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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., Meena, Aznarez, Isabel, Liau, Gene, Isom, Lori L.·2020·Science Translational Medicine

DOI 10.1126/scitranslmed.aaz6100

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

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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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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.
ORCID: Han, Zhou
Chen, Chunling
Lin, Qian
Liu, Chante
Leiser, Steven C.
Aznarez, Isabel
Isom, Lori L.
Venue: Science Translational Medicine
Publisher: American Association for the Advancement of Science (AAAS)
Published: 2020-08-26
Volume: 12
Issue: 558
ISSN: 1946-6234 (ISSN-L)
1946-6242
DOI: 10.1126/scitranslmed.aaz6100
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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Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome

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