Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

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

Human gene therapy - 1 Mar 2020

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

Abstract excerpt

Dravet syndrome (DS) is a neurodevelopmental genetic disorder caused by mutations in the SCN1A gene encoding the α subunit of the NaV1.1 voltage-gated sodium channel that controls neuronal action potential firing. The high density of this mutated channel in GABAergic interneurons results in impaired inhibitory neurotransmission and subsequent excessive activation of excitatory neurons. The syndrome is associated...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to Autistic Disorder
Share to Dependovirus
Share to Epilepsies, Myoclonic

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Epilepsy

Share to Genetic Therapy

Share to NAV1.1 Voltage-Gated Sodium Channel

Share to Seizures

Share to Transgenes

Source

Open in PubMed Open full text

Identifiers

PMID: 31830809
DOI: 10.1089/hum.2019.225
PMCID: PMC7087406

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Sexually Divergent Mortality and Partial Phenotypic Rescue After Gene Therapy in a Mouse Model of Dravet Syndrome.PMID 31830809Read ↗

Neonatal but not juvenile gene therapy reduces seizures and prolongs lifespan in SCN1B–Dravet syndrome miceSemantic proximity Read ↗

Transfer of SCN1A to the brain of adolescent mouse model of Dravet syndrome improves epileptic, motor, and behavioral manifestationsSemantic proximity Read ↗

Interneuron-specific dual-AAV SCN1A gene replacement corrects epileptic phenotypes in mouse models of Dravet syndrome.Semantic proximity Read ↗

AAV-mediated interneuron-specific gene replacement for Dravet syndromeSemantic proximity Read ↗

Cell-Selective Adeno-Associated Virus-Mediated SCN1A Gene Regulation Therapy Rescues Mortality and Seizure Phenotypes in a Dravet Syndrome Mouse Model and Is Well Tolerated in Nonhuman Primates.Semantic proximity Read ↗

Exogenous Na <sub>V</sub> 1.1 activity in excitatory and inhibitory neurons reverts Dravet syndrome comorbidities when delivered post-symptom onset in mice with DravetSemantic proximity Read ↗

Viral vector-mediated expression of NaV1.1, after seizure onset, reduces epilepsy in mice with Dravet syndrome.Semantic proximity Read ↗

Specific deletion of NaV1.1 sodium channels in inhibitory interneurons causes seizures and premature death in a mouse model of Dravet syndrome.Semantic proximity Read ↗

SCN1A as a therapeutic target for Dravet syndrome.Semantic proximity Read ↗

AAV delivery of RNA editing machinery rescues SUDEP and seizure phenotype in a mouse model of Dravet SyndromeSemantic proximity Read ↗

Mapping genetic modifiers of survival in a mouse model of Dravet syndromeSemantic proximity Read ↗

A novel rat model of Dravet syndrome recapitulates clinical hallmarksSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.