010.310","article-title":"TDP-43 regulates its mRNA levels through a negative feedback loop","volume":"30","author":"Ayala","year":"2011","journal-title":"EMBO J."},{"key":"10.1016\/j.molcel.2025.12.009_bib87","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1038\/nn.2779","article-title":"Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43","volume":"14","author":"Polymenidou","year":"2011","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.molcel.2025.12.009_bib88","doi-asserted-by":"crossref","first-page":"452","DOI":"10.1038\/nn.2778","article-title":"Characterizing the RNA targets and position-dependent splicing regulation by TDP-43","volume":"14","author":"Tollervey","year":"2011","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.molcel.2025.12.009_bib89","doi-asserted-by":"crossref","DOI":"10.15252\/embr.202153632","article-title":"Specific RNA interactions promote TDP-43 multivalent phase separation and maintain liquid properties","volume":"22","author":"Grese","year":"2021","journal-title":"EMBO Rep."},{"key":"10.1016\/j.molcel.2025.12.009_bib90","doi-asserted-by":"crossref","DOI":"10.15252\/embj.201797452","article-title":"A single N-terminal phosphomimic disrupts TDP-43 polymerization, phase separation, and RNA splicing","volume":"37","author":"Wang","year":"2018","journal-title":"EMBO J."},{"key":"10.1016\/j.molcel.2025.12.009_bib91","doi-asserted-by":"crossref","first-page":"1537","DOI":"10.1016\/j.str.2016.07.007","article-title":"ALS Mutations Disrupt Phase Separation Mediated by alpha-Helical Structure in the TDP-43 Low-Complexity C-Terminal Domain","volume":"24","author":"Conicella","year":"2016","journal-title":"Structure"},{"key":"10.1016\/j.molcel.2025.12.009_bib92","doi-asserted-by":"crossref","first-page":"6306","DOI":"10.1074\/jbc.RA118.007222","article-title":"The role of liquid-liquid phase separation in aggregation of the TDP-43 low-complexity domain","volume":"294","author":"Babinchak","year":"2019","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.molcel.2025.12.009_bib93","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-019-12740-2","article-title":"Phase separation-deficient TDP43 remains functional in splicing","volume":"10","author":"Schmidt","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.molcel.2025.12.009_bib94","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1038\/s41591-023-02788-5","article-title":"A fluid biomarker reveals loss of TDP-43 splicing repression in presymptomatic ALS-FTD","volume":"30","author":"Irwin","year":"2024","journal-title":"Nat. Med."},{"key":"10.1016\/j.molcel.2025.12.009_bib95","doi-asserted-by":"crossref","first-page":"1140","DOI":"10.1126\/science.abq5622","article-title":"Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies","volume":"379","author":"Baughn","year":"2023","journal-title":"Science"},{"key":"10.1016\/j.molcel.2025.12.009_bib96","article-title":"Multivalent GU-rich oligonucleotides sequester TDP-43 in the nucleus by inducing high molecular weight RNP complexes","volume":"27","author":"Zhang","year":"2024","journal-title":"iScience"},{"key":"10.1016\/j.molcel.2025.12.009_bib97","article-title":"CUTS RNA Biosensor for the Real-Time Detection of TDP-43 Loss-of-Function","author":"Xie","year":"2024","journal-title":"bioRxiv"},{"key":"10.1016\/j.molcel.2025.12.009_bib98","article-title":"Short RNA chaperones promote aggregation-resistant TDP-43 conformers to mitigate neurodegeneration","author":"Copley","year":"2024","journal-title":"bioRxiv"},{"key":"10.1016\/j.molcel.2025.12.009_bib99","article-title":"mRNA 3\u2032UTRs chaperone intrinsically disordered regions to control protein activity","author":"Luo","year":"2025","journal-title":"bioRxiv"},{"key":"10.1016\/j.molcel.2025.12.009_bib100","doi-asserted-by":"crossref","DOI":"10.1101\/cshperspect.a034728","article-title":"What Are 3\u2032 UTRs Doing?","volume":"11","author":"Mayr","year":"2019","journal-title":"Cold Spring Harb. Perspect. Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib101","doi-asserted-by":"crossref","first-page":"508","DOI":"10.1038\/s41586-018-0665-2","article-title":"TDP-43 and RNA form amyloid-like myo-granules in regenerating muscle","volume":"563","author":"Vogler","year":"2018","journal-title":"Nature"},{"key":"10.1016\/j.molcel.2025.12.009_bib102","doi-asserted-by":"crossref","DOI":"10.1186\/s13024-023-00698-1","article-title":"Nuclear-import receptors as gatekeepers of pathological phase transitions in ALS\/FTD","volume":"19","author":"Khalil","year":"2024","journal-title":"Mol. Neurodegener."},{"key":"10.1016\/j.molcel.2025.12.009_bib103","article-title":"Scouring the human Hsp70 network uncovers diverse chaperone safeguards buffering TDP-43 toxicity","author":"Barbieri","year":"2025","journal-title":"bioRxiv"},{"key":"10.1016\/j.molcel.2025.12.009_bib104","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1038\/s41586-021-03824-5","article-title":"DAXX represents a new type of protein-folding enabler","volume":"597","author":"Huang","year":"2021","journal-title":"Nature"},{"key":"10.1016\/j.molcel.2025.12.009_bib105","doi-asserted-by":"crossref","DOI":"10.1016\/j.celrep.2020.108418","article-title":"TRIM11 Prevents and Reverses Protein Aggregation and Rescues a Mouse Model of Parkinson's Disease","volume":"33","author":"Zhu","year":"2020","journal-title":"Cell Rep."},{"key":"10.1016\/j.molcel.2025.12.009_bib106","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1056\/NEJMoa2204705","article-title":"Trial of Antisense Oligonucleotide Tofersen for SOD1 ALS","volume":"387","author":"Miller","year":"2022","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.molcel.2025.12.009_bib107","doi-asserted-by":"crossref","first-page":"689","DOI":"10.1007\/s40263-018-0545-1","article-title":"Nusinersen: A Review in 5q Spinal Muscular Atrophy","volume":"32","author":"Hoy","year":"2018","journal-title":"CNS Drugs"},{"key":"10.1016\/j.molcel.2025.12.009_bib108","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1038\/nbt.3784","article-title":"Oligonucleotide therapies for disorders of the nervous system","volume":"35","author":"Khorkova","year":"2017","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.molcel.2025.12.009_bib109","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1007\/s10928-021-09761-0","article-title":"A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration","volume":"48","author":"Monine","year":"2021","journal-title":"J. Pharmacokinet. Pharmacodyn."},{"key":"10.1016\/j.molcel.2025.12.009_bib110","doi-asserted-by":"crossref","DOI":"10.1177\/17562864251313915","article-title":"Tofersen and other antisense oligonucleotides in ALS","volume":"18","author":"Ludolph","year":"2025","journal-title":"Ther. Adv. Neurol. Disord."},{"key":"10.1016\/j.molcel.2025.12.009_bib111","doi-asserted-by":"crossref","DOI":"10.1111\/ene.16140","article-title":"Breaking barriers with tofersen: Enhancing therapeutic opportunities in amyotrophic lateral sclerosis","volume":"31","author":"Saini","year":"2024","journal-title":"Eur. J. Neurol."},{"key":"10.1016\/j.molcel.2025.12.009_bib112","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1038\/nature22038","article-title":"Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice","volume":"544","author":"Becker","year":"2017","journal-title":"Nature"},{"key":"10.1016\/j.molcel.2025.12.009_bib113","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1038\/s41591-021-01615-z","article-title":"Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis","volume":"28","author":"Korobeynikov","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.molcel.2025.12.009_bib114","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1016\/j.omtn.2023.01.006","article-title":"Sustained therapeutic benefits by transient reduction of TDP-43 using ENA-modified antisense oligonucleotides in ALS\/FTD mice","volume":"31","author":"Takeuchi","year":"2023","journal-title":"Mol. Ther., Nucleic Acids"},{"key":"10.1016\/j.molcel.2025.12.009_bib115","doi-asserted-by":"crossref","first-page":"2075","DOI":"10.1016\/S0140-6736(25)00513-6","article-title":"Antisense oligonucleotide jacifusen for FUS-ALS: an investigator-initiated, multicentre, open-label case series","volume":"405","author":"Shneider","year":"2025","journal-title":"Lancet"},{"key":"10.1016\/j.molcel.2025.12.009_bib116","article-title":"AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice","volume":"16","author":"Amado","year":"2025","journal-title":"Nat. Commun."},{"key":"10.1016\/j.molcel.2025.12.009_bib117","doi-asserted-by":"crossref","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.molcel.2025.12.009_bib118","doi-asserted-by":"crossref","first-page":"10542","DOI":"10.1093\/nar\/gkaa710","article-title":"Weak binding to the A2RE RNA rigidifies hnRNPA2 RRMs and reduces liquid-liquid phase separation and aggregation","volume":"48","author":"Ryan","year":"2020","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.molcel.2025.12.009_bib119","doi-asserted-by":"crossref","first-page":"2039","DOI":"10.1021\/acs.molpharmaceut.6b00165","article-title":"Inhibitory RNA Aptamers of Tau Oligomerization and Their Neuroprotective Roles against Proteotoxic Stress","volume":"13","author":"Kim","year":"2016","journal-title":"Mol. Pharm."},{"key":"10.1016\/j.molcel.2025.12.009_bib120","doi-asserted-by":"crossref","first-page":"3640","DOI":"10.1016\/j.molcel.2025.08.034","article-title":"Matrin-3 forms spherical and wormlike assemblies that are modulated by RNA binding and ALS\/FTD-associated mutations","volume":"85","author":"Sprunger","year":"2025","journal-title":"Mol. Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib121","doi-asserted-by":"crossref","DOI":"10.21769\/BioProtoc.3858","article-title":"Expression and Purification of Recombinant Skd3 (Human ClpB) Protein and Tobacco Etch Virus (TEV) Protease from Escherichia coli","volume":"10","author":"Cupo","year":"2020","journal-title":"Bio Protoc."},{"key":"10.1016\/j.molcel.2025.12.009_bib122","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-022-31098-6","article-title":"NUP62 localizes to ALS\/FTLD pathological assemblies and contributes to TDP-43 insolubility","volume":"13","author":"Gleixner","year":"2022","journal-title":"Nat. Commun."},{"key":"10.1016\/j.molcel.2025.12.009_bib123","doi-asserted-by":"crossref","first-page":"559","DOI":"10.1016\/j.stem.2018.02.012","article-title":"Dissecting the Functional Consequences of De Novo DNA Methylation Dynamics in Human Motor Neuron Differentiation and Physiology","volume":"22","author":"Ziller","year":"2018","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib124","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1016\/j.neuron.2020.01.020","article-title":"Nucleocytoplasmic Proteomic Analysis Uncovers eRF1 and Nonsense-Mediated Decay as Modifiers of ALS\/FTD C9orf72 Toxicity","volume":"106","author":"Ortega","year":"2020","journal-title":"Neuron"},{"key":"10.1016\/j.molcel.2025.12.009_bib125","first-page":"62","article-title":"Validity of nucleic acid purities monitored by 260nm\/280nm absorbance ratios","volume":"18","author":"Glasel","year":"1995","journal-title":"BioTechniques"},{"key":"10.1016\/j.molcel.2025.12.009_bib126","doi-asserted-by":"crossref","DOI":"10.1186\/gb-2009-10-3-r25","article-title":"Ultrafast and memory-efficient alignment of short DNA sequences to the human genome","volume":"10","author":"Langmead","year":"2009","journal-title":"Genome Biol."},{"key":"10.1016\/j.molcel.2025.12.009_bib127","doi-asserted-by":"crossref","first-page":"576","DOI":"10.1016\/j.molcel.2010.05.004","article-title":"Simple combinations of lineage-determining transcription factors prime cis-regulatory elements required for macrophage and B cell identities","volume":"38","author":"Heinz","year":"2010","journal-title":"Mol. Cell"},{"key":"10.1016\/j.molcel.2025.12.009_bib128","doi-asserted-by":"crossref","first-page":"1788","DOI":"10.1016\/j.bpj.2021.02.031","article-title":"Regeneration of PEG slide for multiple rounds of single-molecule measurements","volume":"120","author":"Paul","year":"2021","journal-title":"Biophys. J."},{"key":"10.1016\/j.molcel.2025.12.009_bib129","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1007\/BF00197809","article-title":"NMRPipe: a multidimensional spectral processing system based on UNIX pipes","volume":"6","author":"Delaglio","year":"1995","journal-title":"J. Biomol. NMR"},{"key":"10.1016\/j.molcel.2025.12.009_bib130","doi-asserted-by":"crossref","first-page":"1325","DOI":"10.1093\/bioinformatics\/btu830","article-title":"NMRFAM-SPARKY: enhanced software for biomolecular NMR spectroscopy","volume":"31","author":"Lee","year":"2015","journal-title":"Bioinformatics"},{"key":"10.1016\/j.molcel.2025.12.009_bib131","doi-asserted-by":"crossref","first-page":"21041","DOI":"10.1074\/jbc.M910165199","article-title":"Splicing factors induce cystic fibrosis transmembrane regulator exon 9 skipping through a nonevolutionary conserved intronic element","volume":"275","author":"Pagani","year":"2000","journal-title":"J. Biol. Chem."}],"container-title":["Molecular Cell"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1097276525009839?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1097276525009839?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,19]],"date-time":"2026-03-19T20:48:11Z","timestamp":1773953291000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1097276525009839"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1]]},"references-count":131,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["S1097276525009839"],"URL":"https:\/\/doi.org\/10.1016\/j.molcel.2025.12.009","relation":{"has-preprint":[{"id-type":"doi","id":"10.1101\/2023.09.04.555754","asserted-by":"object"}]},"ISSN":["1097-2765"],"issn-type":[{"value":"1097-2765","type":"print"}],"subject":[],"published":{"date-parts":[[2026,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains","name":"articletitle","label":"Article Title"},{"value":"Molecular Cell","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.molcel.2025.12.009","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Author(s). Published by Elsevier Inc.","name":"copyright","label":"Copyright"}]}}