Q1 2026 Publications from our Innovation Ecosystem
In the first quarter of 2026, researchers across the Target ALS innovation ecosystem published more than 30 peer-reviewed papers, spanning biomarkers, disease mechanisms, therapeutic targets, and more. This is what a fully activated global research community looks like in action.
March 31, 2026 Amy Easton
- Multi-omics analyses identify potential epigenetic loci associated with survival in amyotrophic lateral sclerosis across diverse populations. Gu Y, Chen Y, Tang X, Guo J, Hu J, Yang W, Li J, Chen X, Fan D, Chen GB, He J, Ren Y, Dong Y, Sato C, Chen Y, Zinman L, Rogaeva E, Zhang M. EBioMedicine. 2026 Jan;123:106071. doi: 10.1016/j.ebiom.2025.106071. Epub 2025 Dec 10. PMID: 41380476; PMCID: PMC12754208.
- Early brain-wide disruption of sleep microarchitecture in amyotrophic lateral sclerosis. Lang C, Guillot SJ, Lule D, Balz LT, Knehr A, Weydt P, Dorst J, Kandler K, Muller HP, Kassubek J, Wassermann L, Da Cruz S, Roselli F, Ludolph AC, Bolborea M, Dupuis L. J Clin Invest. 2025 Nov 6;136(1):e194555. doi: 10.1172/JCI194555. PMID: 41196652; PMCID: PMC12721911.
- TBK1 orchestrates autophagy and endo-lysosomal pathways in human neurons. Mordes DA, Smeyers J. Autophagy. 2026 Jan 4:1-3. Doi: 10.1080/15548627.2025.2609924. Epub ahead of print. PMID: 41485128.
- TDP-43-mediated alternative polyadenylation is associated with a reduction in VPS35 and VPS29 expression in frontotemporal dementia. Maheswari Jawahar V, Zeng Y, Armour EM, Yue M, Citrano K, Lovchykova A, Reeves MM, Rawlinson B, DeTure M, Dunmore JA, Song Y, Ball SK, Wszolek ZK, Graff-Radford NR, Boeve BF, Knopman DS, Day GS, Small SA, Dickson DW, Ward ME, Gendron TF, Zhang Y, Prudencio M, Gitler AD, Petrucelli L. PLoS Biol. 2026 Jan 5;24(1):e3003573. doi: 10.1371/journal.pbio.3003573. PMID: 41490046; PMCID: PMC12768243.
- Divergent TDP43-regulated and TDP43-independent cryptic splicing in the cortex and spinal cord. Newton, D.F., Yang, R., Gutierrez, J. et al. Mol. Neurodegener. Adv. 2, 6 (2026). https://doi.org/10.1186/s44477-025-00013-w
- Defining RNA oligonucleotides that reverse deleterious phase transitions of RNA-binding proteins with prion-like domains. Guo L, Mann JR, Mauna JC, Copley KE, Wang H, Rubien JD, Odeh HM, Lin J, Lee BL, Ganser L, Robinson E, Kim KM, Murthy AC, Paul T, Portz B, Gleixner AM, Diaz Z, Carey JL, Smirnov A, Padilla G, Lavorando E, Espy C, Shang Y, Huang EJ, Chesi A, Fawzi NL, Myong S, Donnelly CJ, Shorter J. Mol Cell. 2026 Jan 8;86(1):114-134.e10. doi: 10.1016/j.molcel.2025.12.009. PMID: 37732211; PMCID: PMC10508739.
- Fluid and Neuroimaging Biomarkers in Microgliopathy Colony-Stimulating Factor-1 Receptor-Related Disorders. Chmiela T, Reeves M, Jansen-West K, Dunmore J, Song Y, Strongosky A, Gandhi S, Pikover G, Blurton-Jones M, Spitale RC, Middlebrooks EH, Petrucelli L, Prudencio M, Wszolek ZK. Ann Clin Transl Neurol. 2026 Jan 12. doi: 10.1002/acn3.70250. Epub ahead of print. PMID: 41521943.
- Dietary Non-protein Amino Acid AZE as an Environmental Trigger Activating Latent Genetic Susceptibility in C9orf72-ALS Pathogenesis. Chongjiu Chen, Zhanyun Lv, Guangyu Cui, Hui Gong, Jianfeng Hua, Tao Luo, Hongyun Bi, Xinrui Zhang, Qinqin Cui, Hongying Zhu, Xing Guo, Nengyin Sheng, Chengyong Shen, Dongsheng Fan, Ke Zhang, Huaqing Liu, Ge Bai. bioRxiv 2026.01.12.697838; doi: https://doi.org/10.64898/2026.01.12.697838
- Computationally Designed RNA Aptamers Enable Selective Detection of FUS Pathology in ALS. Elsa Zacco, Martina Gilodi, Abigail Dos Santos, Alexandros Armaos, Rachel James, Francesco Di Palma, Fergal M. Waldron, Marco Scotto, Jakob Rupert, Vlad Korobeynikov, Neil A. Shneider, Mathew H. Horrocks, Jenna M. Gregory, Gian Gaetano Tartaglia. bioRxiv 2025.04.30.651570; doi: https://doi.org/10.1101/2025.04.30.651570
- Hypoxic stress is an early pathogenic event in human VCP-mutant ALS astrocytes. Franklin HD, Crerar H, Parnandi N, Lattke M, Majewski S, Clarke BE, Pallikonda H, Howell M, Boulton SJ, Patani R. Stem Cell Reports. 2026 Jan 13;21(1):102723. doi: 10.1016/j.stemcr.2025.102723. Epub 2025 Dec 4. PMID: 41349534; PMCID: PMC12925969.
- Clonal CD8+ T cells populate the leptomeninges and coordinate with immune cells in human degenerative brain diseases. Hobson R, Levy SHS, Singal CMS, Flaherty D, Xiao H, Ciener B, Reddy H, Zabinyakov N, Kim CY, Teich AF, Shneider NA, Bradshaw EM, Elyaman W. Nat Immunol. 2026 Feb;27(2):323-335. doi: 10.1038/s41590-025-02401-6. Epub 2026 Jan 27. PMID: 41593242; PMCID: PMC12864034.
- TBK1 controls axonal transport in motor neurons. David Villarroel-Campos, Jose Norberto S Vargas, Martin Wallace, Kai Sun, James N Sleigh, Pietro Fratta, Giampietro Schiavo. Life Science Alliance Feb 2026, 9 (4) e202503527; DOI: 10.26508/lsa.202503527
- C9orf72 hexanucleotide repeat RNA drives transcriptional dysregulation through genome-wide DNA:RNA hybrid G-quadruplexes. Liu H, Liu M, Liu Y, Gui G, Paul T, Lu YN, Huang Z, Wang H, Xiao Y, Zheng Z, Periz G, Shi Y, Ichida JK, Myong S, Ji H, Wang J. Neuron. 2026 Feb 4:S0896-6273(25)00932-8. doi: 10.1016/j.neuron.2025.12.005. Epub ahead of print. PMID: 41643661.
- Blocking RAN translation without altering repeat RNAs rescues C9ORF72-related ALS and FTD phenotypes. Jiang X, Schaeffer L, Patni D, Russo T, Lee CZ, Aguilar C, Marques C, Jansen-West K, Hruska-Plochan M, Ray-Soni A, Lim SM, Held A, Yue M, Castellanos Otero P, Aryal S, Beaussant HDAM, Basu H, Takakuwa H, Daughrity LM, Ramesh N, Da Costa P, A A Quadros AR, Nolan M, Reyes CJF, Wheeler H, Moran LC, Griesman G, Wymann B, Trombetta BA, Lopez-De-Silanes ES, Canori M, Krishnan G, Vieira Souza Da Silva Y, Eriani G, Albers MW, Arnold SE, Song Y, Jain A, Chiu IM, Zhang YJ, Gao FB, Wainger BJ, Polymenidou M, Petrucelli L, Martin F, Lagier-Tourenne C. Science. 2026 Feb 5;391(6785):eadv2600. doi: 10.1126/science.adv2600. Epub 2026 Feb 5. PMID: 41643021.
- Organizing research at scale: Reflections on leading multi-team scientific collaborations. Yeo GW. Mol Cell. 2026 Feb 5;86(3):434-438. doi: 10.1016/j.molcel.2026.01.013. PMID: 41650944.
- Transcriptomic profiling uncovers mis-splicing and gene fusions in amyotrophic lateral sclerosis. Huilin Xu, Tiziana Petrozziello, Adel Boudi, Shota Shibata, Sommer S. Huntress, Alireza Shahryari, Xuefang Zhao, Maheswaran Kesavan, Eric J. Granucci, Ayleen L. Castillo Torres, Ranee Zara B. Monsanto, John Lemanski, Bimal Jana, Celin E.F. De Esch, Merit E. Cudkowicz, James D. Berry, NYGC ALS Consortium, Harrison Brand, Michael E. Talkowski, Ricardo Mouro Pinto, Dadi Gao, Ghazaleh Sadri-Vakili. medRxiv 2026.02.05.26345503; doi: https://doi.org/10.64898/2026.02.05.26345503
- Large-scale mapping of the MCH network in ALS mice reveals the vulnerability of dopaminergic and GABAergic neurons in zona incerta. Scekic-Zahirovic J, Antonucci S, Wiesner D, Ebner C, El Hajj H, Aousji O, Halablab K, Fan Y, Zelaya A, Yartas G, Baskar K, Çakmak EA, Bayer D, Sung HK, Dupuis L, Park J, Roselli F. Acta Neuropathol Commun. 2026 Feb 6;14(1):46. doi: 10.1186/s40478-026-02231-z. PMID: 41652471; PMCID: PMC12930930.
- Wild-type C9orf72 expression is a genetic modifier of C9-ALS survival. Stanislav Tsitkov, Akshay Raju, Jie Wu, Jonathan Li, Ryan G Lim, Zhuoxing Wu, Noura Al Bistami, Answer ALS Consortium, Jennifer E. Van Eyk, Clive N Svendsen, Jeffrey D Rothstein, Jonathan D Glass, Steve Finkbeiner, Julia A Kaye, Leslie M Thompson, Ernest Fraenkel. medRxiv 2026.02.06.26345684; doi: https://doi.org/10.64898/2026.02.06.26345684
- Microscale dysfunction and mesoscale compensation in degenerating neuronal networks. Vegard Fiskum, Nicolai Winter-Hjelm, Nicholas Christiansen, Axel Sandvig, Ioanna Sandvig. Network Neuroscience 2026; doi: https://doi.org/10.1162/NETN.a.552
- Accurate strand-specific long-read transcript isoform discovery and quantification at bulk, single-cell, and single-nucleus resolution. Houlin Yu, Christophe Georgescu, Akanksha Khorgade, Ghamdan Al-Eryani, Daniel A Bartlett, Allison Brookhart, Can Kockan, James T Webber, Asa Shin, Emily White, Xylena Reed, Fangle Hu, Sarah Bromberek, Sandra Ndayambaje, Sandeep Aryal, Dennis W Dickson, Mercedes Prudencio, Clotilde Lagier-Tourenne, Michael Ward, Paul Blainey, Victoria Popic, Brian J Haas, Aziz M AlKhafaji. bioRxiv 2026.02.12.705617; doi: https://doi.org/10.64898/2026.02.12.705617
- Fecal microbiota transplantation from psychiatric patients to mice – systematic review of methodologies and a call for standardization. D’Onofrio AM, Gomez-Nguyen A, Camardese G, Scaldaferri F, Burberry A, Cominelli F. Transl Psychiatry. 2026 Feb 12;16(1):107. doi: 10.1038/s41398-026-03847-4. PMID: 41672982; PMCID: PMC12923557.
- TDP-43 pathology is linked to motor neuron loss but is independent of stress granules in vivo. Alicia Dubinski, Ala Ferdi, Mariam Choughari, Holly Spence, Arpita Adhikary, Carolane Fauchon, Melissa Touati, Myriam Gagné, Martha Liu, Sarah Peyrard, Jenna Gregory, Christine Vande Velde. bioRxiv 2026.02.11.705439; doi: https://doi.org/10.64898/2026.02.11.705439
- Munc13-1 couples DAG and Ca2+ signaling to dynamic vesicle priming, synaptic short-term plasticity, and posttetanic potentiation. Ranjan M, Lin KH, Mueller BD, Wojcik SM, Lohse M, Südhof TC, Jorgensen EM, Neher E, Lipstein N, Brose N, Taschenberger H. Sci Adv. 2026 Feb 13;12(7):eaea0449. doi: 10.1126/sciadv.aea0449. Epub 2026 Feb 13. PMID: 41686904; PMCID: PMC12904202.
- IsomiR utility in amyotrophic lateral sclerosis prognostication. Cohen Y, Sinai I, Magen I, Danino YM, Wuu J, Malaspina A, Benatar M, Hornstein E. Med. 2026 Feb 13;7(2):100928. doi: 10.1016/j.medj.2025.100928. Epub 2025 Nov 21. PMID: 41274292.
- Impaired nucleocytoplasmic transport in SOD1-mediated ALS. Argueti-Ostrovsky S, Lim SM, Arogundade OA, Diaz-Garcia S, Yunisova G, Meng A, Hermann A, Ong K, Eremenko E, Bravo-Hernandez M, Driscoll SP, Lee CZ, Jiang X, Stavsky A, Barel S, Shani T, Kahn J, Pfaff SL, Monsonego A, Marsala M, Ravits J, Lagier-Tourenne C, Israelson A. Mol Neurodegener. 2026 Feb 14;21(1):14. doi: 10.1186/s13024-026-00930-8. PMID: 41691309; PMCID: PMC12922372.
- Shake and bake: a robust and cost-effective proteomic sample preparation workflow for plasma and cerebrospinal fluid. Rönnholm J, Weiner S, Fuchs J, Thorsell A, Sihlbom C, Dugom L, Easton A, Zetterberg H, Gobom J. Clin Proteomics. 2026 Feb 15;23(1):12. doi: 10.1186/s12014-026-09589-1. PMID: 41692724; PMCID: PMC12927225.
- C9orf72 in myeloid cells prevents an inflammatory response to microbial glycogen. McCourt B, Lemr K, Chakrabarti S, Woidke E, Ramaiah S, Singh V, Sangwan N, Brown JM, Cominelli F, Rodriguez-Palacios A, Burberry A. Cell Rep. 2026 Jan 23;45(2):116906. doi: 10.1016/j.celrep.2025.116906. Epub ahead of print. PMID: 41581145.
- Statins and genetic inhibition of the mevalonate pathway activate an ATF3-STMN2 regenerative program. Matthew Nolan, Sandeep Aryal, I. Sandra Ndayambaje, Maize Cao, Peyton Lee, Moriah Hovde, Shuqi Yun, Josette Wlaschin, Aaron Held, Hortense Beaussant, Benjamin Wymann, Chao Zong-Lee, Su Min Lim, Xin Jiang, Nandini Ramesh, Ana Rita Agra Almeida Quadros, Ayub Boulos, Nicolas Zinter, Shireen Salem, Laïla El-Tayar, Melinda Beccari, Maximiliano Presa, Charles Jourdan Ferreras Reyes, Yin Yin Ruan, Grant Griesman, Corey Aguilar, James Hawrot, Hayden Wheeler, Zevik Melamed, Benjamin P. Kleinstiver, Mark Albers, Don W. Cleveland, Rudolph E. Tanzi, Cathleen M. Lutz, Robert D. Hubbard, Dione Kobayashi, Michael Ward, Christiano R.R Alves, Brian Wainger, Claire Le Pichon, Clotilde Lagier-Tourenne. bioRxiv 2026.02.23.707492; doi: https://doi.org/10.64898/2026.02.23.707492
- 18F FDG-PET correlates of motor neuron disease motor variants. Boris Deleu, Patrick Dupont, Kato Bracaval, Fouke Ombelet, Frederik Hobin, Nikita Lamaire, Koen Van Laere, Philip Van Damme, Joke De Vocht. medRxiv 2026.02.24.26347019; doi: https://doi.org/10.64898/2026.02.24.26347019
- Prospective Validation of the New PLS Diagnostic Criteria From PLS Natural History Study: EMG and Neurofilament Analyses. Sorenson E, Heitzman D, Lee I, Jang G, Elman L, Habib AA, Wymer J, Hayat G, Goutman SA, Fernandes JAM, Floeter MK, Ajroud-Driss S, Gwathmey K, Kasarskis E, Kisanuki YY, Lomen-Hoerth C, Walk D, Johnston WS, Diaz F, Maragakis NJ, Paganoni S, Shah J, Oskarsson B, Zinman L, Heiman-Patterson T, Jawdat O, Fournier CN, Pulley MT, Scelsa SN, Shoesmith C, Simmons Z, Sherman AV, Hoover BN, Yun RY, Cheung K, Mitsumoto H; PLS NHS Study Group. Muscle Nerve. 2026 Mar 2. doi: 10.1002/mus.70192. Epub ahead of print. PMID: 41772409.
- Disruption of the angiopoietin-like system connects lipid homeostasis and hypothalamic dysfunction in ALS. Krishnamurthy SS, Buzatto AZ, Campkin C, Müller HP, Wiesner D, Weishaupt J, Klose V, Wiesenfarth M, Elmas Z, Herrmann C, Parlak Ö, Günther K, Saad R, Weiland U, Dupuis L, Ludolph A, Li L, Kassubek J, Dorst J, Roselli F. BMC Med. 2026 Mar 3. doi: 10.1186/s12916-026-04749-4. Epub ahead of print. PMID: 41776545.
- Multi-modal dissection of cell-type specific TDP-43 pathology in the motor cortex. Ruf WP, Kühlwein JK, Meier L, Brockmann SJ, LeeBae J, Sadri-Vakili G, Yilmazer-Hanke D, Petri S, Thal DR, Grozdanov V, Danzer KM. Nat Commun. 2026 Mar 9. doi: 10.1038/s41467-026-69944-6. Epub ahead of print. PMID: 41803120.
- Ca2+-phospholipid-dependent regulation of Munc13-1 is essential for post-tetanic potentiation at mossy fiber synapses and supports working memory. López-Murcia FJ, Krueger-Burg D, Wenger S, López-Hernández T, Lipstein N, Taschenberger H, Brose N. Cell Rep. 2026 Feb 19;45(3):117029. doi: 10.1016/j.celrep.2026.117029. Epub ahead of print. PMID: 41719128.
- Metabolic signatures of ferritin and TDP-43 co-pathology provide a mechanistic basis for stratified therapeutic approaches in ALS. Holly Spence, Fiona L. Read, Fergal M. Waldron, Jenna M. Gregory. bioRxiv 2026.03.13.711539; doi: https://doi.org/10.64898/2026.03.13.711539
- Dissecting the relationship between haplotypes around ATXN2 CAG repeats and the number of CAA interruptions by long-read sequencing. Beoung Hun Lee, Joe Chan, Corey T McMillan, NYGC ALS Consortium, Yuanquan Song, Defne A Amado, Kai Wang. medRxiv 2026.03.11.26348169; doi: https://doi.org/10.64898/2026.03.11.26348169
- Granulin loss and TMEM106B risk converge on lysosomal C-terminal fragment pathology in frontotemporal dementia. Yi Zeng, Jian Xiong, Anastasiia Lovchykova, Thao Phuong Nguyen, Abigail Song, Semma W Gitler, Monther Abu-Remaileh, Aaron D Gitler. bioRxiv 2026.03.25.713523; doi: https://doi.org/10.64898/2026.03.25.713523
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