The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration

Alzheimer Disease: An Update on Pathobiology and Treatment Strategies

Long, J. M., & Holtzman, D. M. (2019) · Cell · doi:10.1016/j.cell.2019.09.001 · Cited by 3,204

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

Finkel, R. S., Mercuri, E., Darras, B. T., Connolly, A. M., Kuntz, N. L., Kirschner, J., Chiriboga, C. A., Saito, K., Servais, L., Tizzano, E., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1702752 · Cited by 2,337

New tools for studying microglia in the mouse and human CNS

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RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

Bennett, C. F., & Swayze, E. E. (2010) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev.pharmtox.010909.105654 · Cited by 1,431

From charcot to lou gehrig: deciphering selective motor neuron death in als

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Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

Finkel, R. S., Chiriboga, C. A., Vajsar, J., Day, J., Montes, J., Vivo, D. C. D., Yamashita, M., Rigo, F., Hung, G., Schneider, E., et al. (2016) · The Lancet · doi:10.1016/s0140-6736(16)31408-8 · Cited by 1,039

RNA-Targeted Therapeutics

Crooke, S. T., Witztum, J. L., Bennett, C. F., & Baker, B. F. (2018) · Cell Metabolism · doi:10.1016/j.cmet.2018.03.004 · Cited by 841

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Kordasiewicz, H. B., Stanek, L. M., Wancewicz, E. V., Mazur, C., McAlonis, M. M., Pytel, K. A., Artates, J. W., Weiss, A., Cheng, S. H., Shihabuddin, L. S., et al. (2012) · Neuron · doi:10.1016/j.neuron.2012.05.009 · Cited by 770

Targeting Huntingtin Expression in Patients with Huntington’s Disease

Tabrizi, S. J., Leavitt, B. R., Landwehrmeyer, G. B., Wild, E. J., Saft, C., Barker, R. A., Blair, N. F., Craufurd, D., Priller, J., Rickards, H., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1900907 · Cited by 708

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse modelPreprint

Hua, Y., Sahashi, K., Hung, G., Rigo, F., Passini, M. A., Bennett, C. F., & Krainer, A. R. (2010) · Genes & Development · doi:10.1101/gad.1941310 · Cited by 680

Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide

Tran, H., Moazami, M. P., Yang, H., McKenna-Yasek, D., Douthwright, C. L., Pinto, C., Metterville, J., Shin, M., Sanil, N., Dooley, C., et al. (2022) · Nature Medicine · doi:10.1038/s41591-021-01557-6 · Cited by 174

Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier

Barker, S. J., Thayer, M. B., Kim, C., Tatarakis, D., Simon, M. J., Dial, R., Nilewski, L., Wells, R. C., Zhou, Y., Afetian, M., et al. (2024) · Science Translational Medicine · doi:10.1126/scitranslmed.adi2245 · Cited by 142

Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation

Belgrad, J., Fakih, H. H., & Khvorova, A. (2024) · Nucleic Acid Therapeutics · doi:10.1089/nat.2023.0068 · Cited by 130

Antisense drug discovery and development technology considered in a pharmacological context

Crooke, S. T., Liang, X., Crooke, R. M., Baker, B. F., & Geary, R. S. (2020) · Biochemical Pharmacology · doi:10.1016/j.bcp.2020.114196 · Cited by 109

Considerations in the Preclinical Assessment of the Safety of Antisense Oligonucleotides

Goyenvalle, A., Jimenez-Mallebrera, C., van Roon‐Mom, W., Sewing, S., Krieg, A. Μ., Arechavala‐Gomeza, V., & Andersson, P. U. (2022) · Nucleic Acid Therapeutics · doi:10.1089/nat.2022.0061 · Cited by 102

An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript

Dindot, S. V., Christian, S., Murphy, W. J., Berent, A., Panagoulias, J., Schlafer, A., Ballard, J. L., Radeva, K., Robinson, R., Myers, L. S., et al. (2023) · Science Translational Medicine · doi:10.1126/scitranslmed.abf4077 · Cited by 97

Applications of the versatile CRISPR‐Cas13 RNA targeting system

Kordyś, M., Sen, R., & Warkocki, Z. (2021) · Wiley Interdisciplinary Reviews - RNA · doi:10.1002/wrna.1694 · Cited by 97

Frameworks for transformational breakthroughs in RNA-based medicines

Androsavich, J. R. (2024) · Nature Reviews Drug Discovery · doi:10.1038/s41573-024-00943-2 · Cited by 100

Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases

Holm, A., Hansen, S. N., Klitgaard, H., & Kauppinen, S. (2022) · RNA Biology · doi:10.1080/15476286.2022.2066334 · Cited by 92

Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS

Pachamuthu, K., Liu, Y., Aduda, V., Akare, S., Alam, R., Andreucci, A., Boulay, D., Bowman, K., Byrne, M., Cannon, M., et al. (2022) · Nucleic Acids Research · doi:10.1093/nar/gkac037 · Cited by 77

Therapeutically viable generation of neurons with antisense oligonucleotide suppression of PTB

Maimon, R., Chillon-Marinas, C., Snethlage, C. E., Singhal, S. M., McAlonis-Downes, M., Ling, K., Rigo, F., Bennett, C. F., Da Cruz, S., Hnasko, T. S., et al. (2021) · Nature Neuroscience · doi:10.1038/s41593-021-00864-y · Cited by 62

A single-cell map of antisense oligonucleotide activity in the brain

Mortberg, M. A., Gentile, J. E., Nadaf, N. M., Vanderburg, C., Simmons, S., Dubinsky, D., Slamin, A., Maldonado, S., Petersen, C. L., Jones, N., et al. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad371 · Cited by 54

In vivo base editing extends lifespan of a humanized mouse model of prion disease

An, M., Davis, J. R., Levy, J. M., Serack, F. E., Harvey, J. W., Brauer, P. P., Pirtle, C. P., Berríos, K. N., Newby, G. A., Yeh, W.-H., et al. (2025) · Nature Medicine · doi:10.1038/s41591-024-03466-w · Cited by 58

Nonviral delivery systems for antisense oligonucleotide therapeutics

Huang, S. Q., Hao, X., Li, Y., Wu, J., Xiang, D., & Luo, S. (2022) · Biomaterials Research · doi:10.1186/s40824-022-00292-4 · Cited by 53

Targeting RNA with synthetic oligonucleotides: Clinical success invites new challenges

Hofman, C. R., & Corey, D. R. (2023) · Cell chemical biology · doi:10.1016/j.chembiol.2023.09.005 · Cited by 56

microRNA-132 regulates gene expression programs involved in microglial homeostasis

Walgrave, H., Penning, A., Tosoni, G., Snoeck, S., Davie, K., Davis, E., Wolfs, L., Sierksma, A., Mars, M., Bu, T., et al. (2023) · iScience · doi:10.1016/j.isci.2023.106829 · Cited by 41

Quantifying and mitigating motor phenotypes induced by antisense oligonucleotides in the central nervous system

Moazami, M. P., Rembetsy-Brown, J. M., Sarli, S. L., McEachern, H. R., Wang, F., Ohara, M., Wagh, A. A., Kelly, K. L., Krishnamurthy, P. M., Weiss, A., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.10.024 · Cited by 44

Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection

Byrnes, A. E., Dominguez, S. L., Yen, C.-W., Laufer, B. I., Foreman, O., Reichelt, M., Lin, H., Sagolla, M., Hötzel, K., Ngu, H., et al. (2023) · Molecular Therapy - Nucleic Acids · doi:10.1016/j.omtn.2023.05.005 · Cited by 38

Biodistribution and delivery of oligonucleotide therapeutics to the central nervous system: Advances, challenges, and future perspectives

Goto, A., Yamamoto, S., & Iwasaki, S. (2022) · Biopharmaceutics & Drug Disposition · doi:10.1002/bdd.2338 · Cited by 34

Differential effects of SARM1 inhibition in traumatic glaucoma and EAE optic neuropathies

Liu, P., Chen, W., Jiang, H., Huang, H., Liu, L., Fang, F., Li, L., Feng, X., Liu, D., Dalal, R., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.02.029 · Cited by 33

Ube3a unsilencer for the potential treatment of Angelman syndrome

Vihma, H., Li, K., Welton-Arndt, A., Smith, A. L., Bettadapur, K. R., Gilmore, R. B., Gao, E. B., Cotney, J. L., Huang, H.-C., Collins, J. L., et al. (2024) · Nature Communications · doi:10.1038/s41467-024-49788-8 · Cited by 33

Distribution and biotransformation of therapeutic antisense oligonucleotides and conjugates

Weidolf, L. O. G., Björkbom, A., Dahlén, A., Elebring, M., Gennemark, P., Hölttä, M., Janzén, D. L. I., Li, X., & Andersson, S. (2021) · Drug Discovery Today · doi:10.1016/j.drudis.2021.04.002 · Cited by 31

Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts

Shin, M., Chan, I. L., Cao, Y., Gruntman, A. M., Lee, J. T., Sousa, J., Rodríguez, T., Echeverria, D., Devi, G., Debacker, A. J., et al. (2022) · Nucleic Acids Research · doi:10.1093/nar/gkac630 · Cited by 32

Divalent siRNA for prion disease

Gentile, J. E., Corridon, T. L., Serack, F. E., Echeverria, D., Kennedy, Z., Gallant‐Behm, C. L., Hassler, M. R., Kinberger, G. A., Kelemen, M. N., Kamath, N. G., et al. (2026) · Nucleic Acids Research · doi:10.1093/nar/gkag287 · Cited by 29

Identification and characterization of a MAPT-targeting locked nucleic acid antisense oligonucleotide therapeutic for tauopathies

Easton, A. E., Jensen, M. L., Wang, C., Hagedorn, P., Li, Y.-W., Weed, M. R., Meredith, J. E., Guss, V. L., Jones, K. M., Gill, M. B., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.07.027 · Cited by 27

Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep

Ferguson, C. M., Godinho, B. M. D. C., Alterman, J. F., Coles, A. H., Hassler, M., Echeverria, D., Gilbert, J. W., Knox, E. G., Caiazzi, J., Haraszti, R. A., et al. (2021) · JCI Insight · doi:10.1172/jci.insight.152203 · Cited by 27

Gene therapies for neurogenetic disorders

Devinsky, O., Coller, J., Ahrens‐Nicklas, R. C., Liu, X. S., Ahituv, N., Davidson, B. L., Bishop, K. M., Weiss, Y., & Mingorance, A. (2025) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2025.01.015 · Cited by 29

Regional variability and genotypic and pharmacodynamic effects on PrP concentration in the CNS

Mortberg, M. A., Zhao, H. T., Reidenbach, A. G., Gentile, J. E., Kuhn, E. W., O’Moore, J., Dooley, P. M., Connors, T. R., Mazur, C., Allen, S. W., et al. (2022) · JCI Insight · doi:10.1172/jci.insight.156532 · Cited by 26

Non-invasive Imaging of Antisense Oligonucleotides in the Brain via In Vivo Click Chemistry

Cook, B. E., Archbold, J., Nasr, K., Girmay, S., Goldstein, S. I., Li, P., Dandapani, S., Genung, N. E., Tang, S.-P., McClusky, S., et al. (2022) · Molecular Imaging and Biology · doi:10.1007/s11307-022-01744-y · Cited by 26

Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT

Iwamoto, N., Liu, Y., Frank-Kamenetsky, M. D., Maguire, A., Tseng, W. C., Taborn, K., Kothari, N., Ali, A., Bowman, K., Shelke, J. D., et al. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102246 · Cited by 24

ASOs are an effective treatment for disease-associated oligodendrocyte signatures in premanifest and symptomatic SCA3 mice

Schuster, K. H., Zalon, A. J., DiFranco, D. M., Putka, A. F., Stec, N. R., Jarrah, S. I., Naeem, A., Haque, Z., Zhang, H., Guan, Y., et al. (2024) · Molecular Therapy · doi:10.1016/j.ymthe.2024.02.033 · Cited by 25

Pulmonary Hypertension: Molecular Mechanisms and Clinical Studies

Adu‐Amankwaah, J., You, Q., Liu, X., Jiang, J., Yang, D., Liu, K., Yuan, J., Wang, Y., Hu, Q., & Tan, R. (2025) · MedComm · doi:10.1002/mco2.70134 · Cited by 24

Cerebral Organoids and Antisense Oligonucleotide Therapeutics: Challenges and Opportunities

Lange, J., Zhou, H., & McTague, A. (2022) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2022.941528 · Cited by 22

Gys1 Antisense Therapy Prevents Disease-Driving Aggregates and Epileptiform Discharges in a Lafora Disease Mouse Model

Donohue, K. J., Fitzsimmons, B., Bruntz, R. C., Markussen, K. H., Young, L. E. A., Clarke, H. A., Coburn, P. T., Griffith, L. E., Sanders, W. C., Klier, J. E., et al. (2023) · Neurotherapeutics · doi:10.1007/s13311-023-01434-9 · Cited by 21

Targeting Transferrin Receptor to Transport Antisense Oligonucleotides Across the Blood-Brain BarrierPreprint

Barker, S. J., Thayer, M. B., Kim, C., Tatarakis, D., Simon, M. J., Dial, R. L., Nilewski, L., Wells, R. C., Zhou, Y., Afetian, M. E., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.04.25.538145 · Cited by 21

Antisense drugs for rare and ultra-rare genetic neurological diseases

McCauley, M. E., & Bennett, C. F. (2023) · Neuron · doi:10.1016/j.neuron.2023.05.027 · Cited by 20

Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation

Aggarwal, G., Banerjee, S., Jones, S. A., Benchaar, Y., Bélanger, J., Sévigny, M., Smith, D. M., Niehoff, M. L., Pavlack, M., de Vera, I. M. S., et al. (2023) · Journal of Biological Chemistry · doi:10.1016/j.jbc.2023.105475 · Cited by 20

Self-delivering, chemically modified CRISPR RNAs for AAV co-delivery and genome editing in vivo

Zhang, H., Kelly, K. L., Lee, J. L. C., Echeverria, D., Cooper, D., Panwala, R., Amrani, N., Chen, Z., Gaston, N., Wagh, A. A., et al. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad1125 · Cited by 19

Single-cell RNA sequencing reveals the role of immune-related autophagy in spinal cord injury in rats

Li, E., Yan, R., Yan, K., Zhang, R., Zhang, Q., Zou, P. F., Wang, H., Qiao, H., Li, S., Ma, Q., et al. (2022) · Frontiers in Immunology · doi:10.3389/fimmu.2022.987344 · Cited by 18

Interferon-α receptor antisense oligonucleotides reduce neuroinflammation and neuropathology in a mouse model of cerebral interferonopathy

Viengkhou, B., Hong, C., Mazur, C., Damle, S. S., Gallo, N. B., Fang, T., Henry, K. L., Campbell, I. L., Kamme, F., & Hofer, M. J. (2024) · Journal of Clinical Investigation · doi:10.1172/jci169562 · Cited by 18

Antisense Oligonucleotides for Rapid Translation of Gene Therapy in Glioblastoma

Desgraves, J., Valdez, M. J. M., Chandar, J. S., Gurses, M. E., Henderson, L. J., Castro, J. R., Seetheram, D., Ivan, M. E., Komotar, R. J., & Shah, A. H. (2024) · Cancers · doi:10.3390/cancers16101944 · Cited by 17

Pelizaeus–Merzbacher disease: on the cusp of myelin medicine

Elitt, M. S., & Tesar, P. J. (2024) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2024.03.005 · Cited by 16

Neuronal UBE3A substrates hold therapeutic potential for Angelman syndrome

Krzeski, J. C., Judson, M. C., & Philpot, B. D. (2024) · Current Opinion in Neurobiology · doi:10.1016/j.conb.2024.102899 · Cited by 18

Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials.

Metovic, J., Li, Y., Gong, Y., & Eichler, F. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00443 · Cited by 16

Mechanistic and Therapeutic Insights into Ataxic Disorders with Pentanucleotide Expansions

Zhang, N., & Ashizawa, T. (2022) · Cells · doi:10.3390/cells11091567 · Cited by 14

Regulation of human microglial gene expression and function via RNAase-H active antisense oligonucleotides in vivo in Alzheimer’s disease

Vandermeulen, L., Geric, I., Fumagalli, L., Kreir, M., Lu, A., Nonneman, A., Premereur, J., Wolfs, L., Policarpo, R., Fattorelli, N., et al. (2024) · Molecular Neurodegeneration · doi:10.1186/s13024-024-00725-9 · Cited by 16

Personalized allele-specific antisense oligonucleotides for GNAO1-neurodevelopmental disorder

Shomer, I., Mor, N., Raviv, S., Budick-Harmelin, N., Matchevich, T., Avkin-Nachum, S., Rais, Y., Haffner‐Krausz, R., Haimovich, A., Ziv, A., et al. (2024) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2024.102432 · Cited by 18

Regulation of activated microglia and macrophages by systemically administered DNA/RNA heteroduplex oligonucleotides

Nishi, R., Ohyagi, M., Nagata, T., Mabuchi, Y., & Yokota, T. (2022) · Molecular Therapy · doi:10.1016/j.ymthe.2022.02.019 · Cited by 14

Zinc Finger Repressors mediate widespread PRNP lowering in the nonhuman primate brain and profoundly extend survival in prion disease micePreprint

Chou, S.-W., Mortberg, M. A., Marlen, K., Ojala, D. S., Parman, T., Howard, M., DeSouza-Lenz, K., Lian, Y., Mehrabian, M., Tiffany, M. R., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.03.05.636713 · Cited by 14

LRRK2-targeting antisense oligonucleotide in Parkinson’s disease: a phase 1 randomized controlled trial

Mabrouk, O. S., Tichler, B., Arnold, H. M., Thoma, E. C., Alexanian, S. M., Chen, J., Liu, T.-Y., Hirschhorn, B., Llorens‐Arenas, R., Annand, J. W., et al. (2026) · Nature Medicine · doi:10.1038/s41591-026-04262-4 · Cited by 12

Developing antisense oligonucleotides for a TECPR2 mutation-induced, ultra-rare neurological disorder using patient-derived cellular models

Williams, L. A., Gerber, D. J., Elder, A. E., Tseng, W. C., Baru, V., Delaney-Busch, N., Ambrosi, C. M., Mahimkar, G., Joshi, V., Shah, H., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2022.06.015 · Cited by 11

Promising therapeutic aspects in human genetic imprinting disorders

Chao, Y., Qin, Y., Zou, X., Wang, X., Hu, C., Xia, F., & Zou, C. (2022) · Clinical Epigenetics · doi:10.1186/s13148-022-01369-6 · Cited by 14

Scn2a insufficiency alters spontaneous neuronal Ca2+ activity in somatosensory cortex during wakefulness

Li, M., El‐Tabbal, M., Tran, H.-D., & Kühn, B. (2023) · iScience · doi:10.1016/j.isci.2023.108138 · Cited by 11

Modulating alternative splicing of MECP2 is a potential therapeutic strategy for Rett syndrome

Tirumala, H. P., Wang, L., Li, Y., Bajikar, S. S., Anderson, A. G., Wang, W., Trostle, A. J., Zahabiyon, M., Bajić, A., Kim, J. J., et al. (2026) · Science Translational Medicine · doi:10.1126/scitranslmed.adq4529 · Cited by 10

Serine Palmitoyltransferase (SPT)-related Neurodegenerative and Neurodevelopmental Disorders

Mohassel, P., Abdullah, M., Eichler, F., & Dunn, T. M. (2024) · Journal of Neuromuscular Diseases · doi:10.3233/jnd-240014 · Cited by 10

Tailored antisense oligonucleotides for ultrarare CNS diseases: An experience-based best practice framework for individual patient evaluation

Schüle, R., Graeßner, H., Aartsma-Rus, A. M., van Roon‐Mom, W., & Synofzik, M. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102615 · Cited by 12

Functional Intercellular Transmission of miHTT via Extracellular Vesicles: An In Vitro Proof-of-Mechanism Study

Morais, R. D. V. S., Sogorb-González, M., Bar, C., Timmer, N. C., van der Bent, M. L., Wartel, M., & Vallès, A. (2022) · Cells · doi:10.3390/cells11172748 · Cited by 10

Acute neuronal inhibition response caused by phosphorothioate antisense oligonucleotides following local delivery to the central nervous system

O’Rourke, J. G., Bachmann, G., Mazur, C., Zhou, K., Platoshyn, O., Hernández, M. B., Klein, S. K., Nguyen, J., Burel, S. A., Hoffmaster, C. M., et al. (2025) · Nucleic Acids Research · doi:10.1093/nar/gkaf1333 · Cited by 17

Predicting the pharmacokinetics and pharmacodynamics of antisense oligonucleotides: an overview of various approaches and opportunities for PBPK/PD modelling

Gao, X., Diep, J. K., Norris, D. A., Yu, R. Z., & Geary, R. S. (2023) · Expert Opinion on Drug Metabolism & Toxicology · doi:10.1080/17425255.2023.2283524 · Cited by 9

Targeted ASO-mediated Atp1a2 knockdown in astrocytes reduces SOD1 aggregation and accelerates disease onset in mutant SOD1 mice

Iyer, A. K., Schoch, K. M., Verbeck, A. C., Galasso, G., Chen, H., Smith, S. E., Oldenborg, A., Miller, T. M., Karch, C. M. M., & Bonni, A. (2023) · PLoS ONE · doi:10.1371/journal.pone.0294731 · Cited by 9

PrP turnover in vivo and the time to effect of prion disease therapeutics

Corridon, T. L., O’Moore, J., Seerley, A., Sprague, D. A., Lian, Y., Laversenne, V., Noble, B., Kamath, N. G., Serack, F. E., Shaikh, A. B., et al. (2026) · PLoS Pathogens · doi:10.1371/journal.ppat.1014263 · Cited by 8

Quantitative Model Analysis and Simulation of Pharmacokinetics and Metastasis-Associated Lung Adenocarcinoma 1 RNA Knockdown Effect After Systemic Administration of Cholesterol-Conjugated DNA/RNA Heteroduplex Oligonucleotide Crossing Blood–Brain Barrier of Mice

Goto, A., Yamamoto, S., Igari, T., Matsumoto, S.-I., Chisaki, I., Iida, K., Nakayama, M., Oda, A., Kakoi, Y., Uchida, A., et al. (2022) · Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.122.001331 · Cited by 8

DNAzyme as a rising gene-silencing agent in theranostic settings

Zhang, N. (2022) · Neural Regeneration Research · doi:10.4103/1673-5374.335157 · Cited by 8

Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks

Muiños-Bühl, A., Rombo, R., Janzen, E., Ling, K., Hupperich, K., Rigo, F., Bennett, C. F., & Wirth, B. (2022) · Neurobiology of Disease · doi:10.1016/j.nbd.2022.105795 · Cited by 12

Genotypic and phenotypic analysis of 173 patients with extremely rare pathogenic mutations who applied for experimental antisense oligonucleotide treatmentPreprint

Crooke, S. T., Cole, T. A., Carroll, J. B., Gleeson, J. G., Mignon, L., Douville, J., Chung, W. K., Bain, J., Berry‐Kravis, E., Leung, N., et al. (2024) · medRxiv · doi:10.1101/2024.08.05.24310862 · Cited by 8

Unraveling and controlling late-onset neurotoxicity of antisense oligonucleotides through strategic chemical modifications

Kuroda, T., Yoshioka, K., Mon, S. S. L., Katsuyama, M., Sato, K., Isogai, E., Yoshida‐Tanaka, K., Iwata-Hara, R., Yamaguchi, T., Obika, S., et al. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102692 · Cited by 9

Areas of research priorities in epilepsy: A position paper of the European Reference Network for Rare and Complex Epilepsies, EpiCARE

Tchaicha, S., Auvin, S., Beniczky, S., Brunklaus, A., Lagae, L., Perucca, E., Surges, R., Adler, S., Helmstaedter, C., Jansen, F. E., et al. (2026) · Epilepsia Open · doi:10.1002/epi4.70210 · Cited by 8

Addressing the needs of nano-rare patients: the n-Lorem experience

Crooke, S. T., Glass, S., Gleeson, J. G., Mignon, L., Skourti-Stathaki, K., Douville, J., Knutsen, M., Pu, H., Bain, J., Berry‐Kravis, E., et al. (2026) · Nucleic Acids Research · doi:10.1093/nar/gkag504 · Cited by 7

A fluorescent splice-switching mouse model enables high-throughput, sensitive quantification of antisense oligonucleotide delivery and activity

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Elucidating brain transport pathways and cell type-dependent gene silencing of a durable lipid–siRNA conjugate administered into cerebrospinal fluid

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Biodistribution of Radioactively Labeled Splice Modulating Antisense Oligonucleotides After Intracerebroventricular and Intrathecal Injection in Mice

Metz, T., Welling, M. M., Suidgeest, E., Nieuwenhuize, E., de Vlaam, T., Curtis, D., Hailu, T. T., van der Weerd, L., & van Roon‐Mom, W. (2024) · Nucleic Acid Therapeutics · doi:10.1089/nat.2023.0018 · Cited by 6

Targeting spinal mechanistic target of rapamycin complex 2 alleviates inflammatory and neuropathic pain

Wong, C., Rodriguez-Hernandez, L. D., Lister, K. C., Gu, N., Cai, W., Hooshmandi, M., Fan, J., Brown, N., Nguyen, V., Ribeiro‐da‐Silva, A., et al. (2024) · Brain · doi:10.1093/brain/awae275 · Cited by 6

OligoDistiller: A Platform Agnostic Software Tool for MS and MS 2 Data Analysis of Complex Oligonucleotides and Their Impurities

Liu, Y., Lippens, J. L., Prostko, P., de Vries, R., Valkenborg, D., & Vijlder, T. D. (2024) · Analytical Chemistry · doi:10.1021/acs.analchem.4c02667 · Cited by 7

Nucleic Acid Therapeutics

Watts, J. K. (2022) · Royal Society of Chemistry eBooks · doi:10.1039/9781837671328-00350 · Cited by 6

Evaluating the efficacy of purchased antisense oligonucleotides to reduce mouse and human tau in vivo

Vemula, P., Schoch, K. M., & Miller, T. M. (2023) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2023.1320182 · Cited by 5

Comparison of MALAT1 antisense oligonucleotide distribution following intracerebroventricular and lumbar intrathecal routes of administration

Boyd, M. M., Mazur, C., Pribnow, K., Zanon, R. L., Adams, E., DuGal, J., Klein, S., & Shafer, L. L. (2025) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2025.102742 · Cited by 5

Utility of oligonucleotide in upregulating circular RNA production in a cellular model

Ni, L., Yamada, T., & Nakatani, K. (2024) · Scientific Reports · doi:10.1038/s41598-024-58663-x · Cited by 6

Brain somatic mutations as RNA therapeutic targets in neurological disorders

Lee, S., & Lee, J. H. (2022) · Annals of the New York Academy of Sciences · doi:10.1111/nyas.14786 · Cited by 5

Targeting nonsense-mediated RNA decay does not increase progranulin levels in the Grn R493X mouse model of frontotemporal dementia

Smith, D. M., Niehoff, M. L., Ling, K., Jafar‐Nejad, P., Rigo, F., Farr, S. A., Wilkinson, M., & Nguyen, A. D. (2023) · PLoS ONE · doi:10.1371/journal.pone.0282822 · Cited by 5

Single-dose administration of therapeutic divalent siRNA targeting MECP2 prevents lethality for one year in an MECP2 duplication mouse modelPreprint

Hariharan, V. N., Summers, A., Clipperton‐Allen, A. E., Caiazzi, J., Hildebrand, S. R., Reilly, D. O., Tang, Q., Kennedy, Z., Echeverria, D., McHugh, N., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.03.26.645328 · Cited by 5

Transient acute neuronal activation response caused by high concentrations of oligonucleotides in the cerebral spinal fluid

Bravo‐Hernández, M., Mazur, C., Chen, H., Fradkin, L., Searcy, J., Burel, S. A., Kelly, M., Bruening, D., O’Rourke, J. G., Cai, Y., et al. (2026) · Nucleic Acids Research · doi:10.1093/nar/gkag057 · Cited by 9

The Versatile Applications of Antisense Oligonucleotides in Modern Medicine

Liang, X., & Zhang, L. (2026) · International Journal of Molecular Sciences · doi:10.3390/ijms27125612 · Cited by 5

Transient Acute Neuronal Activation Response Caused by High Concentrations of Oligonucleotides in the Cerebral Spinal FluidPreprint

Hernandez, M. B., Mazur, C., Chen, H., Fradkin, L., Searcy, J., Burel, S. A., Kelly, M. E., Bruening, D., O’Rourke, J. G., Cai, Y., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.02.13.638138 · Cited by 4

Cell Type Distribution of Intrathecal Antisense Oligonucleotide Activity in Deep Brain Regions of Non-Human Primates

Frei, J. A., Gentile, J. E., Lian, Y., Mortberg, M. A., Capitanio, J. S., Jafar‐Nejad, P., Vallabh, S. M., Zhao, H. T., & Minikel, E. V. (2025) · Nucleic Acid Therapeutics · doi:10.1177/21593337251371594 · Cited by 4

Artificial miRNA-mediated reduction of SNCA for the treatment of α-synucleinopathies

Elmer, B. M., Wischhof, E., Chan, T., Mahendran, T., Ardinger, J. A., Richards, B., Liu, D., Bladon, J. H., He, Y., Jackson, R., et al. (2025) · Molecular Therapy · doi:10.1016/j.ymthe.2025.11.017 · Cited by 3

Morpholino–RNA duplex exhibits robust, sustained, and safe steric-block antisense activity by intracerebroventricular and intrathecal injection

Yanagidaira, M., Nagata, T., Hasegawa, J., Ishii, T., Ebihara, S., Nawa, M., & Yokota, T. (2025) · Nature Communications · doi:10.1038/s41467-025-66765-x · Cited by 3

Antisense Oligonucleotides in Parkinson's Disease: Challenges, Opportunities, and the Promise of Low‐Intensity Focused Ultrasound for Brain Delivery

Obika, S., & Blesa, J. (2026) · Journal of Neurochemistry · doi:10.1111/jnc.70445 · Cited by 3

Modulation of prion protein expression through cryptic splice site manipulation

Gentile, J. E., Corridon, T. L., Mortberg, M. A., D’Souza, E. N., Whiffin, N., Minikel, E. V., & Vallabh, S. M. (2024) · Journal of Biological Chemistry · doi:10.1016/j.jbc.2024.107560 · Cited by 3

Recent advances in RNA-based therapeutics for neurodevelopmental disorders

Tirumala, H. P., & Zoghbi, H. Y. (2025) · Current Opinion in Genetics & Development · doi:10.1016/j.gde.2025.102339 · Cited by 3

A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsiesPreprint

Dame, H. B., Kopalle, H., Miñana, B., Klaft, Z., Fahey, J. B., McWatters, D. C., Rao, V., Suresh, R., Aigner, S., Hammer, M. F., et al. (2025) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2025.10.22.683934 · Cited by 3

Cell Type-Specific Contributions of UBE3A to Angelman Syndrome Behavioral Phenotypes

Ringelberg, N. W., Mayfield, R. E., Lord, J. S., Diering, G. H., Burette, A. C., & Philpot, B. D. (2025) · eNeuro · doi:10.1523/eneuro.0453-24.2025 · Cited by 2

Antisense Oligonucleotide Quantification via Splint-Ligation PCR Assay in Nonhuman Primate Central Nervous System Tissues and Biofluids

Jepp, T., Christian, S., & Dindot, S. V. (2025) · Nucleic Acid Therapeutics · doi:10.1177/21593337251391564 · Cited by 2

Gene therapy for huntington’s disease: advances, challenges, and future perspectives

Kaushal, R., Yadav, M., Kosey, S., & Dwivedi, M. (2026) · Neurogenetics · doi:10.1007/s10048-026-00887-2 · Cited by 2

Management of spinocerebellar ataxia

Vinokurov, E. K., Marinina, K. S., & Борисович, Б. И. (2026) · Neurodegenerative Disease Management · doi:10.1080/17582024.2026.2665356 · Cited by 2

Ext1-targeted gene therapy ameliorates tauopathy by inhibiting heparan sulfate-dependent tau propagation

Huang, R., Ye, X., Liu, J., Yang, C., Meng, X., Dou, T., Zhuang, X., Yang, J., Zheng, N., Zhao, Y., et al. (2026) · Pharmacological Research · doi:10.1016/j.phrs.2026.108265 · Cited by 2

Beyond muscle: Delivering RNA therapeutics to the CNS in Duchenne muscular dystrophy

Vacca, O., Nagy, C., & Goyenvalle, A. (2025) · Journal of Neuromuscular Diseases · doi:10.1177/22143602251360668 · Cited by 2

Exploring the pleiotropic effects of lncRNA in different repeat expansion disorders

Das, S., Khandelwal, S., Shukla, S., & Kumar, A. (2025) · Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease · doi:10.1016/j.bbadis.2025.168089 · Cited by 2

Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivoPreprint

Zhang, H., Kelly, K. L., Lee, J., Echeverria, D., Cooper, D., Panwala, R., Chen, Z., Gaston, N., Newby, G. A., Xie, J., et al. (2023) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2023.03.20.533459 · Cited by 2

How can MSI enhance our understanding of ASO distribution?

van der Vloet, L., Hilaire, P. B. S., Bouillod, C., Isin, E. M., Heeren, R. M. A., & Vandenbosch, M. (2024) · Drug Discovery Today · doi:10.1016/j.drudis.2024.104275 · Cited by 1

Therapeutic suppression of Tubb4a rescues H-ABC leukodystrophy

Sase, S., Hacker, J. L., Napit, P. R., Bhagavatula, A., Woidill, S., D’Alessandro, A., Jeffries, M. A., Almad, A., Takanohashi, A., Padiath, Q. S., et al. (2026) · Molecular Therapy · doi:10.1016/j.ymthe.2026.01.016 · Cited by 1

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

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A single-cell map of antisense oligonucleotide activity in the brain

Mortberg, M. A., Gentile, J. E., Nadaf, N. M., Vanderburg, C., Simmons, S., Dubinsky, D., Slamin, A., Maldonado, S., Petersen, C. L., Jones, N., et al. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad371 · Cited by 54

Antisense oligonucleotide drugs for neurological and neuromuscular diseases

Lane, R. M., & Bennett, C. F. (2024) · Elsevier eBooks · doi:10.1016/b978-0-443-19041-4.00039-x

Antisense Oligonucleotide Therapy for Neurodevelopmental Disorders

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Wrangling RNA: Antisense oligonucleotides for neurological disorders

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