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An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model

EMBO Molecular Medicine(2023)10.15252/emmm.202215968Source: DataRank Database

An innovative hematopoietic stem cell gene therapy approach benefits CLN1 disease in the mouse model is a research paper published in EMBO Molecular Medicine (2023). On theSindex it has a DataRank of 0. It has been cited 9 times.

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0DataRank · unranked

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Open Access9 citations

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datarank_citation_only_1hop_v6· scope data_onlyMethodology

Pipeline:MetadataData-paper checkEnrichmentCitation networkScoring

Enrichment:Funding (4 grants)OA: goldTopics (38)IDs (PubMed, PMC)

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Findable (1/2)

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Accessible (1/2)

Open Access

Interoperable (0/2)

Reusable (0/3)

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Authors (12)

Sabyasachi Das ORCID,Janki Patel ORCID,Odella C. Jno-Charles,Rajesh Kumar ORCID,Ana Zguro

Live enrichment

PubMed:36876653 PMC:PMC10086581

National Institutes of Health

Grant: R01 HD095935‐04

European Research Council

Grant: 617162

HSC-based therapies for LSDs: understanding the modalities of cell replacement in the LSD brain for improving therapeutic efficacy

NICHD NIH HHS

Grant: R01 HD095935

National Institutes of Health

Grant: 5R01HD095935-04

Hematopoietic stem cell gene therapy for infantile neuronal ceroid lipofuscinosis

goldLicense: cc-by

publisherhttps://onlinelibrary.wiley.com/doi/pdfdirect/10.15252/emmm.202215968

publisherhttps://link.springer.com/content/pdf/10.15252/emmm.202215968.pdf

publisherhttps://onlinelibrary.wiley.com/doi/full-xml/10.15252/emmm.202215968

publisherhttps://link.springer.com/article/10.15252/emmm.202215968/fulltext.html

publisherhttps://www.embopress.org/doi/pdf/10.15252/emmm.202215968

Fields of Study

0303 health sciences03 medical and health sciencesAnimalsMiceBrainCentral Nervous SystemDisease Models, AnimalGenetic TherapyHematopoietic Stem CellsThiolester Hydrolases

MeSH Terms

Central Nervous SystemBrainHematopoietic Stem CellsAnimalsMiceDisease Models, AnimalThiolester HydrolasesGenetic Therapy

Keywords

MicrogliaLysosomal Storage DisorderCln1Hematopoietic Stem Cell Gene TherapyCentral Nervous SystemMedicine (General)CLN1; hematopoietic stem cell gene therapy; lysosomal storage disorder; microglia610BrainArticlesGenetic TherapyQH426-470Hematopoietic Stem CellsMiceDisease Models, AnimalR5-920616GeneticsAnimalsThiolester Hydrolases