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.
“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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- 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 › Psychiatry and Mental health › 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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