Cited by 341Epilepsy research and treatmentGenetics and Neurodevelopmental DisordersPharmacological Effects and Toxicity Studies

Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome

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Han, Zhou, Chen, Chunling, Christiansen, Anne, Ji, Sophina, Lin, Qian, Anumonwo, Charles, Liu, Chante, Leiser, Steven C. +4 more·2020·Science Translational Medicine

Abstract

Antisense oligonucleotide administration reduces seizures and SUDEP incidence in a mouse model of Dravet syndrome.

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Crossref · 10.1126/scitranslmed.aaz6100

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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
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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