Antisense drugs make sense for neurological diseases
Open accessAbstract
The genetic basis for most inherited neurodegenerative diseases has been identified, yet there are limited disease-modifying therapies for these patients. A new class of drugs—antisense oligonucleotides (ASOs)—show promise as a therapeutic platform for treating neurological diseases. ASOs are designed to bind to the RNAs either by promoting degradation of the targeted RNA or by elevating expression by RNA splicing. Intrathecal injection into the cerebral spinal fluid results in broad distribution of antisense drugs and long-term effects. Approval of nusinersen in 2016 demonstrated that effective treatments for neurodegenerative diseases can be identified and that treatments not only slow disease progression but also improve some symptoms. Antisense drugs are currently in development for amyotrophic lateral sclerosis, Huntington's disease, Alzheimer's disease, Parkinson's disease, and Angelman syndrome, and several drugs are in late-stage research for additional neurological diseases. This review highlights the advances in antisense technology as potential treatments for neurological diseases.
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Integrity signals
Registry
Cites
Statistics
Claims the paper makes
The claims and statistics of this paper haven’t been checked yet.
Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.
Claims other papers make about this one
Each is checked against this paper’s own text: does it say what the citing paper says it does?
The FDA has approved eight ASO drugs since 2016, and the approval of nusinersen provides a strong basis for using ASO technology to develop novel therapies for neurodegenerative diseases.
“The FDA has approved eight ASO drugs since 2016, and the approval of the ASO-based drug nusinersen provides a strong basis for using this technology to develop novel and effective therapies for neurodegenerative diseases”
From ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease (2022) · cites this paper as Bennett et al 2021
Split into 2 atoms — 1 confirmed, 1 partially supported, 0 disputed, 0 misattributed, 0 unverifiable. Expand the claim to see per-atom verdicts.
Experimental — accuracy not yet measuredASO targeting MAPT (Tau) for AD treatment is under Phase 1/2 clinical trial.
“Currently, ASO targeting MAPT (Tau) for AD treatment is under Phase 1/2 clinical trial”
From ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease (2022) · cites this paper as Bennett et al 2021
Self-check
Percentages, sums and reported differences re-computed from the paper’s own numbers.
The self-check needs the paper’s full text.
Cites
Staging of brain pathology related to sporadic Parkinson’s disease
Braak, H., Tredici, K. D., Rüb, U., de Vos, R. A. I., Jansen Steur, E. N. H., & Braak, E. (2003) · Neurobiology of Aging · doi:10.1016/s0197-4580(02)00065-9 · Cited by 10,777 · see citing works
Amyloid oligomers exacerbate tau pathology in a mouse model of tauopathy
Selenica, M.-L. B., Brownlow, M., Jimenez, J. P., Lee, D. C., Pena, G., Dickey, C. A., Gordon, M. N., & Morgan, D. (2013) · Neurodegenerative Diseases · doi:10.1159/000337230 · Cited by 10,359 · see citing works
Polazzi, E., Mengoni, I., Caprini, M., Peña‐Altamira, E., Kurtys, E., & Monti, B. (2012) · Neurosignals · doi:10.1159/000337115 · Cited by 9,023 · see citing works
MACDONALD, M. (1993) · Cell · doi:10.1016/0092-8674(93)90585-e · Cited by 8,366 · see citing works
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 · see citing works
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 · see citing works
Iliff, J. J., Wang, M., Liao, Y., Plogg, B. A., Peng, W., Gundersen, G. A., Benveniste, H., Vates, G. E., Deane, R., Goldman, S. A., et al. (2012) · Science Translational Medicine · doi:10.1126/scitranslmed.3003748 · Cited by 5,824 · cited 1× · see citing works
“The exchange of CSF and interstitial fluid is facilitated by convective influx of CSF along the periarterial space into the brain parenchyma through the glymphatic system, with these convective forces driving movement of macromolecules through the parenchyma (52).”
BackgroundPoewe, W., Seppi, K., Tanner, C. M., Halliday, G. M., Brundin, P., Volkmann, J., Schrag, A.-E., & Lang, A. E. (2017) · Nature Reviews Disease Primers · doi:10.1038/nrdp.2017.13 · Cited by 5,415 · see citing works
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 · see citing works
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 · see citing works
Motor Neuron Degeneration in Mice that Express a Human Cu,Zn Superoxide Dismutase Mutation
Gurney, M. E., Pu, H., Chiu, A. Y., Dal Canto, M. C., Polchow, C. Y., Alexander, D. D., Caliendo, J., Hentati, A., Kwon, Y. W., Deng, H.-X., et al. (1994) · Science · doi:10.1126/science.8209258 · Cited by 4,143 · see citing works
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 · see citing works
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 · see citing works
Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
Adams, D., Gonzalez-Duarte, A., O’Riordan, W. D., Yang, C.-C., Ueda, M., Kristen, A. V., Tournev, I., Schmidt, H. H., Coelho, T., Berk, J. L., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716153 · Cited by 3,047 · see citing works
Luk, K. C., Kehm, V., Carroll, J., Zhang, B., O’Brien, P., Trojanowski, J. Q., & Lee, V. M.-Y. (2012) · Science · doi:10.1126/science.1227157 · Cited by 2,563 · see citing works
Brown, R. H., & Al‐Chalabi, A. (2017) · New England Journal of Medicine · doi:10.1056/nejmra1603471 · Cited by 2,472 · see citing works
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 · see citing works
Self-citationThe glymphatic system: a beginner's guide
Jessen, N. A., Munk, A. S. F., Lundgaard, I., & Nedergaard, M. (2015) · Neurochemical Research · doi:10.1007/s11064-015-1581-6 · Cited by 2,075 · see citing works
Decoding ALS: from genes to mechanism
Taylor, J. P., Brown, R. H., & Cleveland, D. W. (2016) · Nature · doi:10.1038/nature20413 · Cited by 2,047 · see citing works
Self-citationReducing Endogenous Tau Ameliorates Amyloid ß-Induced Deficits in an Alzheimer's Disease Mouse Model
Roberson, E. D., Scearce-Levie, K., Palop, J. J., Yan, F., Cheng, I. H., Wu, T., Gerstein, H., Yu, G.-Q., & Mucke, L. (2007) · Science · doi:10.1126/science.1141736 · Cited by 1,892 · see citing works
Onset and Progression in Inherited ALS Determined by Motor Neurons and Microglia
Boillée, S., Yamanaka, K., Lobsiger, C. S., Copeland, N. G., Jenkins, N. A., Kassiotis, G., Kollias, G., & Cleveland, D. W. (2006) · Science · doi:10.1126/science.1123511 · Cited by 1,623 · see citing works
Self-citationInotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
Benson, M. D., Waddington‐Cruz, M., Berk, J. L., Polydefkis, M. J., Dyck, P. J. B., Wang, A. K., Planté‐Bordeneuve, V., Barroso, F. A., Merlini, G., Obici, L. P., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1716793 · Cited by 1,452 · see citing works
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
Mercuri, E., Darras, B. T., Chiriboga, C. A., Day, J. W., Campbell, C., Connolly, A. M., Iannaccone, S. T., Kirschner, J., Kuntz, N. L., Saito, K., et al. (2018) · New England Journal of Medicine · doi:10.1056/nejmoa1710504 · Cited by 1,433 · see citing works
Self-citationBennett, C. F., & Swayze, E. E. (2010) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev.pharmtox.010909.105654 · Cited by 1,381 · see citing works
Self-citationUNRAVELING THE MECHANISMS INVOLVED IN MOTOR NEURON DEGENERATION IN ALS
Bruijn, L. I., Miller, T. M., & Cleveland, D. W. (2004) · Annual Review of Neuroscience · doi:10.1146/annurev.neuro.27.070203.144244 · Cited by 1,371 · see citing works
Self-citationTrinucleotide Repeat Disorders
Orr, H. T., & Zoghbi, H. Y. (2007) · Annual Review of Neuroscience · doi:10.1146/annurev.neuro.29.051605.113042 · Cited by 1,365 · see citing works
Bruijn, L. I., Becher, M. W., Lee, M. K., Anderson, K. L., Jenkins, N. A., Copeland, N. G., Sisodia, S. S., Rothstein, J. D., Borchelt, D. R., Price, D. L., et al. (1997) · Neuron · doi:10.1016/s0896-6273(00)80272-x · Cited by 1,341 · see citing works
Self-citationLong pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43
Polymenidou, M., Lagier-Tourenne, C., Hutt, K. R., Huelga, S. C., Moran, J., Liang, T. Y., Ling, S.-C., Sun, E., Wancewicz, E., Mazur, C., et al. (2011) · Nature Neuroscience · doi:10.1038/nn.2779 · Cited by 1,335 · see citing works
Self-citationAtaxin-2 intermediate-length polyglutamine expansions are associated with increased risk for ALS
Elden, A. C., Kim, H.-J., Hart, M. P., Chen‐Plotkin, A. S., Johnson, B. S., Fang, X., Armakola, M., Geser, F., Greene, R. A., Lu, M. M., et al. (2010) · Nature · doi:10.1038/nature09320 · Cited by 1,327 · see citing works
The C9orf72 GGGGCC Repeat Is Translated into Aggregating Dipeptide-Repeat Proteins in FTLD/ALS
Mori, K., Weng, S., Arzberger, T., May, S., Rentzsch, K., Kremmer, E., Schmid, B., Kretzschmar, H. A., Cruts, M., Broeckhoven, C. V., et al. (2013) · Science · doi:10.1126/science.1232927 · Cited by 1,248 · see citing works
Aggregation and Motor Neuron Toxicity of an ALS-Linked SOD1 Mutant Independent from Wild-Type SOD1
Bruijn, L. I., Houseweart, M. K., Kato, S., Anderson, K. L., Anderson, S., Ohama, E., Reaume, A. G., Scott, R. W., & Cleveland, D. W. (1998) · Science · doi:10.1126/science.281.5384.1851 · Cited by 1,231 · see citing works
Self-citationCorrelation between severity and SMN protein level in spinal muscular atrophy
Lefebvre, S., Burlet, P., Liu, Q., Bertrandy, S., Clermont, O., Münnich, A., Dreyfuss, G., & Melki, J. (1997) · Nature Genetics · doi:10.1038/ng0797-265 · Cited by 1,100 · see citing works
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 1,019 · see citing works
Self-citationMicroRNA-143 Regulates Adipocyte Differentiation
Esau, C., Kang, X., Peralta, E. R., Hanson, E. M., Marcusson, E. G., Ravichandran, L. V., Sun, Y., Koo, S., Perera, R. J., Jain, R., et al. (2004) · Journal of Biological Chemistry · doi:10.1074/jbc.c400438200 · Cited by 1,017 · see citing works
Self-citationSaudou, F., & Humbert, S. (2016) · Neuron · doi:10.1016/j.neuron.2016.02.003 · Cited by 945 · see citing works
RNA Toxicity from the ALS/FTD C9ORF72 Expansion Is Mitigated by Antisense Intervention
Donnelly, C. J., Zhang, P.-W., Pham, J. T., Haeusler, A. R., Mistry, N. A., Vidensky, S., Daley, E. L., Poth, E. M., Hoover, B., Fines, D. M., et al. (2013) · Neuron · doi:10.1016/j.neuron.2013.10.015 · Cited by 877 · see citing works
Self-citationCrooke, 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 · see citing works
Self-citationPatient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
Kim, J., Hu, C., Achkar, C. M. E., Black, L. E., Douville, J., Larson, A. A., Pendergast, M. K., Goldkind, S. F., Lee, E. A., Kuniholm, A., et al. (2019) · New England Journal of Medicine · doi:10.1056/nejmoa1813279 · Cited by 835 · see citing works
Eteplirsen for the treatment of Duchenne muscular dystrophy
Mendell, J. R., Rodino‐Klapac, L. R., Sahenk, Z., Roush, K., Bird, L., Lowes, L. P., Alfano, L. N., Gomez, A. M., Lewis, S., Kota, J., et al. (2013) · Annals of Neurology · doi:10.1002/ana.23982 · Cited by 819 · see citing works
A new look at cerebrospinal fluid circulation
Brinker, T., Stopa, E., Morrison, J., & Klinge, P. (2014) · Fluids and Barriers of the CNS · doi:10.1186/2045-8118-11-10 · Cited by 819 · see citing works
Ribonuclease H: the enzymes in eukaryotes
Cerritelli, S. M., & Crouch, R. J. (2009) · FEBS Journal · doi:10.1111/j.1742-4658.2009.06908.x · Cited by 816 · see citing works
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 · see citing works
Self-citationHua, Y., Sahashi, K., Rigo, F., Hung, G., Horev, G., Bennett, C. F., & Krainer, A. R. (2011) · Nature · doi:10.1038/nature10485 · Cited by 744 · see citing works
Self-citationTargeting 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 · see citing works
Self-citationObika, S., Nanbu, D., Hari, Y., Andoh, J., Morio, K., Doi, T., & Imanishi, T. (1998) · Tetrahedron Letters · doi:10.1016/s0040-4039(98)01084-3 · Cited by 701 · see citing works
Freier, S. (1997) · Nucleic Acids Research · doi:10.1093/nar/25.22.4429 · Cited by 692 · see citing works
TDP-43 repression of nonconserved cryptic exons is compromised in ALS-FTD
Ling, J. P., Pletniková, O., Troncoso, J. C., & Wong, P. C. (2015) · Science · doi:10.1126/science.aab0983 · Cited by 673 · see citing works
Sareen, D., O’Rourke, J. G., Meera, P., Muhammad, A. K. M. G., Grant, S., Simpkinson, M., Bell, S., Carmona, S., Ornelas, L., Sahabian, A., et al. (2013) · Science Translational Medicine · doi:10.1126/scitranslmed.3007529 · Cited by 670 · see citing works
Self-citationAntisense 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 665 · see citing works
Self-citationALS-implicated protein TDP-43 sustains levels of STMN2, a mediator of motor neuron growth and repair
Klim, J. R., Williams, L. A., Limone, F., Guerra San Juan, I., Davis-Dusenbery, B. N., Mordes, D. A., Burberry, A., Steinbaugh, M. J., Gamage, K. K., Kirchner, R., et al. (2019) · Nature Neuroscience · doi:10.1038/s41593-018-0300-4 · Cited by 653 · see citing works
Melamed, Z., López-Erauskin, J., Baughn, M. W., Zhang, O., Drenner, K., Sun, Y., Freyermuth, F., McMahon, M. A., Beccari, M. S., Artates, J. W., et al. (2019) · Nature Neuroscience · doi:10.1038/s41593-018-0293-z · Cited by 635 · see citing works
Self-citationVivo, D. C. D., Bertini, E., Swoboda, K. J., Hwu, W., Crawford, T. O., Finkel, R. S., Kirschner, J., Kuntz, N. L., Parsons, J. A., Ryan, M. M., et al. (2019) · Neuromuscular Disorders · doi:10.1016/j.nmd.2019.09.007 · Cited by 634 · see citing works
Miller, T. M., Pestronk, A., David, W., Rothstein, J., Simpson, E., Appel, S. H., Andres, P. L., Mahoney, K., Allred, P., Alexander, K., et al. (2013) · The Lancet Neurology · doi:10.1016/s1474-4422(13)70061-9 · Cited by 617 · see citing works
Self-citationResults from a phase 1 study of nusinersen (ISIS-SMN Rx ) in children with spinal muscular atrophy
Chiriboga, C. A., Swoboda, K. J., Darras, B. T., Iannaccone, S. T., Montes, J., Vivo, D. C. D., Norris, D. A., Bennett, C. F., & Bishop, K. M. (2016) · Neurology · doi:10.1212/wnl.0000000000002445 · Cited by 613 · see citing works
Self-citationTherapeutic Antisense Oligonucleotides Are Coming of Age
Bennett, C. F. (2019) · Annual Review of Medicine · doi:10.1146/annurev-med-041217-010829 · Cited by 584 · see citing works
Self-citationTDP-43 pathology disrupts nuclear pore complexes and nucleocytoplasmic transport in ALS/FTD
Chou, C., Zhang, Y., Umoh, M. E., Vaughan, S. W., Lorenzini, I., Liu, F., Sayegh, M., Donlin-Asp, P., Chen, Y. H., Duong, D. M., et al. (2018) · Nature Neuroscience · doi:10.1038/s41593-017-0047-3 · Cited by 583 · see citing works
Hua, Y., Vickers, T. A., Okunola, H. L., Bennett, C. F., & Krainer, A. R. (2008) · The American Journal of Human Genetics · doi:10.1016/j.ajhg.2008.01.014 · Cited by 576 · see citing works
Self-citationTherapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice
Becker, L. A., Huang, B., Bieri, G., Ma, R., Knowles, D. A., Jafar-Nejad, P., Messing, J., Kim, H. J., Soriano, A., Auburger, G., et al. (2017) · Nature · doi:10.1038/nature22038 · Cited by 571 · see citing works
Phase 1–2 Trial of Antisense Oligonucleotide Tofersen for <i>SOD1</i> ALS
Miller, T., Cudkowicz, M., Shaw, P. J., Andersen, P. M., Atassi, N., Bucelli, R. C., Genge, A., Glass, J., Ladha, S., Ludolph, A. L., et al. (2020) · New England Journal of Medicine · doi:10.1056/nejmoa2003715 · Cited by 565 · see citing works
Self-citationLagier‐Tourenne, C., Baughn, M. W., Rigo, F., Sun, S., Liu, P., Li, H., Jiang, J., Watt, A. T., Chun, S. J., Katz, M., et al. (2013) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1318835110 · Cited by 555 · see citing works
Self-citationPassini, M. A., Bu, J., Richards, A. M., Kinnecom, C., Sardi, S. P., Stanek, L. M., Hua, Y., Rigo, F., Matson, J. E., Hung, G., et al. (2011) · Science Translational Medicine · doi:10.1126/scitranslmed.3001777 · Cited by 547 · see citing works
Self-citationJiang, J., Zhu, Q., Gendron, T. F., Saberi, S., McAlonis-Downes, M., Seelman, A., Stauffer, J. E., Jafar-nejad, P., Drenner, K., Schulte, D., et al. (2016) · Neuron · doi:10.1016/j.neuron.2016.04.006 · Cited by 536 · see citing works
Self-citationEpidemiology of mutations in superoxide dismutase in amyotrophic lateal sclerosis
Cudkowicz, M., McKenna‐Yasek, D., Sapp, P. C., Chin, W., Geller, B. A., Hayden, D. L., Schoenfeld, D., Hosler, B. A., Horvitz, H. R., & Brown, R. H. (1997) · Annals of Neurology · doi:10.1002/ana.410410212 · Cited by 533 · see citing works
Observational study of spinal muscular atrophy type I and implications for clinical trials
Finkel, R. S., McDermott, M., Kaufmann, P., Darras, B. T., Chung, W. K., Sproule, D. M., Kang, P. B., Foley, A. R., Yang, M. L., Martens, W. B., et al. (2014) · Neurology · doi:10.1212/wnl.0000000000000741 · Cited by 530 · see citing works
Towards a therapy for Angelman syndrome by targeting a long non-coding RNA
Meng, L., Ward, A. J., Chun, S. J., Bennett, C. F., Beaudet, A. L., & Rigo, F. (2014) · Nature · doi:10.1038/nature13975 · Cited by 520 · see citing works
Self-citationTarget Protectors Reveal Dampening and Balancing of Nodal Agonist and Antagonist by miR-430
Choi, W.-Y., Giráldez, A. J., & Schier, A. F. (2007) · Science · doi:10.1126/science.1147535 · Cited by 517 · see citing works
DeVos, S. L., Miller, R. L., Schoch, K. M., Holmes, B. B., Kebodeaux, C. S., Wegener, A. J., Chen, G., Shen, T., Tran, H., Nichols, B., et al. (2017) · Science Translational Medicine · doi:10.1126/scitranslmed.aag0481 · Cited by 516 · see citing works
Self-citationAntisense oligonucleotide therapy for neurodegenerative disease
Smith, R. A. (2006) · Journal of Clinical Investigation · doi:10.1172/jci25424 · Cited by 506 · see citing works
Inhibition of axonal growth by SNAP-25 antisense oligonucleotides in vitro and in vivo
Osen‐Sand, A., Catsicas, M., Staple, J. K., Jones, K. A., Ayala, G., Knowles, J., Grenningloh, G., & Catsicas, S. (1993) · Nature · doi:10.1038/364445a0 · Cited by 442 · see citing works
Pharmacology of Antisense Drugs
Bennett, C. F., Baker, B. F., Pham, N. C., Swayze, E. E., & Geary, R. S. (2016) · The Annual Review of Pharmacology and Toxicology · doi:10.1146/annurev-pharmtox-010716-104846 · Cited by 429 · see citing works
Self-citationRestoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides.
Domiński, Z., & Kole, R. (1993) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.90.18.8673 · Cited by 426 · see citing works
Scott, L. J. (2020) · Drugs · doi:10.1007/s40265-020-01269-0 · Cited by 422 · see citing works
The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy
Webster, C. P., Smith, E. F., Bauer, C. S., Moller, A., Hautbergue, G. M., Ferraiuolo, L., Myszczynska, M. A., Higginbottom, A., Walsh, M. J., Whitworth, A. J., et al. (2016) · The EMBO Journal · doi:10.15252/embj.201694401 · Cited by 404 · see citing works
Phosphorothioate Oligodeoxynucleotides: What Is Their Origin and What Is Unique About Them?
Eckstein, F. (2000) · Antisense and Nucleic Acid Drug Development · doi:10.1089/oli.1.2000.10.117 · Cited by 383 · see citing works
Therapeutic developments for Duchenne muscular dystrophy
Verhaart, I. E. C., & Aartsma-Rus, A. M. (2019) · Nature Reviews Neurology · doi:10.1038/s41582-019-0203-3 · Cited by 378 · see citing works
Wahlestedt, C., Golanov, E. V., Yamamoto, S., Yee, F., Ericson, H., Yoo, H., Inturrisi, C. E., & Reis, D. J. (1993) · Nature · doi:10.1038/363260a0 · Cited by 372 · see citing works
Spinocerebellar ataxia: an update
Sullivan, R., Yau, W. Y., O’Connor, E., & Houlden, H. (2018) · Journal of Neurology · doi:10.1007/s00415-018-9076-4 · Cited by 353 · see citing works
LNA (Locked Nucleic Acid): An RNA Mimic Forming Exceedingly Stable LNA:LNA Duplexes
Koshkin, A. A., Nielsen, P., Meldgaard, M., Rajwanshi, V. K., Singh, S. K., & Wengel, J. (1998) · Journal of the American Chemical Society · doi:10.1021/ja9822862 · Cited by 336 · see citing works
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
Frank, D. E., Schnell, F. J., Akana, C., El-Husayni, S. H., Desjardins, C. A., Morgan, J. E., Charleston, J. S., Sardone, V., Domingos, J., Dickson, G., et al. (2020) · Neurology · doi:10.1212/wnl.0000000000009233 · Cited by 332 · see citing works
Antisense oligonucleotide therapy for spinocerebellar ataxia type 2
Scoles, D. R., Meera, P., Schneider, M. D., Paul, S., Dansithong, W., Figueroa, K. P., Hung, G., Rigo, F., Bennett, C. F., Otis, T. S., et al. (2017) · Nature · doi:10.1038/nature22044 · Cited by 319 · see citing works
Self-citationC9ORF72 poly(GA) aggregates sequester and impair HR23 and nucleocytoplasmic transport proteins
Zhang, Y., Gendron, T. F., Grima, J. C., Sasaguri, H., Jansen‐West, K. R., Xu, Y.-F., Katzman, R. B., Gass, J. M., Murray, M. E., Shinohara, M., et al. (2016) · Nature Neuroscience · doi:10.1038/nn.4272 · Cited by 315 · see citing works
Self-citationAntisense Reduction of Tau in Adult Mice Protects against Seizures
DeVos, S. L., Goncharoff, D. K., Chen, G., Kebodeaux, C. S., Yamada, K., Stewart, F. R., Schuler, D. R., Maloney, S. E., Wozniak, D. F., Rigo, F., et al. (2013) · Journal of Neuroscience · doi:10.1523/jneurosci.2107-13.2013 · Cited by 311 · see citing works
Self-citationCarroll, J. B., Warby, S. C., Southwell, A. L., Doty, C. N., Greenlee, S., Skotte, N. H., Hung, G., Bennett, C. F., Freier, S. M., & Hayden, M. R. (2011) · Molecular Therapy · doi:10.1038/mt.2011.201 · Cited by 294 · see citing works
Self-citationAntisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells
van Deutekom, J. C. T. (2001) · Human Molecular Genetics · doi:10.1093/hmg/10.15.1547 · Cited by 293 · see citing works
Koller, E., Vincent, T. M., Chappell, A. E., De, S., Manoharan, M., & Bennett, C. F. (2011) · Nucleic Acids Research · doi:10.1093/nar/gkr089 · Cited by 284 · see citing works
Self-citationRigo, F., Chun, S. J., Norris, D. A., Hung, G., Lee, S., Matson, J., Fey, R. A., Gaus, H., Hua, Y., Grundy, J. S., et al. (2014) · The Journal of Pharmacology and Experimental Therapeutics · doi:10.1124/jpet.113.212407 · Cited by 283 · see citing works
Self-citationAntisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models
McCampbell, A., Cole, T., Wegener, A. J., Tomassy, G. S., Setnicka, A., Farley, B. J., Schoch, K. M., Hoye, M. L., Shabsovich, M., Sun, L., et al. (2018) · Journal of Clinical Investigation · doi:10.1172/jci99081 · Cited by 279 · see citing works
Self-citationYu, D., Pendergraff, H. M., Liu, J., Kordasiewicz, H. B., Cleveland, D. W., Swayze, E. E., Lima, W. F., Crooke, S. T., Prakash, T. P., & Corey, D. R. (2012) · Cell · doi:10.1016/j.cell.2012.08.002 · Cited by 277 · see citing works
Self-citationGendron, T. F., Chew, J., Stankowski, J. N., Hayes, L. R., Zhang, Y.-J., Prudencio, M., Carlomagno, Y., Daughrity, L. M., Jansen-West, K., Perkerson, E. A., et al. (2017) · Science Translational Medicine · doi:10.1126/scitranslmed.aai7866 · Cited by 265 · see citing works
Nusinersen in later-onset spinal muscular atrophy
Darras, B. T., Chiriboga, C. A., Iannaccone, S. T., Swoboda, K. J., Montes, J., Mignon, L., Xia, S., Bennett, C. F., Bishop, K. M., Shefner, J. M., et al. (2019) · Neurology · doi:10.1212/wnl.0000000000007527 · Cited by 262 · see citing works
Self-citationPfister, E. L., Kennington, L., Straubhaar, J., Wagh, S., Liu, W., DiFiglia, M., Landwehrmeyer, B., Vonsattel, J.-P., Zamore, P. D., & Aronin, N. (2009) · Current Biology · doi:10.1016/j.cub.2009.03.030 · Cited by 261 · see citing works
Hu, J., Matsui, M., Gagnon, K. T., Schwartz, J. C., Gabillet, S., Arar, K., Wu, J., Борисович, Б. И., & Corey, D. R. (2009) · Nature Biotechnology · doi:10.1038/nbt.1539 · Cited by 242 · see citing works
Comparative inhibition of rabbit globin mRNA translation by modified antisense oligodeoxynucleotides
Cazenave, C., Stein, C. A., Loreau, N., Thuong, N. T., Neckers, L., Subasinghe, C., Hélène, C., Cohen, J. S., & Toulmé, J. (1989) · Nucleic Acids Research · doi:10.1093/nar/17.11.4255 · Cited by 238 · see citing works
Eteplirsen treatment for Duchenne muscular dystrophy
Charleston, J. S., Schnell, F. J., Dworzak, J., Donoghue, C., Lewis, S., Chen, L., Young, G. D., Milici, A. J., Voss, J., DeAlwis, U., et al. (2018) · Neurology · doi:10.1212/wnl.0000000000005680 · Cited by 232 · see citing works
Alterman, J. F., Godinho, B. M. D. C., Hassler, M. R., Ferguson, C. M., Echeverria, D., Sapp, E., Haraszti, R. A., Coles, A. H., Conroy, F., Miller, R., et al. (2019) · Nature Biotechnology · doi:10.1038/s41587-019-0205-0 · Cited by 227 · cited 1× · see citing works
“To date, we know of no siRNA drugs in clinical development for central nervous system diseases, although progress is being made optimizing design and delivery of siRNAs for CNS diseases (18).”
BackgroundAntisense Drug Technology: Principles, Strategies, and Applications
Szymkowski, D. E. (2002) · Drug Discovery Today · doi:10.1016/s1359-6446(01)02123-7 · Cited by 221 · see citing works
Zhu, Q., Jiang, J., Gendron, T. F., McAlonis-Downes, M., Jiang, L., Taylor, A., Diaz Garcia, S., Ghosh Dastidar, S., Rodriguez, M. J., King, P., et al. (2020) · Nature Neuroscience · doi:10.1038/s41593-020-0619-5 · Cited by 220 · see citing works
Self-citationDrowning stars: reassessing the role of astrocytes in brain edema
Thrane, A. S., Thrane, V. R., & Nedergaard, M. (2014) · Trends in Neurosciences · doi:10.1016/j.tins.2014.08.010 · Cited by 218 · see citing works
Zhao, H. T., John, N., Delic, V., Ikeda-Lee, K., Kim, A., Weihofen, A., Swayze, E. E., Kordasiewicz, H. B., West, A. B., & Volpicelli‐Daley, L. A. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.08.002 · Cited by 217 · see citing works
Self-citationLiang, X., Shen, W., Sun, H., Migawa, M. T., Vickers, T. A., & Crooke, S. T. (2016) · Nature Biotechnology · doi:10.1038/nbt.3589 · Cited by 214 · see citing works
Seth, P. P., Vasquez, G., Allerson, C. A., Berdeja, A., Gaus, H. J., Kinberger, G. A., Prakash, T. P., Migawa, M. T., Bhat, B., & Swayze, E. E. (2010) · The Journal of Organic Chemistry · doi:10.1021/jo902560f · Cited by 211 · see citing works
Prion-like Properties of Tau Protein: The Importance of Extracellular Tau as a Therapeutic Target
Holmes, B. B., & Diamond, M. I. (2014) · Journal of Biological Chemistry · doi:10.1074/jbc.r114.549295 · Cited by 202 · see citing works
Kumar, V. B., Farr, S. A., Flood, J. F., Kamlesh, V., Franko, M. W., Banks, W. A., & Morley, J. E. (2000) · Peptides · doi:10.1016/s0196-9781(00)00339-9 · Cited by 193 · see citing works
Age-dependent SMN expression in disease-relevant tissue and implications for SMA treatment
Ramos, D. M., d’Ydewalle, C. V. O., Gabbeta, V., Dakka, A., Klein, S., Norris, D. A., Matson, J. E., Taylor, S. J., Zaworski, P. G., Prior, T. W., et al. (2019) · Journal of Clinical Investigation · doi:10.1172/jci124120 · Cited by 190 · see citing works
Self-citationLiang, X., Sun, H., Shen, W., & Crooke, S. T. (2015) · Nucleic Acids Research · doi:10.1093/nar/gkv143 · Cited by 184 · see citing works
Increased 4R-Tau Induces Pathological Changes in a Human-Tau Mouse Model
Schoch, K. M., DeVos, S. L., Miller, R. L., Chun, S. J., Norrbom, M., Wozniak, D. F., Dawson, H. N., Bennett, C. F., Rigo, F., & Miller, T. M. (2016) · Neuron · doi:10.1016/j.neuron.2016.04.042 · Cited by 175 · see citing works
Self-citationLieberman, A. P., Yu, Z., Murray, S., Peralta, R., Low, A., Guo, S., Yu, X. X., Cortes, C. J., Bennett, C. F., Monia, B. P., et al. (2014) · Cell Reports · doi:10.1016/j.celrep.2014.02.008 · Cited by 169 · see citing works
Self-citationLRRK2 pathobiology in Parkinson's disease
Martin, I., Kim, J. W., Dawson, V. L., & Dawson, T. M. (2014) · Journal of Neurochemistry · doi:10.1111/jnc.12949 · Cited by 168 · see citing works
Properties of Cloned and Expressed Human RNase H1
Wu, H., Lima, W. F., & Crooke, S. T. (1999) · Journal of Biological Chemistry · doi:10.1074/jbc.274.40.28270 · Cited by 159 · see citing works
α-Synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s disease
Cole, T. A., Zhao, H. T., Collier, T. J., Sandoval, I. M., Sortwell, C. E., Steece‐Collier, K., Daley, B. F., Booms, A., Lipton, J. W., Welch, M., et al. (2021) · JCI Insight · doi:10.1172/jci.insight.135633 · Cited by 157 · see citing works
Self-citationSouthwell, A. L., Skotte, N. H., Kordasiewicz, H. B., Østergaard, M. E., Watt, A. T., Carroll, J. B., Doty, C. N., Villanueva, E. B., Petoukhov, E., Vaid, K., et al. (2014) · Molecular Therapy · doi:10.1038/mt.2014.153 · Cited by 144 · see citing works
Self-citationAntisense oligonucleotides extend survival of prion-infected mice
Raymond, G. J., Zhao, H. T., Race, B., Raymond, L. D., Williams, K., Swayze, E. E., Graffam, S., Le, J., Caron, T. J., Stathopoulos, J., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.131175 · Cited by 144 · see citing works
Self-citationLiang, X., Sun, H., Shen, W., Wang, S., Yao, J., Migawa, M. T., Bui, H., Damle, S. S., Riney, S., Graham, M. J., et al. (2017) · Nucleic Acids Research · doi:10.1093/nar/gkx632 · Cited by 142 · see citing works
Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice
McLoughlin, H. S., Moore, L. R., Chopra, R., Komlo, R., McKenzie, M., Blumenstein, K. G., Zhao, H. T., Kordasiewicz, H. B., Shakkottai, V. G., & Paulson, H. L. (2018) · Annals of Neurology · doi:10.1002/ana.25264 · Cited by 140 · see citing works
Self-citationEvaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse Models
Moore, L. R., Rajpal, G., Dillingham, I. T., Qutob, M., Blumenstein, K. G., Gattis, D., Hung, G., Kordasiewicz, H. B., Paulson, H. L., & McLoughlin, H. S. (2017) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2017.04.005 · Cited by 137 · see citing works
Self-citationFully modified 2' MOE oligonucleotides redirect polyadenylation
Vickers, T. A. (2001) · Nucleic Acids Research · doi:10.1093/nar/29.6.1293 · Cited by 127 · see citing works
Huntingtin suppression restores cognitive function in a mouse model of Huntington’s disease
Southwell, A. L., Kordasiewicz, H. B., Langbehn, D., Skotte, N. H., Parsons, M. P., Villanueva, E. B., Caron, N. S., Østergaard, M. E., Anderson, L. M., Xie, Y., et al. (2018) · Science Translational Medicine · doi:10.1126/scitranslmed.aar3959 · Cited by 126 · see citing works
Self-citationFriedrich, J., Kordasiewicz, H. B., O’Callaghan, B., Handler, H. P., Wagener, C., Duvick, L., Swayze, E. E., Rainwater, O., Hofstra, B., Benneyworth, M., et al. (2018) · JCI Insight · doi:10.1172/jci.insight.123193 · Cited by 121 · see citing works
Self-citationBrain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging
Mazur, C., Powers, B., Zasadny, K., Sullivan, J. M., Dimant, H., Kamme, F., Hesterman, J., Matson, J., Oestergaard, M., Seaman, M., et al. (2019) · JCI Insight · doi:10.1172/jci.insight.129240 · Cited by 114 · see citing works
Self-citationSpinocerebellar ataxia: relationship between phenotype and genotype – a review
Sun, Y.-M., Lu, C., & Wu, Z.-Y. (2016) · Clinical Genetics · doi:10.1111/cge.12808 · Cited by 113 · see citing works
Refined Characterization of the Expression and Stability of the SMN Gene Products
Vitte, J., Fassier, C., Tiziano, F. D., Dalard, C., Soave, S., Roblot, N., Brahe, C., Saugier-Véber, P., Bonnefont, J., & Melki, J. (2007) · American Journal Of Pathology · doi:10.2353/ajpath.2007.070399 · Cited by 112 · see citing works
Antisense oligonucleotide–directed inhibition of nonsense-mediated mRNA decay
Nomakuchi, T. T., Rigo, F., Aznarez, I., & Krainer, A. R. (2015) · Nature Biotechnology · doi:10.1038/nbt.3427 · Cited by 110 · see citing works
2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF
Shen, W., Liang, X., Sun, H., & Crooke, S. T. (2015) · Nucleic Acids Research · doi:10.1093/nar/gkv298 · Cited by 109 · see citing works
Targeting Several CAG Expansion Diseases by a Single Antisense Oligonucleotide
Evers, M. M., Pepers, B. A., van Deutekom, J. C. T., Mulders, S. A. M., den Dunnen, J. T., Aartsma-Rus, A. M., van Ommen, G. B., & van Roon‐Mom, W. (2011) · PLoS ONE · doi:10.1371/journal.pone.0024308 · Cited by 103 · see citing works
Advances in the treatment of hereditary transthyretin amyloidosis: a review
Gertz, M. A., Mauermann, M. L., Grogan, M., & Coelho, T. (2019) · Brain and Behavior · doi:10.1002/brb3.1371 · Cited by 102 · see citing works
Synthetic oligonucleotides recruit ILF2/3 to RNA transcripts to modulate splicing
Rigo, F., Hua, Y., Chun, S. J., Prakash, T. P., Krainer, A. R., & Bennett, C. F. (2012) · Nature Chemical Biology · doi:10.1038/nchembio.939 · Cited by 102 · see citing works
Self-citationA new look at cerebrospinal fluid movement
Orešković, D., & Klarica, M. (2014) · Fluids and Barriers of the CNS · doi:10.1186/2045-8118-11-16 · Cited by 101 · see citing works
Uehara, T., Choong, C., Nakamori, M., Hayakawa, H., Nishiyama, K., Kasahara, Y., Baba, K., Nagata, T., Yokota, T., Tsuda, H., et al. (2019) · Scientific Reports · doi:10.1038/s41598-019-43772-9 · Cited by 101 · see citing works
Bruijn, L. I., & Cleveland, D. W. (1996) · Neuropathology and Applied Neurobiology · doi:10.1111/j.1365-2990.1996.tb00907.x · Cited by 99 · see citing works
Self-citationDefining SOD1 ALS natural history to guide therapeutic clinical trial design
Bali, T., Self, W. K., Liu, J., Siddique, T., Wang, L. H., Bird, T. D., Ratti, E., Atassi, N., Boylan, K., Glass, J. D., et al. (2016) · Journal of Neurology Neurosurgery & Psychiatry · doi:10.1136/jnnp-2016-313521 · Cited by 92 · see citing works
Hooper, M. L., Chiasson, B. J., & Robertson, H. A. (1994) · Neuroscience · doi:10.1016/0306-4522(94)90559-2 · Cited by 90 · see citing works
Antisense suppression of glial fibrillary acidic protein as a treatment for Alexander disease
Hagemann, T. L., Powers, B. E., Mazur, C., Kim, A., Wheeler, S., Hung, G., Swayze, E. E., & Messing, A. (2017) · Annals of Neurology · doi:10.1002/ana.25118 · Cited by 89 · see citing works
Niu, C., Prakash, T. P., Kim, A., Quach, J., Huryn, L. A., Yang, Y., Lopez, E., Jazayeri, A., Hung, G., Sopher, B. L., et al. (2018) · Science Translational Medicine · doi:10.1126/scitranslmed.aap8677 · Cited by 89 · see citing works
Self-citationKay, C., Collins, J. A., Skotte, N. H., Southwell, A. L., Warby, S. C., Caron, N. S., Doty, C. N., Nguyen, B., Griguoli, A., Ross, C. J., et al. (2015) · Molecular Therapy · doi:10.1038/mt.2015.128 · Cited by 88 · see citing works
Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides
Ward, A. J., Norrbom, M., Chun, S. J., Bennett, C. F., & Rigo, F. (2014) · Nucleic Acids Research · doi:10.1093/nar/gku184 · Cited by 87 · see citing works
Self-citationNusinersen improves walking distance and reduces fatigue in later‐onset spinal muscular atrophy
Montes, J., Young, S. D., Mazzone, E. S., Pasternak, A. E., Glanzman, A. M., Finkel, R. S., Darras, B. T., Francesco Muntoni, A., Mercuri, E. M., Vivo, D. C. D., et al. (2019) · Muscle & Nerve · doi:10.1002/mus.26633 · Cited by 87 · see citing works
Self-citationGeary, R. S., Wancewicz, E. V., Matson, J. E., Pearce, M. E., Siwkowski, A. M., Swayze, E. E., & Bennett, F. (2009) · Biochemical Pharmacology · doi:10.1016/j.bcp.2009.04.013 · Cited by 85 · see citing works
Scoles, D. R., & Pulst, S. M. (2018) · Advances in experimental medicine and biology · doi:10.1007/978-3-319-71779-1_8 · Cited by 85 · see citing works
Rudnik‐Schöneborn, S., Berg, C., Zerres, K., Betzler, C., Grimm, T., Eggermann, T., Eggermann, K., Wirth, R., Wirth, B., & Heller, R. (2009) · Clinical Genetics · doi:10.1111/j.1399-0004.2009.01200.x · Cited by 81 · see citing works
Antisense oligodeoxynucleotide inhibits D2 dopamine receptor-mediated behavior and D2 messenger RNA
Weiss, B., Zhou, L.-W., Zhang, S. P., & Qin, Z. (1993) · Neuroscience · doi:10.1016/0306-4522(93)90426-g · Cited by 74 · see citing works
Sud, R., Geller, E. T., & Schellenberg, G. D. (2014) · Molecular Therapy — Nucleic Acids · doi:10.1038/mtna.2014.30 · Cited by 69 · see citing works
Stanek, L. M., Yang, W., Angus, S., Sardi, P., Hayden, M. R., Hung, G., Bennett, C. F., Cheng, S. H., & Shihabuddin, L. S. (2013) · Journal of Huntington s Disease · doi:10.3233/jhd-130057 · Cited by 66 · see citing works
Self-citationc-fos antisense generates apomorphine and amphetamine-induced rotation
Dragunow, M., Lowlor, P., Chiasson, B. J., & Robertson, H. (1993) · Neuroreport · doi:10.1097/00001756-199312000-00031 · Cited by 65 · see citing works
Bogdanik, L. P., Osborne, M. A., Davis, C., Martin, W., Austin, A., Rigo, F., Bennett, C. F., & Lutz, C. M. (2015) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1509758112 · Cited by 63 · see citing works
Self-citationErickson, M. A., Niehoff, M. L., Farr, S. A., Morley, J. E., Dillman, L. A., Lynch, K. M., & Banks, W. A. (2012) · Journal of Alzheimer s Disease · doi:10.3233/jad-2011-111517 · Cited by 57 · see citing works
Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy
Sahashi, K., Katsuno, M., Hung, G., Adachi, H., Kondo, N., Nakatsuji, H., Tohnai, G., Iida, M., Bennett, C. F., & Sobue, G. (2015) · Human Molecular Genetics · doi:10.1093/hmg/ddv300 · Cited by 57 · see citing works
Self-citation“Huntingtin Holiday”: Progress toward an Antisense Therapy for Huntington's Disease
Lu, X., & Yang, X. W. (2012) · Neuron · doi:10.1016/j.neuron.2012.06.001 · Cited by 56 · see citing works
Coulson, E. J., Barrett, G. L., Storey, E., Bartlett, P. F., Beyreuther, K. T., & Masters, C. L. (1997) · Brain Research · doi:10.1016/s0006-8993(97)00757-9 · Cited by 52 · see citing works
Sullivan, J. M., Mazur, C., Wolf, D. A., Horky, L. L., Currier, N. V., Fitzsimmons, B., Hesterman, J. Y., Pauplis, R., Haller, S., Powers, B. E., et al. (2020) · Journal of Translational Medicine · doi:10.1186/s12967-020-02461-2 · Cited by 47 · see citing works
Self-citationMohan, A., Fitzsimmons, B., Zhao, H. T., Jiang, Y., Mazur, C., Swayze, E. E., & Kordasiewicz, H. B. (2017) · Pain · doi:10.1097/j.pain.0000000000001074 · Cited by 46 · see citing works
Self-citationKostova, F. V., Williams, V. C., Heemskerk, J., Iannaccone, S. T., DiDonato, C. J., Swoboda, K. J., & Maria, B. L. (2007) · Journal of Child Neurology · doi:10.1177/0883073807305662 · Cited by 46 · see citing works
Moore, L. R., Keller, L., Bushart, D. D., Delatorre, R. G., Li, D., McLoughlin, H. S., Costa, M. D. C., Shakkottai, V. G., Smith, G. D., & Paulson, H. L. (2019) · Stem Cell Research · doi:10.1016/j.scr.2019.101504 · Cited by 42 · see citing works
TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animalsPreprint
Sahashi, K., Hua, Y., Ling, K., Hung, G., Rigo, F., Horev, G., Katsuno, M., Sobue, G., Ko, C., Bennett, C. F., et al. (2012) · Genes & Development · doi:10.1101/gad.197418.112 · Cited by 40 · see citing works
Self-citationTransependymal Cerebrospinal Fluid Flow: Opportunity for Drug Delivery?
Casaca-Carreira, J., Temel, Y., Hescham, S., & Jahanshahi, A. (2017) · Molecular Neurobiology · doi:10.1007/s12035-017-0501-y · Cited by 37 · see citing works
A new era in the management of Duchenne muscular dystrophy
Korinthenberg, R. (2018) · Developmental Medicine & Child Neurology · doi:10.1111/dmcn.14129 · Cited by 36 · see citing works
Ong, A. A. L., Tan, J., Bhadra, M., Dezanet, C., Patil, K. M., Chong, M. S., Kierzek, R., Décout, J., Roca, X., & Chen, G. (2019) · Molecules · doi:10.3390/molecules24163020 · Cited by 28 · see citing works
Role of spinal p38α and β MAPK in inflammatory hyperalgesia and spinal COX-2 expression
Fitzsimmons, B., Zattoni, M., Svensson, C. I., Steinauer, J., Hua, X., & Yaksh, T. L. (2010) · Neuroreport · doi:10.1097/wnr.0b013e32833774bf · Cited by 28 · see citing works
Tailored treatment for ALS poised to move ahead
Arnold, C. N. (2019) · Nature Medicine · doi:10.1038/d41591-019-00013-w · Cited by 24 · see citing works
Activating frataxin expression by single-stranded siRNAs targeting the GAA repeat expansion
Shen, X., Kilikevičius, A., O’Reilly, D., Prakash, T. P., Damha, M. J., Rigo, F., & Corey, D. R. (2018) · Bioorganic & Medicinal Chemistry Letters · doi:10.1016/j.bmcl.2018.07.033 · Cited by 21 · see citing works
Alpha-synuclein antisense oligonucleotides as a disease-modifying therapy for Parkinson’s diseasePreprint
Cole, T. A., Zhao, H. T., Collier, T. J., Sandoval, I. M., Sortwell, C. E., Steece‐Collier, K., Daley, B. F., Booms, A., Lipton, J. W., Welch, M., et al. (2019) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/830554 · Cited by 10 · see citing works
Self-citationDenman, R. B. (1997) · Biochemical and Biophysical Research Communications · doi:10.1006/bbrc.1996.5926 · Cited by 9 · see citing works
Flagged first, then by citations
Research works printed but not linked: 2
- [29]Liang X- H, Shen W, Sun H, Migawa MT, Vickers TA, Crooker ST. 2016. Specific increase in translation of proteins by antisense oligonucleotides targeting uORFs. Nat Biotechnol. In Press 27398791
- [140]Yu D, Pendergraff H, Liu J, Kordasiewicz H, Cleveland DW, 2012. Single-Stranded RNAs Act Through RNAi, Allele-Selectively Target Expanded CAG Repeats, and Potently Inhibit Huntingtin Expression. Cell In press PMC3444165 22939619
Cited by
Sorted by citations
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 236 · see citing works
Self-citationReexamining assumptions about miRNA-guided gene silencing
Kilikevicius, A., Meister, G., & Corey, D. R. (2022) · Nucleic Acids Research · doi:10.1093/nar/gkab1256 · Cited by 194 · see citing works
S. aureus drives itch and scratch-induced skin damage through a V8 protease-PAR1 axis
Deng, L., Costa, F. G., Blake, K. J., Choi, S., Chandrabalan, A., Yousuf, M. S., Shiers, S. I., Dubreuil, D., Vega‐Mendoza, D., Rolland, C., et al. (2023) · Cell · doi:10.1016/j.cell.2023.10.019 · Cited by 162 · see citing works
Antisense Oligonucleotide Therapy: From Design to the Huntington Disease Clinic
Rook, M. E., & Southwell, A. L. (2022) · BioDrugs · doi:10.1007/s40259-022-00519-9 · Cited by 114 · see citing works
Therapeutics in the Pipeline Targeting α-Synuclein for Parkinson's Disease
Jasutkar, H. G., Oh, S. E., & Mouradian, M. M. (2022) · Pharmacological Reviews · doi:10.1124/pharmrev.120.000133 · Cited by 102 · see citing works
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 96 · see citing works
Quadros, A. R. A. D. A., Li, Z., Wang, X., Ndayambaje, I. S., Aryal, S., Ramesh, N., Nolan, M., Jayakumar, R., Han, Y., Stillman, H., et al. (2024) · Acta Neuropathologica · doi:10.1007/s00401-023-02655-0 · Cited by 74 · see citing works
Self-citationHagedorn, P., Brown, J. M., Easton, A. E., Pierdomenico, M., Jones, K. M., Olson, R. E., Mercer, S. E., Li, D., Loy, J. K., Høg, A. M., et al. (2022) · Nucleic Acid Therapeutics · doi:10.1089/nat.2021.0071 · Cited by 64 · see citing works
RNA dysregulation in neurodegenerative diseases
Li, Y., & Sun, S. (2025) · The EMBO Journal · doi:10.1038/s44318-024-00352-6 · Cited by 62 · see citing works
ApoE4 reduction: An emerging and promising therapeutic strategy for Alzheimer's disease
Li, Y., Macyczko, J. R., Liu, C.-C., & Bu, G. (2022) · Neurobiology of Aging · doi:10.1016/j.neurobiolaging.2022.03.011 · Cited by 61 · see citing works
Malong, L., Napoli, I., Casal, G., White, I. J., Stierli, S., Vaughan, A., Cattin, A.-L., Burden, J. J., Hng, K. I., Bossio, A., et al. (2023) · Developmental Cell · doi:10.1016/j.devcel.2023.01.002 · Cited by 59 · see citing works
Hsu, X.-R., Wu, J., Wu, Y.-Y., Hsiao, S., Liang, J., Wu, Y.-J., Tung, C.-H., Huang, M., Lin, M.-S., Yang, P., et al. (2023) · Journal of Experimental & Clinical Cancer Research · doi:10.1186/s13046-023-02859-y · Cited by 58 · see citing works
Alternative processing of human HTT mRNA with implications for Huntington’s disease therapeutics
Fieńko, S., Landles, C., Sathasivam, K., McAteer, S. J., Milton, R. E., Osborne, G. F., Smith, E. J., Jones, S. T., Bondulich, M. K., Danby, E. C. E., et al. (2022) · Brain · doi:10.1093/brain/awac241 · Cited by 56 · see citing works
Self-citationShao, E., Chang, C.-W., Li, Z., Yu, X., Ho, K., Zhang, M., Wang, X., Simms, J., Lo, I., Speckart, J., et al. (2022) · Science Translational Medicine · doi:10.1126/scitranslmed.abm5527 · Cited by 54 · see citing works
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 · see citing works
Self-citationPoly(GR) and poly(GA) in cerebrospinal fluid as potential biomarkers for C9ORF72-ALS/FTD
Krishnan, G., Raitcheva, D., Bartlett, D. A., Prudencio, M., McKenna-Yasek, D. M., Douthwright, C. L., Oskarsson, B., Ladha, S. S., King, O. D., Barmada, S. J., et al. (2022) · Nature Communications · doi:10.1038/s41467-022-30387-4 · Cited by 50 · see citing works
Bunting, E. L., Donaldson, J., Cumming, S. A., Olive, J. F., Broom, E. L., Miclăuș, M., Hamilton, J., Tegtmeyer, M., Zhao, H. T., Brenton, J., et al. (2025) · Science Translational Medicine · doi:10.1126/scitranslmed.adn4600 · Cited by 48 · see citing works
Self-citationStem cell therapeutics and gene therapy for neurologic disorders
Chen, K. S., Koubek, E. J., Sakowski, S. A., & Feldman, E. L. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00427 · Cited by 44 · see citing works
Lordén, G., Wozniak, J. M., Doré, K., Dozier, L. E., Cates‐Gatto, C., Patrick, G. N., Gonzalez, D. J., Roberts, A. J., Tanzi, R. E., & Newton, A. C. (2022) · Nature Communications · doi:10.1038/s41467-022-34679-7 · Cited by 41 · see citing works
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 39 · see citing works
Luan, W., Wright, A. L., Brown-Wright, H., Le, S., Gil, R. S., Martin, L. M. S., Ling, K., Jafar‐Nejad, P., Rigo, F., & Walker, A. K. (2023) · Molecular Psychiatry · doi:10.1038/s41380-023-02036-9 · Cited by 37 · see citing works
Byrnes, A. E., Domínguez, S. L., Yen, C., Laufer, B. I., Foreman, O., Reichelt, M., Lin, H., Sagolla, M. S., Hötzel, K., Ngu, H., et al. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.05.005 · Cited by 35 · see citing works
LncRNAs as the Regulators of Brain Function and Therapeutic Targets for Alzheimer’s Disease
Liu, Y., Chen, X., Che, Y., Li, H., Zhang, Z., Peng, W., & Yang, J. (2022) · Aging and Disease · doi:10.14336/ad.2021.1119 · Cited by 35 · see citing works
Therapeutic approaches targeting aging and cellular senescence in Huntington's disease
Bhat, A. A., Moglad, E. H. O., Afzal, M., Thapa, R., Almalki, W. H., Kazmi, I. A., Alzarea, S. I., Ali, H., Pant, K., Singh, T. G., et al. (2024) · CNS Neuroscience & Therapeutics · doi:10.1111/cns.70053 · Cited by 32 · see citing works
Latest advances on new promising molecular-based therapeutic approaches for Huntington’s disease
Cheng, Y., Zhang, S., & Shang, H. (2024) · Journal of Translational Internal Medicine · doi:10.2478/jtim-2023-0142 · Cited by 30 · see citing works
Yeoh, Y. Q., Amin, A., Cuic, B., Tomas, D., Turner, B. J., & Shabanpoor, F. (2024) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2024.116737 · Cited by 29 · see citing works
Genome-wide CRISPR screen reveals v-ATPase as a drug target to lower levels of ALS protein ataxin-2
Kım, G., Nakayama, L., Blum, J. A., Akiyama, T., Boeynaems, S., Chakraborty, M., Couthouis, J., Tassoni-Tsuchida, E., Rodriguez, C. M., Bassik, M. C., et al. (2022) · Cell Reports · doi:10.1016/j.celrep.2022.111508 · Cited by 29 · see citing works
Lordén, G., & Newton, A. C. (2021) · Neuronal Signaling · doi:10.1042/ns20210036 · Cited by 27 · see citing works
(Dis)Solving the problem of aberrant protein states
Fare, C. M., & Shorter, J. (2021) · Disease Models & Mechanisms · doi:10.1242/dmm.048983 · Cited by 27 · see citing works
Wu, X., Xia, P., Yang, L., Lü, C., & Lu, Z. (2024) · Non-coding RNA Research · doi:10.1016/j.ncrna.2024.03.008 · Cited by 26 · see citing works
Villar-Pazos, S., Thomas, L., Yang, Y., Chen, K., Lyles, J. B., Deitch, B. J., Ochaba, J., Ling, K., Powers, B. E., Gingras, S., et al. (2023) · Nature Communications · doi:10.1038/s41467-023-42176-8 · Cited by 25 · see citing works
Self-citationSynucleinopathy in Amyotrophic Lateral Sclerosis: A Potential Avenue for Antisense Therapeutics?
Roberts, B., Theunissen, F., Mastaglia, F. L., Akkari, P. A., & Flynn, L. L. (2022) · International Journal of Molecular Sciences · doi:10.3390/ijms23169364 · Cited by 23 · see citing works
Suvarna, V., Deshmukh, K., & Murahari, M. (2022) · Frontiers in Pharmacology · doi:10.3389/fphar.2022.1034072 · Cited by 22 · see citing works
Zhou, L., Bi, J., Chang, S., Bai, Z., Yu, J., Wang, R., Li, Z., Zhang, X., Chou, J. J., & Pan, L. (2024) · Angewandte Chemie International Edition · doi:10.1002/anie.202415272 · Cited by 20 · see citing works
Increased chitotriosidase 1 concentration following nusinersen treatment in spinal muscular atrophy
Freigang, M., Steinacker, P., Wurster, C. D., Schreiber‐Katz, O., Osmanovic, A., Petri, S., Koch, J. C., Rostasy, K. M., Falkenburger, B. H., Ludolph, A. C., et al. (2021) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-021-01961-8 · Cited by 20 · see citing works
NLRP3 ‐directed antisense oligonucleotides reduce microglial immunoactivities in vitro
Braatz, C., Komes, M. P., Ravichandran, K. A., de Fragas, M. G., Griep, A., Schwartz, S., McManus, R. M., & Heneka, M. T. (2023) · Journal of Neurochemistry · doi:10.1111/jnc.15778 · Cited by 19 · see citing works
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 19 · see citing works
Self-citationRibeiro, J., Lopes, I., & Gomes, A. C. (2023) · Molecules · doi:10.3390/molecules28166015 · Cited by 19 · see citing works
Sarli, S. L., Fakih, H. H., Kelly, K., Devi, G., Rembetsy-Brown, J. M., McEachern, H. R., Ferguson, C. M., Echeverria, D., Lee, J., Sousa, J., et al. (2024) · Nucleic Acids Research · doi:10.1093/nar/gkae260 · Cited by 18 · see citing works
Bhati, V., Prasad, S., & Kabra, A. (2025) · Molecular and Cellular Neuroscience · doi:10.1016/j.mcn.2025.104010 · Cited by 18 · see citing works
Antisense oligonucleotides provide optimism to the therapeutic landscape for tauopathies
Harris, G. A., & Hirschfeld, L. R. (2024) · Neural Regeneration Research · doi:10.4103/nrr.nrr-d-23-02057 · Cited by 18 · see citing works
Advances in RNA therapeutics: Classes, innovations and clinical applications
Shahid, U. (2025) · The Journal of Precision Medicine Health and Disease · doi:10.1016/j.premed.2025.100016 · Cited by 17 · see citing works
Pan, Y., Guan, J. Z., Gao, Y., Zhu, Y., Li, H., Guo, H., He, Q., Guan, Z., & Yang, Z. (2023) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2023.04.028 · Cited by 16 · see citing works
Yao, J.-Y., Liu, T., Hu, X.-R., Sheng, H., Chen, Z.-H., Zhao, H., Li, X., Wang, Y., & Hao, L. (2024) · Biomedicine & Pharmacotherapy · doi:10.1016/j.biopha.2024.117557 · Cited by 14 · see citing works
Nusinersen Wearing-Off in Adult 5q-Spinal Muscular Atrophy Patients
Osmanovic, A., Schreiber‐Katz, O., & Petri, S. (2021) · Brain Sciences · doi:10.3390/brainsci11030367 · Cited by 14 · see citing works
A common molecular and cellular pathway in developing Alzheimer and cancer
Ali, M., Wani, S. U. D., Dey, T., Sridhar, S. B., & QADRIE, Z. L. (2023) · Biochemistry and Biophysics Reports · doi:10.1016/j.bbrep.2023.101625 · Cited by 13 · see citing works
Neuraxial drug delivery in pain management: An overview of past, present, and future
Yaksh, T. L., dos Santos, G. G., Lemes, J. B. P., & Malange, K. F. (2023) · Best practice & research. Clinical anaesthesiology · doi:10.1016/j.bpa.2023.04.003 · Cited by 12 · see citing works
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 · see citing works
Advances in gene therapy for neurogenetic diseases: a brief review
Xie, Y., Lv, W., Chen, Y., Hong, S., Yao, X., Chen, W., & Zhao, M. (2021) · Journal of Molecular Medicine · doi:10.1007/s00109-021-02167-y · Cited by 11 · see citing works
Derré, A., Soler, N. M., Billoux, V., Benizri, S., Vialet, B., Rivat, C., Barthélémy, P., Carroll, P., Pattyn, A., & Ventéo, S. (2023) · JCI Insight · doi:10.1172/jci.insight.161246 · Cited by 10 · see citing works
Kumari, S., Gupta, S., Sukhija, R., Gurjar, S., Dubey, S. K., & Taliyan, R. (2024) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2024.177123 · Cited by 9 · see citing works
Antisense oligonucleotides: A promising advancement in neurodegenerative disease treatment
Dudzisz, K., & Wandzik, I. (2025) · European Journal of Pharmacology · doi:10.1016/j.ejphar.2025.177644 · Cited by 9 · see citing works
Protein kinase inhibitors as therapeutics in neurodegenerative and psychiatric disorders
Tridente, G., Jana, A., Nath, A., & Ashraf, G. M. (2023) · Receptor Tyrosine Kinases in Neurodegenerative and Psychiatric Disorders · doi:10.1016/b978-0-443-18677-6.00015-4 · Cited by 8 · see citing works
Non-coding RNA biomarkers in Alzheimer’s disease
Mohammadpour, M., Saeidi, K., Ferdosi, F., Khanifar, H., Dadgostar, E., Zakizadeh, F., Abdolghaderi, S., & Khatami, S. H. (2025) · Clinica Chimica Acta · doi:10.1016/j.cca.2025.120427 · Cited by 8 · see citing works
Oligonucleotides: A novel area of interest for drug delivery in neurodegenerative diseases
Alharbi, K. S., Shaikh, M. A. J., Afzal, O., Altamimi, A. S. A., Almalki, W. H., Kazmi, I. A., Al-Abbasi, F. A., Alzarea, S. I., Babu, M. R., Singh, S. K., et al. (2022) · Journal of Drug Delivery Science and Technology · doi:10.1016/j.jddst.2022.103849 · Cited by 8 · see citing works
Thibonnier, M., & Ghosh, S. (2023) · International Journal of Molecular Sciences · doi:10.3390/ijms24087126 · Cited by 8 · see citing works
Promising RNA-based therapies for viral infections, genetic disorders, and cancer
Musa, D. A., Raji, M. O., Sikiru, A. B., Aremu, H. K., & Aigboeghian, E. A. (2024) · Academia molecular biology and genomics. · doi:10.20935/acadmolbiogen7329 · Cited by 7 · see citing works
Expanding horizons of tandem repeats in biology and medicine: Why ‘genomic dark matter’ matters
Hannan, A. J. (2023) · Emerging Topics in Life Sciences · doi:10.1042/etls20230075 · Cited by 7 · see citing works
Advances in RNA therapeutics for modulation of ‘undruggable’ targets
Martinsen, E., Jinnurine, T., Subramani, S., & Rogne, M. (2024) · Progress in molecular biology and translational science · doi:10.1016/bs.pmbts.2023.12.003 · Cited by 7 · see citing works
Peters, S., Wirkert, E., Kuespert, S., Heydn, R., Johannesen, S. W., Friedrich, A., Mailänder, S., Korte, S., Mecklenburg, L., Aigner, L., et al. (2022) · Pharmaceutics · doi:10.3390/pharmaceutics14010200 · Cited by 6 · see citing works
Delivery and controllable release of anti-sense DNA based on frame-guided assembly strategy
Zhang, Y., Hou, X., Piao, J., Yuan, W., Zhou, B., Zhao, X., Hao, Z., Zhuang, Y., Xu, L., Dong, Y., et al. (2022) · European Polymer Journal · doi:10.1016/j.eurpolymj.2022.111187 · Cited by 6 · see citing works
Short structural variants as informative genetic markers for ALS disease risk and progression
Theunissen, F., Flynn, L. L., Anderton, R. S., & Akkari, P. A. (2022) · BMC Medicine · doi:10.1186/s12916-021-02206-y · Cited by 6 · see citing works
RNA therapeutics 2.0: Expanding the landscape from mRNA vaccines to splicing modulators and beyond
Karwa, P. N., & Sakle, N. S. (2026) · Biotechnology Advances · doi:10.1016/j.biotechadv.2026.108862 · Cited by 5 · see citing works
Experimental Model Systems Used in the Preclinical Development of Nucleic Acid Therapeutics
Zhou, H., Arechavala‐Gomeza, V., & Garanto, A. (2023) · Nucleic Acid Therapeutics · doi:10.1089/nat.2023.0001 · Cited by 5 · see citing works
Re-Activation of Neurogenic Niches in Aging BrainPreprint
Maimon, R., Chillón-Marinas, C., Vazquez‐Sanchez, S., Kern, C., Jenie, I., Malukhina, K., Moore, S. R., Cui, J., Goginashvili, A., Moghadami, S., et al. (2024) · bioRxiv (Cold Spring Harbor Laboratory) · doi:10.1101/2024.01.27.575940 · Cited by 5 · see citing works
Self-citationFrei, 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 · see citing works
Zhao, X., Zheng, Y., Cai, X. T., Yao, Y., & Qin, D. (2026) · Pharmaceuticals · doi:10.3390/ph19010092 · Cited by 4 · see citing works
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 · see citing works
Buijsen, R. A. M., van der Graaf, L. M., Kuijper, E. C., Pepers, B. A., Daoutsali, E., Weel, L., Raz, V., Parfitt, D. A., & van Roon‐Mom, W. (2024) · Biomedicines · doi:10.3390/biomedicines12091933 · Cited by 3 · see citing works
Fard, S. A. Z., Abuei, H., Farhadi, A., Dehbidi, G. R., Zare, F., Nejabat, M., & Behzad‐Behbahani, A. (2023) · Biomedical and Biotechnology Research Journal (BBRJ) · doi:10.4103/bbrj.bbrj_163_23 · Cited by 3 · see citing works
Gu, L., Liu, J., Wang, C., Shan, X., Li, S., Zhang, X., Xia, L., & Li, J. (2026) · International Journal of Pharmaceutics X · doi:10.1016/j.ijpx.2026.100503 · Cited by 3 · see citing works
Fard, S. A. Z., Abuei, H., Behzad‐Behbahani, A., Dehbidi, G. R., Zare, F., Nejabat, M., Safarpour, A. R., & Farhadi, A. (2024) · Advanced Pharmaceutical Bulletin · doi:10.34172/apb.2024.045 · Cited by 3 · see citing works
Jepp, T., Christian, S., & Dindot, S. V. (2025) · Nucleic Acid Therapeutics · doi:10.1177/21593337251391564 · Cited by 2 · see citing works
Li, C., Zhao, X., Jiang, K., Kang, H., Liu, S., Jiang, L., Bai, B., Cui, H., Zhang, Z., Dong, M., et al. (2026) · Advanced Materials · doi:10.1002/adma.202521427 · Cited by 2 · see citing works
Fard, S. A. Z., Abuei, H., Farhadi, A., Dehbidi, G. R., Zare, F., Abbasfard, Z., & Behzad‐Behbahani, A. (2023) · Future Virology · doi:10.2217/fvl-2022-0207 · Cited by 2 · see citing works
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 · see citing works
An antisense oligonucleotide targeting STK25 as a long-acting therapeutic candidate for early MASLD
Dong, A., Li, N., Lang, X., Chen, S., Qin, Y., Wang, Y., Wang, H., Guo, M., Yan, A., Song, K., et al. (2026) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2026.103038 · Cited by 1 · see citing works
Gene Silencing Therapies for Huntington’s Disease
Hao, C., & Ji, H. (2025) · Neurology Letters · doi:10.61882/nl.4.1.69 · Cited by 1 · see citing works
Transient Cyclic Structured Oligonucleotide Designs for Therapeutic Applications
Agrawal, S. (2026) · Current Protocols · doi:10.1002/cpz1.70319 · Cited by 1 · see citing works
Kabel, A. M., & AlBarraq, A. A. (2026) · Pediatric Research · doi:10.1038/s41390-026-04954-0 · Cited by 1 · see citing works
Basic Science in Movement Disorders: Fueling the Engine of Translation into Clinical Practice
Outeiro, T. F., Kalia, L. V., Bézard, E., Ferrario, J. E., Lin, C., Salama, M., Standaert, D. G., Taiwo, L., Takahashi, R., Vila, M., et al. (2024) · Movement Disorders · doi:10.1002/mds.29802 · Cited by 1 · see citing works
Kim, L. J. Y., Kundu, B., Moretti, P. M., Lozano, A. M., & Rahimpour, S. (2024) · Neurotherapeutics · doi:10.1016/j.neurot.2024.e00452 · Cited by 1 · see citing works
Zhou, L., Bi, J., Chang, S., Bai, Z., Yu, J., Wang, R., Li, Z., Zhang, X., Chou, J. J., & Pan, L. (2024) · Angewandte Chemie · doi:10.1002/ange.202415272 · Cited by 1 · see citing works
Abbott, M., Bender, K. J., Brunklaus, A., Demarest, S., Egan, S. M., Haviland, I., Kearney, J. A., Myers, L. S., Olson, H. E., Sanders, S., et al. (2024) · Cambridge University Press eBooks · doi:10.1017/9781009530361 · Cited by 1 · see citing works
Antisense oligonucleotides in rare neurogenetic disorders
Shin, H. J., Ko, A., Oh, J., & Kang, H. (2024) · Journal of genetic medicine · doi:10.5734/jgm.2024.21.2.41 · Cited by 1 · see citing works
Small Interfering RNAs in Neurodegenerative Diseases: A Therapeutic Horizon
El-Nablaway, M. (2025) · Advanced Health Science and Technology Journal · doi:10.4103/ahstj.ahstj_8_25 · Cited by 1 · see citing works
Dudhat, K. R. (2022) · Bioequivalence & Bioavailability International Journal · doi:10.23880/beba-16000177
Novel disease-modifying treatments for synucleinopathies
Singer, W., Palma, J., Kaufmann, H., & Low, P. A. (2023) · Elsevier eBooks · doi:10.1016/b978-0-323-85492-4.00110-1
Preclinical Evaluation of Neuraxial Drugs for Safety
Yaksh, T. L., Boyd, R. B., & Keifer, O. P. (2023) · Springer eBooks · doi:10.1007/978-3-031-39558-1_12
Bogen, O., Araldi, D., Sucher, A., Kober, K. M., Ohara, P. T., & Levine, J. D. (2024) · Molecular Pain · doi:10.1177/17448069241230419
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
Self-citationA perspective: neuraxial therapeutics in pain management: now and future
DeAndres, J., Dickenson, A. H., Hayek, S. M., Linninger, A. A., & Yaksh, T. L. (2024) · Frontiers in Pain Research · doi:10.3389/fpain.2024.1505019
Nucleic Acid-Based Systems for Regenerative Medicine
Martín, L., & Grzela, D. P. (2025) · Springer eBooks · doi:10.1007/978-3-031-87744-5_8
Freigang, M., Steinacker, P., Wurster, C. D., Schreiber‐Katz, O., Osmanovic, A., Petri, S., Koch, J. C., Rostasy, K. M., Falkenburger, B. H., Ludolph, A. C., et al. (2021) · Figshare · doi:10.6084/m9.figshare.15071519
Freigang, M., Steinacker, P., Wurster, C. D., Schreiber‐Katz, O., Osmanovic, A., Petri, S., Koch, J. C., Rostasy, K. M., Falkenburger, B. H., Ludolph, A. C., et al. (2021) · Figshare · doi:10.6084/m9.figshare.15071513
Freigang, M., Steinacker, P., Wurster, C. D., Schreiber‐Katz, O., Osmanovic, A., Petri, S., Koch, J. C., Rostasy, K. M., Falkenburger, B. H., Ludolph, A. C., et al. (2021) · Figshare · doi:10.6084/m9.figshare.15071516
Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA
Kaymaz, A. Y., Bora, G., Erdem‐Yurter, H., & Grunseich, C. (2026) · Genes · doi:10.3390/genes17040419
- Authors
- Bennett, C. F., Kordasiewicz, H. B., & Cleveland, D. W.
- Affiliations
- Ionis Pharmaceuticals (United States) (US)
- Ludwig Cancer Research (BE)
- Venue
- Annual Review of Pharmacology and Toxicology
- Publisher
- Annual Reviews
- Published
- 2021-01-06
- Volume
- 61
- Issue
- 1
- Pages
- 831-852
- ISSN
- 0362-1642 (ISSN-L)
- 1545-4304
- PMC
- PMC8682074
- OpenAlex
- W3091999396
- Cited by
- 108
- References
- 161
- Citations by year
- 2026: 15 · 2025: 11 · 2024: 29 · 2023: 24 · 2022: 19 · 2021: 10
- FWCI
- 1.26
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- The Annual Review of Pharmacology and Toxicology (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institute of Neurological Disorders and Stroke (R01NS112503, R01NS027036)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Keywords
- antisense oligonucleotides, neurodegenerative diseases, RNA degradation, intrathecal drug delivery, nusinersen, amyotrophic lateral sclerosis, Huntington's disease, Alzheimer's disease, Parkinson's disease, Angelman syndrome, disease-modifying therapy
- MeSH terms
- Pharmaceutical Preparations, Humans, Parkinson Disease, RNA, Oligonucleotides, Antisense, Neurodegenerative Diseases
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
Are you an author, or do you see a problem with this page? Request a review
