Evidence map›Paper›PMID 42521212›Full record

ArticleAnnals of clinical and translational neurology2026

Augmenting and Assaying Nav1.1 Protein Quantity for Dravet Syndrome Therapy.

Aiswarya Saravanan, Jordan Safran, Anna Loughman, Grace Lin, Shivanshi Vaid, Dana Allababidi, Peter M Tessier, Louis T Dang

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Annals of clinical and translational neurology, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.

0numbers the graph read from it
0cells of the map it votes in
0citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

0 citing papers in PubMed.

No citing paper in PubMed yet.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Aiswarya SaravananDepartment of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.ORCID https://orcid.org/0009-0000-9619-4345
Jordan SafranDepartment of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
Anna LoughmanDepartment of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
Grace LinDepartment of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
Shivanshi VaidDepartment of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.
Dana AllababidiDepartment of Chemical Engineering, University of Michigan, Ann Arbor, Michigan, USA.
Peter M TessierDepartment of Chemical Engineering, University of Michigan, Ann Arbor, Michigan, USA.
Louis T DangDepartment of Pediatrics, University of Michigan, Ann Arbor, Michigan, USA.ORCID https://orcid.org/0000-0003-4853-4952

Funding

CD98hc Brain Shuttles for Delivering Off-the-shelf Neuroprotective Antibodies in Alzheimer's DiseaseR01AG080016 · NIA · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI Colin Fred Greineder, Peter M Tessier · 2023 to 2026
$3.2M
Neuron-specific modulation of gene expression using systemically administered bispecific antibody-ASO conjugatesR01NS138455 · NINDS · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI Peter M Tessier, Greg Thurber · 2024 to 2026
$1.8M
Child Neurology Society/FoundationNIA NIH HHS R01 AG080016NIA NIH HHS R01AG080016NINDS NIH HHS R01 NS138455NINDS NIH HHS R01NS138455University of Michigan Department of Pediatrics
6 · The paper itself

Abstract

Dravet Syndrome (DS) is a developmental and epileptic encephalopathy predominantly caused by heterozygous loss-of-function variants in SCN1A, which encodes Nav1.1. Conserved upstream open reading frames (uORFs) in SCN1A were validated to regulate translation in reporter assays, demonstrating the therapeutic viability of increasing Nav1.1 from a wild-type allele. A human induced pluripotent stem cell (iPSC) line with endogenously HiBiT-tagged Nav1.1 showed dose-dependent Nav1.1 downregulation with gapmer antisense oligonucleotides (ASOs) and upregulation with splice-switching ASOs, validating a sensitive quantification platform for endogenous Nav1.1 translation. This iPSC line provides a timely resource for screening translational modulators and validating therapeutic strategies for SCN1A-related disorders.

Indexed as

epilepsyHiBiThigh throughput screen

Identifiers

PMID42521212
PMCPMC13413088

What OpenQuestion holds

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LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.