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Brain Res. 2022 Jan 15;1775:147743. doi: 10.1016/j.brainres.2021.147743. Epub 2021 Nov 26.

Targeted Augmentation of Nuclear Gene Output (TANGO) of Scn1a rescues parvalbumin interneuron excitability and reduces seizures in a mouse model of Dravet Syndrome.

Brain research

Eric R Wengert, Pravin K Wagley, Samantha M Strohm, Nuha Reza, Ian C Wenker, Ronald P Gaykema, Anne Christiansen, Gene Liau, Manoj K Patel

Affiliations

  1. Department of Anesthesiology, University of Virginia Health System, Charlottesville, VA, United States; Neuroscience Graduate Program, University of Virginia School of Medicine, Charlottesville, VA, United States.
  2. Department of Anesthesiology, University of Virginia Health System, Charlottesville, VA, United States.
  3. Stoke Therapeutics, Bedford, MA, United States.
  4. Department of Anesthesiology, University of Virginia Health System, Charlottesville, VA, United States; Neuroscience Graduate Program, University of Virginia School of Medicine, Charlottesville, VA, United States. Electronic address: [email protected].

PMID: 34843701 DOI: 10.1016/j.brainres.2021.147743

Abstract

Dravet Syndrome (DS) is a severe developmental and epileptic encephalopathy typically caused by loss-of-function de novo mutations in the SCN1A gene which encodes the voltage-gated sodium channel isoform Na

Copyright © 2021. Published by Elsevier B.V.

Keywords: Antisense oligonucleotide (ASO); Epilepsy; Parvalbumin-positive (PV) inhibitory interneuron; STK-001; Voltage-gated sodium channel

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