Antisense Oligonucleotide Therapies for Neurodegenerative Diseases

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes

MACDONALD, M. (1993) · Cell · doi:10.1016/0092-8674(93)90585-e · Cited by 8,366

Mutations in Cu/Zn superoxide dismutase gene are associated with familial amyotrophic lateral sclerosis

Rosen, D. R., Siddique, T., Patterson, D., Figlewicz, D. A., Sapp, P., Hentati, A., Donaldson, D., Goto, J., O'Regan, J. P., Deng, H.-X., et al. (1993) · Nature · doi:10.1038/362059a0 · Cited by 7,062

Ubiquitinated TDP-43 in Frontotemporal Lobar Degeneration and Amyotrophic Lateral Sclerosis

Neumann, M., Sampathu, D. M., Kwong, L. K., Truax, A. C., Micsenyi, M. C., Chou, T. T., Bruce, J., Schuck, T., Grossman, M., Clark, C. M., et al. (2006) · Science · doi:10.1126/science.1134108 · Cited by 6,576

Expanded GGGGCC Hexanucleotide Repeat in Noncoding Region of C9ORF72 Causes Chromosome 9p-Linked FTD and ALS

DeJesus-Hernandez, M., Mackenzie, I. R., Boeve, B. F., Boxer, A. L., Baker, M., Rutherford, N. J., Nicholson, A. M., Finch, N. A., Flynn, H., Adamson, J., et al. (2011) · Neuron · doi:10.1016/j.neuron.2011.09.011 · Cited by 4,936 · cited 3×

A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD

Renton, A. E., Majounie, E., Waite, A., Simón-Sánchez, J., Rollinson, S., Gibbs, J. R., Schymick, J. C., Laaksovirta, H., van Swieten, J. C., Myllykangas, L., et al. (2011) · Neuron · doi:10.1016/j.neuron.2011.09.010 · Cited by 4,409

Identification and characterization of a spinal muscular atrophy-determining gene

Lefebvre, S., Bürglen, L., Reboullet, S., Clermont, O., Burlet, P., Viollet, L., Bénichou, B., Cruaud, C., Millasseau, P., Zeviani, M., et al. (1995) · Cell · doi:10.1016/0092-8674(95)90460-3 · Cited by 3,995

A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. The Huntington's Disease Collaborative Research Group.

Shah, M. A., Datson, N. A., Srinidhi, L., Stanton, V. P., MacDonald, M. E., Allard, M. W., Youngman, S., Am, F., Mott, R., Draths, K., et al. (1993) · Cell · Cited by 3,055

Mechanisms of gene silencing by double-stranded RNA

Meister, G., & Tuschl, T. (2004) · Nature · doi:10.1038/nature02873 · Cited by 2,598

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

Amyotrophic lateral sclerosis

Hardiman, O. M., Al‐Chalabi, A., Chiò, A., Corr, E. M., Logroscino, G., Robberecht, W., Shaw, P. J., Simmons, Z., & van den Berg, L. H. (2017) · Nature Reviews Disease Primers · doi:10.1038/nrdp.2017.71 · Cited by 2,237

Strategies for delivering therapeutics across the blood–brain barrier

Terstappen, G. C., Meyer, A. H., Bell, R. D., & Zhang, W. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-021-00139-y · Cited by 1,138

Antisense technology: an overview and prospectus

Crooke, S. T., Baker, B. F., Crooke, R. M., & Liang, X.-H. (2021) · Nature Reviews Drug Discovery · doi:10.1038/s41573-021-00162-z · Cited by 775

Phosphorylated Tau in Alzheimer’s Disease and Other Tauopathies

Rawat, P., Sehar, U., Bisht, J., Selman, A., Culberson, J., & Reddy, P. H. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms232112841 · Cited by 509

Reversing a model of Parkinson's disease with in situ converted nigral neurons

Qian, H., Kang, X., Hu, J., Zhang, D., Liang, Z., Meng, F., Zhang, X., Xue, Y., Maimon, R., Dowdy, S. F., et al. (2020) · Nature · doi:10.1038/s41586-020-2388-4 · Cited by 480

Pathological mechanisms and therapeutic strategies for Alzheimer’s disease

Tam, K. Y., & Ju, Y. (2021) · Neural Regeneration Research · doi:10.4103/1673-5374.320970 · Cited by 422

Antisense technology: A review

Crooke, S. T., Liang, X., Baker, B. F., & Crooke, R. M. (2021) · Journal of Biological Chemistry · doi:10.1016/j.jbc.2021.100416 · Cited by 350

Tauopathies: new perspectives and challenges

Zhang, Y., Wu, K.-M., Yang, L., Dong, Q., & Yu, J.-T. (2022) · Molecular Neurodegeneration · doi:10.1186/s13024-022-00533-z · Cited by 323

Challenges and hopes for Alzheimer’s disease

Tatulian, S. A. (2022) · Drug Discovery Today · doi:10.1016/j.drudis.2022.01.016 · Cited by 291

RNA genome conservation and secondary structure in SARS-CoV-2 and SARS-related viruses: a first look

Rangan, R., Zheludev, I. N., Hagey, R. J., Pham, E. A., Wayment-Steele, H. K., Glenn, J. S., & Das, R. (2020) · RNA · doi:10.1261/rna.076141.120 · Cited by 278

Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies

Baughn, M. W., Melamed, Z., López‐Erauskin, J., Beccari, M. S., Ling, K., Zuberi, A. R., Presa, M. F., Gonzalo-Gil, E., Maimon, R., Vazquez‐Sanchez, S., et al. (2023) · Science · doi:10.1126/science.abq5622 · Cited by 252

Naturally occurring modified ribonucleosides

McCown, P. J., Ruszkowska, A., Kunkler, C. N., Breger, K., Hulewicz, J. P., Wang, M. C., Springer, N. A., & Brown, J. A. (2020) · Wiley Interdisciplinary Reviews - RNA · doi:10.1002/wrna.1595 · Cited by 219

Bacteria loaded with glucose polymer and photosensitive ICG silicon-nanoparticles for glioblastoma photothermal immunotherapy

Sun, R., Liu, M., Lu, J., Chu, B., Yang, Y., Song, B., Wang, H., & He, Y. (2022) · Nature Communications · doi:10.1038/s41467-022-32837-5 · Cited by 192

Alternative splicing in neurodegenerative disease and the promise of RNA therapies

Nikom, D., & Zheng, S. (2023) · Nature reviews. Neuroscience · doi:10.1038/s41583-023-00717-6 · Cited by 201

Pathogenesis of SCA3 and implications for other polyglutamine diseases

McLoughlin, H. S., Moore, L. R., & Paulson, H. L. (2019) · Neurobiology of Disease · doi:10.1016/j.nbd.2019.104635 · Cited by 195

Design of a Randomized, Placebo-Controlled, Phase 3 Trial of Tofersen Initiated in Clinically Presymptomatic SOD1 Variant Carriers: the ATLAS Study

Benatar, M. G., Wuu, J., Andersen, P. M., Bucelli, R. C., Andrews, J. A., Otto, M., Farahany, N. A., Harrington, E. A., Chen, W., Mitchell, A. A., et al. (2022) · Neurotherapeutics · doi:10.1007/s13311-022-01237-4 · Cited by 182

How Is Precursor Messenger RNA Spliced by the Spliceosome?

Wan, R., Bai, R., Zhan, X., & Shi, Y. (2019) · Annual Review of Biochemistry · doi:10.1146/annurev-biochem-013118-111024 · Cited by 165

Nano drug delivery systems for antisense oligonucleotides (ASO) therapeutics

Ramasamy, T., Ruttala, H. B., Munusamy, S., Chakraborty, N., & Kim, J. O. (2022) · Journal of Controlled Release · doi:10.1016/j.jconrel.2022.10.050 · Cited by 156

Scn8a Antisense Oligonucleotide Is Protective in Mouse Models of SCN8A Encephalopathy and Dravet Syndrome

Lenk, G. M., Jafar‐Nejad, P., Hill, S. F., Huffman, L. D., Smolen, C., Wagnon, J. L., Petit, H. J., Yu, W., Ziobro, J. M., Bhatia, K., et al. (2020) · Annals of Neurology · doi:10.1002/ana.25676 · Cited by 150

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

The Interaction of Phosphorothioate-Containing RNA Targeted Drugs with Proteins Is a Critical Determinant of the Therapeutic Effects of These Agents

Crooke, S. T., Seth, P. P., Vickers, T. A., & Liang, X. (2020) · Journal of the American Chemical Society · doi:10.1021/jacs.0c04928 · Cited by 131

Regulated cell death: discovery, features and implications for neurodegenerative diseases

Cui, J., Zhao, S., Li, Y., Zhang, D., Wang, B., Xie, J., & Wang, J. (2021) · Cell Communication and Signaling · doi:10.1186/s12964-021-00799-8 · Cited by 130

Telomere length is key to hepatocellular carcinoma diversity and telomerase addiction is an actionable therapeutic target

Ningarhari, M., Caruso, S., Hirsch, T. Z., Bayard, Q., Franconi, A., Védié, A., Noblet, B., Blanc, J., Amaddeo, G., Ganne‐Carrié, N., et al. (2020) · Journal of Hepatology · doi:10.1016/j.jhep.2020.11.052 · Cited by 118

Absorption, Distribution, Metabolism, and Excretion of US Food and Drug Administration–Approved Antisense Oligonucleotide Drugs

Migliorati, J. M., Liu, S., Liu, A., Gogate, A., Nair, S., Bahal, R., Rasmussen, T. P., Manautou, J. E., & Zhong, X. (2022) · Drug Metabolism and Disposition · doi:10.1124/dmd.121.000417 · Cited by 110

Protein Misfolding and Aggregation in Proteinopathies: Causes, Mechanism and Cellular Response

Ajmal, M. R. (2023) · Diseases · doi:10.3390/diseases11010030 · Cited by 116

Non-canonical DNA structures: Diversity and disease association

Bansal, A., Kaushik, S., & Kukreti, S. (2022) · Frontiers in Genetics · doi:10.3389/fgene.2022.959258 · Cited by 111

Creutzfeldt–Jakob disease and other prion diseases

Zerr, I., Ladogana, A., Mead, S., Hermann, P., Forloni, G., & Appleby, B. S. (2024) · Nature Reviews Disease Primers · doi:10.1038/s41572-024-00497-y · Cited by 108

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

DNA Repair and Therapeutic Strategies in Cancer Stem Cells

Gillespie, M. S., Ward, C. M., & Davies, C. C. (2023) · Cancers · doi:10.3390/cancers15061897 · Cited by 101

Gene therapy for CNS disorders: modalities, delivery and translational challenges

Gao, J., Gunasekar, S., Xia, Z. J., Shalin, K., Jiang, C., Chen, H., Lee, D., Lee, S., Pisal, N. D., Luo, J. N., et al. (2024) · Nature reviews. Neuroscience · doi:10.1038/s41583-024-00829-7 · Cited by 100

Aβ plaques

Walker, L. C. (2020) · Free Neuropathology · doi:10.17879/freeneuropathology-2020-3025 · Cited by 113

Region-specific vulnerability in neurodegeneration: lessons from normal ageing

Pandya, V. A., & Patani, R. (2021) · Ageing Research Reviews · doi:10.1016/j.arr.2021.101311 · Cited by 98

Targeted gene silencing in the nervous system with CRISPR-Cas13

Powell, J. E., Lim, C. K. W., Krishnan, R. S. G., McCallister, T. X., Saporito-Magriñá, C., C., M. A. Z., McPheron, G. D., & Gaj, T. (2022) · Science Advances · doi:10.1126/sciadv.abk2485 · Cited by 96

Apolipoprotein E4 Reduction with Antisense Oligonucleotides Decreases Neurodegeneration in a Tauopathy Model

Litvinchuk, A., Huynh, T. V., Shi, Y., Jackson, R. J., Finn, M. B., Manis, M., Francis, C. M., Tran, A. C., Sullivan, P. M., Ulrich, J. D., et al. (2021) · Annals of Neurology · doi:10.1002/ana.26043 · Cited by 91

Fluid biomarkers for amyotrophic lateral sclerosis: a review

Irwin, K. E., Sheth, U., Wong, P. C., & Gendron, T. F. (2024) · Molecular Neurodegeneration · doi:10.1186/s13024-023-00685-6 · Cited by 95

Deliver the promise: RNAs as a new class of molecular entities for therapy and vaccination

Yu, A., & Tu, M. (2021) · Pharmacology & Therapeutics · doi:10.1016/j.pharmthera.2021.107967 · Cited by 94

Preparing n-of-1 Antisense Oligonucleotide Treatments for Rare Neurological Diseases in Europe: Genetic, Regulatory, and Ethical Perspectives

Synofzik, M., van Roon‐Mom, W., Marckmann, G., van Duyvenvoorde, H. A., Graeßner, H., Schüle, R., & Aartsma-Rus, A. M. (2021) · Nucleic Acid Therapeutics · doi:10.1089/nat.2021.0039 · Cited by 85

Overview of ADNI MRI

Jack, C. R., Arani, A., Borowski, B. J., Cash, D. M., Crawford, K., Das, S. R., DeCarli, C., Fletcher, E., Fox, N. C., Gunter, J. L., et al. (2024) · Alzheimer's & Dementia · doi:10.1002/alz.14166 · Cited by 82

Gene Therapy in Amyotrophic Lateral Sclerosis

Fang, T., Je, G., Pacut, P., Keyhanian, K., Gao, J., & Ghasemi, M. (2022) · Cells · doi:10.3390/cells11132066 · Cited by 88

Antisense oligonucleotides for Alzheimer's disease therapy: from the mRNA to miRNA paradigm

Grabowska, W., Want, A., Leszek, J., & Wojda, U. (2021) · EBioMedicine · doi:10.1016/j.ebiom.2021.103691 · Cited by 81

Mechanisms and Emerging Regulators of Neuroinflammation: Exploring New Therapeutic Strategies for Neurological Disorders

Kim, M. E., & Lee, J. S. (2024) · Current Issues in Molecular Biology · doi:10.3390/cimb47010008 · Cited by 82

Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy

Hammond, S. M., Abendroth, F., Goli, L., Burrell, M., Thom, G., Gurrell, I., Stoodley, J., Ahlskog, N., Gait, M. J., Wood, M. J. A., et al. (2022) · JCI Insight · doi:10.1172/jci.insight.154142 · Cited by 80

Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology

Amanat, M., Nemeth, C. L., Fine, A. S., Leung, D. G., & Fatemi, A. S. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14112389 · Cited by 78

Understanding Amyotrophic Lateral Sclerosis: Pathophysiology, Diagnosis, and Therapeutic Advances

Rizea, R. E., Corlatescu, A.-D., Costin, H. P., Dumitru, A. V., & Ciurea, A. V. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25189966 · Cited by 76

Circular RNA Encoded Amyloid Beta peptides—A Novel Putative Player in Alzheimer’s Disease

Mo, D., Li, X., Raabe, C. A., Rozhdestvensky, T. S., Skryabin, B. V., & Brosius, J. (2020) · Cells · doi:10.3390/cells9102196 · Cited by 74

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

Intrathecal Drug Delivery: Advances and Applications in the Management of Chronic Pain Patient

Andrés, J. A. D., Hayek, S. M., Perruchoud, C., Lawrence, M. M., Reina, M. Á., Andrés-Serrano, C. D., Rubio-Haro, R., Hunt, M. A., & Yaksh, T. L. (2022) · Frontiers in Pain Research · doi:10.3389/fpain.2022.900566 · Cited by 72

Bispecific antibody shuttles targeting CD98hc mediate efficient and long-lived brain delivery of IgGs

Pornnoppadol, G., Bond, L. G., Lucas, M. J., Zupancic, J. M., Kuo, Y., Zhang, B., Greineder, C. F., & Tessier, P. M. (2023) · Cell chemical biology · doi:10.1016/j.chembiol.2023.09.008 · Cited by 75

A microRNA signature that correlates with cognition and is a target against cognitive decline

Islam, M. R., Kaurani, L., Berulava, T., Heilbronner, U., Budde, M., Centeno, T. P., Elerdashvili, V., Zafieriou, M., Benito, E., Sertel, S. M., et al. (2021) · EMBO Molecular Medicine · doi:10.15252/emmm.202013659 · Cited by 74

Dysregulated miRNAs as Biomarkers and Therapeutical Targets in Neurodegenerative Diseases

Gentile, G., Morello, G., Cognata, V. L., Guarnaccia, M., Conforti, F. L., & Cavallaro, S. (2022) · Journal of Personalized Medicine · doi:10.3390/jpm12050770 · Cited by 71

A physiologically-based pharmacokinetic model to describe antisense oligonucleotide distribution after intrathecal administration

Monine, M. I., Norris, D. A., Wang, Y., & Nestorov, I. A. (2021) · Journal of Pharmacokinetics and Pharmacodynamics · doi:10.1007/s10928-021-09761-0 · Cited by 70

Cas9-mediated replacement of expanded CAG repeats in a pig model of Huntington’s disease

Yan, S., Zheng, X., Lin, Y., Li, C., Liu, Z., Li, J., Tu, Z., Zhao, Y., Huang, C., Chen, Y., et al. (2023) · Nature Biomedical Engineering · doi:10.1038/s41551-023-01007-3 · Cited by 69

Voltage-gated sodium channels in excitable cells as drug targets

Alsaloum, M., Dib‐Hajj, S. D., Page, D. A., Ruben, P. C., Krainer, A. R., & Waxman, S. G. (2025) · Nature Reviews Drug Discovery · doi:10.1038/s41573-024-01108-x · Cited by 69

Heterogeneous Nuclear Ribonucleoproteins: Implications in Neurological Diseases

Low, Y. H., Asi, Y. T., Foti, S. C., & Lashley, T. (2020) · Molecular Neurobiology · doi:10.1007/s12035-020-02137-4 · Cited by 69

Advances on Therapeutic Strategies for Alzheimer’s Disease: From Medicinal Plant to Nanotechnology

Hassan, N. A., Alshamari, A. K., Hassan, A. A., Elharrif, M. G., Alhajri, A. M., Sattam, M., & Khattab, R. R. (2022) · Molecules · doi:10.3390/molecules27154839 · Cited by 68

Current and emerging treatment modalities for spinocerebellar ataxias

Ghanekar, S. D., Kuo, S., Staffetti, J. S., & Zesiewicz, T. A. (2022) · Expert Review of Neurotherapeutics · doi:10.1080/14737175.2022.2029703 · Cited by 65

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

Antisense therapy in a rat model of Alexander disease reverses GFAP pathology, white matter deficits, and motor impairment

Hagemann, T. L., Powers, B. E., Lin, N., Mohamed, A. F., Dague, K. L., Hannah, S. C., Bachmann, G., Mazur, C., Rigo, F., Olsen, A. L., et al. (2021) · Science Translational Medicine · doi:10.1126/scitranslmed.abg4711 · Cited by 62

Inhibition of SARS-CoV-2 coronavirus proliferation by designer antisense-circRNAs

Pfafenrot, C., Schneider, T. P., Müller, C., Hung, L.-H., Schreiner, S., Ziebuhr, J., & Bindereif, A. (2021) · Nucleic Acids Research · doi:10.1093/nar/gkab1096 · Cited by 59

RNA-mediated toxicity in C9orf72 ALS and FTD

McEachin, Z. T., Parameswaran, J., Raj, N., Bassell, G. J., & Jiang, J. (2020) · Neurobiology of Disease · doi:10.1016/j.nbd.2020.105055 · Cited by 63

Causes, functions, and therapeutic possibilities of RNA secondary structure ensembles and alternative states

Bose, R., Saleem, I., & Mustoe, A. M. (2024) · Cell chemical biology · doi:10.1016/j.chembiol.2023.12.010 · Cited by 64

Role of Tau in Various Tauopathies, Treatment Approaches, and Emerging Role of Nanotechnology in Neurodegenerative Disorders

Kaur, P., Khera, A., Alajangi, H. K., Sharma, A., Jaiswal, P. K., Singh, G., & Barnwal, R. P. (2022) · Molecular Neurobiology · doi:10.1007/s12035-022-03164-z · Cited by 55

Alternative splicing coupled mRNA decay shapes the temperature‐dependent transcriptome

Neumann, A., Meinke, S., Goldammer, G., Strauch, M., Schubert, D. S. P., Timmermann, B., Heyd, F., & Preußner, M. (2020) · EMBO Reports · doi:10.15252/embr.202051369 · Cited by 54

Collective cancer cell invasion requires RNA accumulation at the invasive front

Chrisafis, G., Wang, T., Moissoglu, K., Gasparski, A. N., Ng, Y., Weigert, R., Lockett, S., & Mili, S. (2020) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.2010872117 · Cited by 49

U7 snRNA: A tool for gene therapy

Gadgil, A., & Raczyńska, K. D. (2021) · The Journal of Gene Medicine · doi:10.1002/jgm.3321 · Cited by 52

Diverse targets of SMN2 -directed splicing-modulating small molecule therapeutics for spinal muscular atrophy

Ottesen, E. W., Singh, N. N., Luo, D., Kaas, B., Gillette, B., Seo, J., Jorgensen, H. J., & Singh, R. N. (2023) · Nucleic Acids Research · doi:10.1093/nar/gkad259 · Cited by 49

Steering research on mRNA splicing in cancer towards clinical translation

Anczuków, O., Allain, F. H. ‐., Angarola, B. L., Black, D. L., Brooks, A. N., Cheng, C., Conesa, A., Crosse, E. I., Eyras, E., Guccione, E., et al. (2024) · Nature reviews. Cancer · doi:10.1038/s41568-024-00750-2 · Cited by 50

Autophagy‐associated non‐coding RNAs: Unraveling their impact on Parkinson's disease pathogenesis

Hussain, M. S., Moglad, E. H. O., Afzal, M., Sharma, S., Gupta, G., Sivaprasad, G. V., Deorari, M., Almalki, W. H., Kazmi, I. A., Alzarea, S. I., et al. (2024) · CNS Neuroscience & Therapeutics · doi:10.1111/cns.14763 · Cited by 47

Pre-mRNA splicing-associated diseases and therapies

Love, S. L., Emerson, J. D., Koide, K., & Hoskins, A. A. (2023) · RNA Biology · doi:10.1080/15476286.2023.2239601 · Cited by 50

Astrocytes in Parkinson’s Disease: From Role to Possible Intervention

Wang, T., Sun, Y., & Dettmer, U. (2023) · Cells · doi:10.3390/cells12192336 · Cited by 48

From basic research to the clinic: innovative therapies for ALS and FTD in the pipeline

Liščić, R. M., Alberici, A., Cairns, N. J., Romano, M., & Buratti, E. (2020) · Molecular Neurodegeneration · doi:10.1186/s13024-020-00373-9 · Cited by 48

Specificity, synergy, and mechanisms of splice-modifying drugs

Ishigami, Y., Wong, M. S., Martí‐Gómez, C., Ayaz, A., Kooshkbaghi, M., Hanson, S. M., McCandlish, D. M., Krainer, A. R., & Kinney, J. B. (2024) · Nature Communications · doi:10.1038/s41467-024-46090-5 · Cited by 45

Delta power robustly predicts cognitive function in Angelman syndrome

Ostrowski, L. M., Spencer, E. R., Bird, L. M., Thibert, R. L., Komorowski, R. W., Kramer, M., & Chu, C. J. (2021) · Annals of Clinical and Translational Neurology · doi:10.1002/acn3.51385 · Cited by 45

Opinion: more mouse models and more translation needed for ALS

Fisher, E., Greensmith, L., Malaspina, A., Fratta, P., Hanna, M. G., Schiavo, G., Isaacs, A. M., Orrell, R. W., Cunningham, T. J., & Acevedo‐Arozena, A. (2023) · Molecular Neurodegeneration · doi:10.1186/s13024-023-00619-2 · Cited by 45

Antisense Oligonucleotides (ASOs) in Motor Neuron Diseases: A Road to Cure in Light and Shade

Cantara, S., Simoncelli, G., & Ricci, C. (2024) · International Journal of Molecular Sciences · doi:10.3390/ijms25094809 · Cited by 44

Pathogenic Mechanisms and Therapy Development for C9orf72 Amyotrophic Lateral Sclerosis/Frontotemporal Dementia

Jiang, J., & Ravits, J. (2019) · Neurotherapeutics · doi:10.1007/s13311-019-00797-2 · Cited by 44

Therapeutic Strategies for Dystrophin Replacement in Duchenne Muscular Dystrophy

Mbakam, C. H., Lamothe, G., & Tremblay, J. P. (2022) · Frontiers in Medicine · doi:10.3389/fmed.2022.859930 · Cited by 46

Lipid nanoparticles and transcranial focused ultrasound enhance the delivery of SOD1 antisense oligonucleotides to the murine brain for ALS therapy

Ediriweera, G. R., Sivaram, A. J., Cowin, G., Brown, M. L., McAlary, L., Lum, J. S., Fletcher, N. L., Robinson, L., Simpson, J. D., Chen, L., et al. (2024) · Journal of Controlled Release · doi:10.1016/j.jconrel.2024.11.074 · Cited by 41

New and potential strategies for the treatment of PMM2-CDG

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ASO targeting RBM3 temperature‐controlled poison exon splicing prevents neurodegeneration in vivo

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