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Effective knockdown-replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy

Molecular Therapy(2024)10.1016/j.ymthe.2024.08.009Source: DataRank Database

Effective knockdown-replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy is a research paper published in Molecular Therapy (2024). On theSindex it has a DataRank of 0. It has been cited 16 times.

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0DataRank · unranked

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Open Access16 citations

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datarank_citation_only_1hop_v6· scope data_onlyMethodology

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Enrichment:Funding (5 grants)Impact (FWCI 2.82)OA: hybridTopics (58)IDs (PubMed, PMC, OpenAlex)Annotations (1)

FAIR Checklist

Context only (not used in score)

Findable (1/2)

Has DOI

Accessible (1/2)

Open Access

Interoperable (0/2)

Reusable (0/3)

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Authors (12)

Divya Soundararajan,Noah K. Taylor ORCID,Osasumwen V. Aimiuwu ORCID,Pranav Mathkar ORCID,Amy N. Shore

Live enrichment

PubMed:39127888 PMC:PMC11489538 OpenAlex:https://openalex.org/W4401487756

Nationwide Children's Hospital

Grant: 63/639,576

National Institutes of Health

Grant: R01 NS031348

National Institutes of Health

Grant: F31 NS111808

National Institutes of Health

Grant: 1F31NS111808-01A1

Modeling gene-specific therapy of intractable childhood epileptic encephalopathy

National Institutes of Health

Grant: 5R01NS031348-04

GENETIC DETERMINANTS OF EPILEPSY

FWCI

2.82

Citation Percentile

0.9%

Citation Trend

2025

2026

hybridLicense: cc-by-nc-nd

journalhttp://www.cell.com/article/S1525001624005318/pdf

publisherhttp://www.cell.com/article/S1525001624005318/pdf

publisherhttps://api.elsevier.com/content/article/PII:S1525001624005318?httpAccept=text/xml

publisherhttps://api.elsevier.com/content/article/PII:S1525001624005318?httpAccept=text/plain

journalhttps://doi.org/10.1016/j.ymthe.2024.08.009

Fields of Study

CRISPR and Genetic EngineeringRNA and protein synthesis mechanismsGenetics and Neurodevelopmental DisordersAnimalsHumansMiceDependovirusDisease Models, AnimalDynamin IEpilepsyGABAergic NeuronsGene Knockdown TechniquesGenetic TherapyGenetic VectorsMutationRNA InterferenceRNA, Small Interfering

MeSH Terms

DependovirusAnimalsDisease Models, AnimalEpilepsyGenetic VectorsHumansMutationGenetic TherapyDynamin IRNA InterferenceRNA, Small InterferingMiceGene Knockdown TechniquesGABAergic Neurons

Keywords

Gene knockdownBiologyRNA interferenceMutantGeneTranscriptomeGeneticsRNAGene expressionEpilepsyNeurodevelopmentDynamin-1Developmental And Epileptic EncephalopathyDeno-associated VirusGenetic VectorsGenetic TherapyDependovirusMiceDisease Models, AnimalGene Knockdown TechniquesMutationAnimalsHumansOriginal ArticleGABAergic NeuronsRNA, Small InterferingDynamin I

Effective knockdown-replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy

FAIR Checklist

Authors (12)

Funding (5 grants)

Impact

Open Access

Topics

Annotations (Europe PMC)