Evidence map›Paper›PMID 31830809›Full record

ArticleHuman gene therapy2020

Sexually Divergent Mortality and Partial Phenotypic Rescue After Gene Therapy in a Mouse Model of Dravet Syndrome.

Yosuke Niibori, Shiron J Lee, Berge A Minassian, David R Hampson

Open access · hybridAbstract read
In one paragraph

Article in Human gene therapy, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers.

0numbers the graph read from it
0cells of the map it votes in
46citing papers in PubMed
3.6field-weighted citation impact, top 6% of its field
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

46 citing papers in PubMed, 75 citations in OpenAlex.

  1. Article
  2. Article
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  4. [Adeno-associated virus-mediated gene therapy for genetic epilepsy: prospects and challenges].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2026
    Review
  5. Article
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  7. Article
  8. Review
  9. Article
  10. Review
  11. Dravet syndrome: novel insights intoFrontiers in neuroscience · 2025
    Review
  12. Article
  13. Review
  14. Fine mapping and candidate gene analysis of Dravet syndrome modifier loci on mouse chromosomes 7 and 8.Mammalian genome : official journal of the International Mammalian Genome Society · 2024
    Article
  15. Review
  16. Article
  17. Article
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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

4 authors at 2 institutions in 2 countries.

Yosuke NiiboriDepartment of Pharmaceutical Sciences, Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Canada.
Shiron J LeeDepartment of Pharmaceutical Sciences, Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Canada.
Berge A MinassianDepartment of Pediatrics, University of Texas Southwest Medical Center, Dallas, Texas.
David R HampsonDepartment of Pharmaceutical Sciences, Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, Canada.
University of Toronto · CAThe University of Texas Southwestern Medical Center · US

Funding

Suppressing glycogen storage with small molecule inhibitors as a therapeutic approach to Lafora DiseaseP01NS097197 · NINDS · UNIVERSITY OF KENTUCKY · PI GAO, TIANYAN · 2016 to 2021
$9.0M
CIHRNINDS NIH HHS P01 NS097197
6 · The paper itself

Abstract

Dravet syndrome (DS) is a neurodevelopmental genetic disorder caused by mutations in the

Indexed as

Genetic TherapyAnimalsAutistic DisorderDependovirusDisease Models, AnimalEpilepsies, MyoclonicEpilepsyFemaleGenetic VectorsHumansMaleMiceMice, Inbred C57BLMutationNAV1.1 Voltage-Gated Sodium ChannelPhenotypeNAV1.1 Voltage-Gated Sodium ChannelScn1a protein, mouseScn1b protein, mouseVoltage-Gated Sodium Channel beta-1 Subunitadeno-associated virusautismcerebral spinal fluidepilepsyneurodevelopmental disorderSCN1ASCN1B

Identifiers

PMID31830809
PMCPMC7087406
OpenAlexW2995066388

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.