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Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency

Molecular Therapy(2024)10.1016/j.ymthe.2024.08.015Source: DataRank Database

Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency is a research paper published in Molecular Therapy (2024). On theSindex it has a DataRank of 0. It has been cited 3 times.

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Open Access3 citations

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Enrichment:Funding (6 grants)OA: hybridTopics (23)IDs (PubMed, PMC)Annotations (1)

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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 (16)

Lucas Tricoli ORCID,Xinying Hong ORCID,Parith Wongkittichote ORCID,Carlo Castruccio Castracani ORCID,Amaliris Guerra ORCID

Live enrichment

PubMed:39169621 PMC:PMC11573602

National Institutes of Health

Grant: K23NS114113

National Institutes of Health

Grant: U54TR002823

Health Research Board

CHEO Research Institute

Meso Scale Diagnostics

The Children's Hospital of Philadelphia

hybridLicense: cc-by-nc-nd

publisherhttps://doi.org/10.1016/j.ymthe.2024.08.015

publisherhttps://api.elsevier.com/content/article/PII:S1525001624005380?httpAccept=text/xml

publisherhttps://api.elsevier.com/content/article/PII:S1525001624005380?httpAccept=text/plain

repositoryhttps://pmc.ncbi.nlm.nih.gov/articles/PMC11573602/pdf/main.pdf

repositoryhttps://resolver.sub.uni-goettingen.de/purl?gro-2/144887

MeSH Terms

Hematopoietic Stem CellsAnimalsHumansMiceLentivirusDisease Models, AnimalSulfatasesGlycosaminoglycansHematopoietic Stem Cell TransplantationGenetic VectorsOxidoreductases Acting on Sulfur Group DonorsMultiple Sulfatase Deficiency DiseaseGenetic Therapy

Keywords

Lysosomal Storage DisorderFormylglycine-generating EnzymeMsdHematopoietic Stem Cell TransplantEx vivo Gene TherapySumf1Multiple Sulfatase DeficiencyLentiviral Gene TherapyFgeSulfatase Modifying Factor 1

Hematopoietic stem cell gene therapy improves outcomes in a clinically relevant mouse model of multiple sulfatase deficiency

FAIR Checklist

Authors (16)

Funding (6 grants)

Open Access

Topics

Annotations (Europe PMC)