The DOI matches its Crossref record; 29 of the 30 references it cites were found in a registry or an index.
Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome
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Antisense oligonucleotide administration reduces seizures and SUDEP incidence in a mouse model of Dravet syndrome.
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“The use of antisense oligonucleotides to increase the production of translated mRNA, termed Targeted Augmentation of Nuclear Gene Output (TANGO), has been used successfully in an animal model of Dravet syndrome to increase the production of sodium channels 239”
From Developmental and epileptic encephalopathies (2024) · cites this paper as [239]
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Cites
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
The core Dravet syndrome phenotype
Dravet, C. (2011) · Epilepsia · doi:10.1111/j.1528-1167.2011.02994.x · Cited by 630
Cooper, M. S., McIntosh, A. M., Crompton, D. E., McMahon, J. M., Schneider, A., Farrell, K., Ganesan, V., Gill, D. S., Kivity, S., Lerman‐Sagie, T., et al. (2016) · Epilepsy Research · doi:10.1016/j.eplepsyres.2016.10.006 · Cited by 323
Wirrell, E. C., Laux, L., Donner, E. J., Jetté, N., Knupp, K. G., Meskis, M. A., Miller, I., Sullivan, J. E., Welborn, M., & Berg, A. T. (2017) · Pediatric Neurology · doi:10.1016/j.pediatrneurol.2017.01.025 · Cited by 287
The genetics of Dravet syndrome
Marini, C., Scheffer, I. E., Nabbout, R., Suls, A., Jonghe, P. D., Zara, F., & Guerrini, R. (2011) · Epilepsia · doi:10.1111/j.1528-1167.2011.02997.x · Cited by 276
Incidence of Dravet Syndrome in a US Population
Wu, Y. W., Sullivan, J., McDaniel, S. S., Meisler, M. H., Walsh, E. M., Li, S. X., & Kuzniewicz, M. W. (2015) · Pediatrics · doi:10.1542/peds.2015-1807 · Cited by 248
Colasante, G., Lignani, G., Brusco, S., Berardino, C. D., Carpenter, J. C., Giannelli, S. G., Valassina, N., Bido, S., Ricci, R., Castoldi, V., et al. (2019) · Molecular Therapy · doi:10.1016/j.ymthe.2019.08.018 · Cited by 246
Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
Corey, D. R. (2017) · Nature Neuroscience · doi:10.1038/nn.4508 · Cited by 242
Cannabinoids in treatment-resistant epilepsy: A review
O’Connell, B. K., Gloss, D. S., & Devinsky, O. (2017) · Epilepsy & Behavior · doi:10.1016/j.yebeh.2016.11.012 · Cited by 228
Lim, K., Zhou, H., Jeon, H. Y., Kach, J., Jing, E., Weyn‐Vanhentenryck, S. M., Downs, M., Corrionero, A., Oh, R. S., Scharner, J., et al. (2020) · Nature Communications · doi:10.1038/s41467-020-17093-9 · Cited by 216
Self-citation
Research works printed but not linked: 1
- C. Dravet, M. Bureau, H. Oguni, Y. Fukuyama, O. Cokar, Severe myoclonic epilepsy in infancy: Dravet syndrome. Adv. Neurol. 95, 71–102 (2005).
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Sorted by citations
Lauffer, M. C., van Roon‐Mom, W., Aartsma-Rus, A. M., & Collaborative, N. =. 1. (2024) · Communications Medicine · doi:10.1038/s43856-023-00419-1 · Cited by 295
International consensus on diagnosis and management of Dravet syndrome
Wirrell, E. C., Hood, V., Knupp, K. G., Meskis, M. A., Nabbout, R., Scheffer, I. E., Wilmshurst, J., & Sullivan, J. (2022) · Epilepsia · doi:10.1111/epi.17274 · Cited by 249
Sodium channelopathies in neurodevelopmental disorders
Meisler, M. H., Hill, S. F., & Yu, W. (2021) · Nature reviews. Neuroscience · doi:10.1038/s41583-020-00418-4 · Cited by 210
Developmental and epileptic encephalopathies: from genetic heterogeneity to phenotypic continuum
Guerrini, R., Conti, V., Mantegazza, M., Balestrini, S., Galanopoulou, A. S., & Benfenati, F. (2023) · Physiological Reviews · doi:10.1152/physrev.00063.2021 · Cited by 196
The gain of function SCN1A disorder spectrum: novel epilepsy phenotypes and therapeutic implications
Brunklaus, A., Brünger, T., Feng, T., Fons, C., Lehikoinen, A., Panagiotakaki, E., Vintan, M.-A., Symonds, J., Andrew, J., Arzimanoglou, A., et al. (2022) · Brain · doi:10.1093/brain/awac210 · Cited by 167
Metadata
- Authors
- Han, Z., Chen, C., Christiansen, A., Ji, S., Lin, Q., Anumonwo, C., Liu, C., Leiser, S. C., Meena, Aznarez, I., Liau, G., & Isom, L. L.
- Affiliations
- Stoke Therapeutics Inc., Bedford, MA 01730, USA.
- University of Michigan (US)
- Stoke Therapeutics, Inc. (United States) (US)
- Department of Pharmacology, University of Michigan, Ann Arbor, MI 48109, USA.
- PsychoGenics Inc., Paramus, NJ 07652, USA.
- Corresponding author
- Isom, Lori L.
- Venue
- Science Translational Medicine
- Published
- 2020-08-26
- Volume
- 12
- Issue
- 558
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W3081439212
- Cited by
- 341
- References
- 30
- Citations by year
- 2026: 47 · 2025: 59 · 2024: 52 · 2023: 57 · 2022: 66 · 2021: 52 · 2020: 8
- FWCI
- 33.37
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- Deep Blue (University of Michigan) (submittedVersion)
- Funders
- Stoke Therapeutics (N/A)
- Topics
- Health Sciences › Medicine › Clinical Neurology › Epilepsy research and treatment
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmacological Effects and Toxicity Studies
- Keywords
- antisense oligonucleotides, Dravet syndrome, SCN1A, sodium channel alpha subunit, seizures, sudden unexpected death in epilepsy, tango, gene-specific therapy, mouse model
- MeSH terms
- Sudden Unexpected Death in Epilepsy, Animals, Epilepsies, Myoclonic, Mice, Inbred C57BL, Seizures, Incidence, Oligonucleotides, Antisense, Mice, NAV1.1 Voltage-Gated Sodium Channel
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