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Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency

Science Translational Medicine(2024)10.1126/scitranslmed.adh8162Source: DataRank Database

Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency is a research paper published in Science Translational Medicine (2024). On theSindex it has a DataRank of 0. It has been cited 23 times.

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0DataRank · unranked

Scored on demand from live citation data

Open Access23 citations

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›Methodology & internals

datarank_citation_only_1hop_v6· scope data_onlyMethodology

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Enrichment:Funding (4 grants)Impact (FWCI 3.92)OA: greenTopics (49)IDs (PubMed, PMC, OpenAlex)Annotations (3)SDGs (2)

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 (34)

Chiara Brandas ORCID,Samuele Ferrari ORCID,Simona Porcellini ORCID,Nicolò Sacchetti,Daniele Canarutto ORCID

Live enrichment

PubMed:38324638 PMC:PMC11149094 OpenAlex:https://openalex.org/W4391593863

Intramural NIH HHS

Grant: ZIA AI001222

NIAID NIH HHS

Grant: R01 AI155796

National Institutes of Health

Grant: 1ZIAAI001222-08

Characterization of the cellular and molecular bases of inborn errors of immunity

National Institutes of Health

Grant: 7R01AI155796-04

Development, Optimization and Preclinical Modeling of Hematopoietic Stem Cell Gene Editing for the Treatment of RAG1 Immunodeficiency

FWCI

3.92

Citation Percentile

0.9%

Citation Trend

2024

2025

2026

greenLicense: CC BY NC ND

repositoryhttps://pmc.ncbi.nlm.nih.gov/articles/PMC11149094/pdf/nihms-1986073.pdf

publisherhttps://www.science.org/doi/pdf/10.1126/scitranslmed.adh8162

repositoryhttps://www.ncbi.nlm.nih.gov/pmc/articles/11149094

journalhttps://doi.org/10.1126/scitranslmed.adh8162

Fields of Study

CRISPR and Genetic EngineeringVirus-based gene therapy researchCAR-T cell therapy research0301 basic medicine0303 health sciences03 medical and health sciencesAnimalsHumansMiceExonsGene EditingHematopoietic Stem Cell TransplantationHematopoietic Stem CellsHomeodomain Proteins

MeSH Terms

Gene EditingAnimalsExonsHematopoietic Stem CellsHumansHematopoietic Stem Cell TransplantationHomeodomain ProteinsMice

Keywords

Recombination-activating geneBiologyRAG2Severe combined immunodeficiencyGenome editingProgenitor cellExonHaematopoiesisGeneGeneticsGene knockinGenetic enhancementStem cellCell biologyCancer researchCRISPRRecombinationGene EditingHomeodomain ProteinsHematopoietic Stem Cell TransplantationExonsHematopoietic Stem CellsGene Therapy, BioinformaticsMiceExonic knockout and knockin gene editingAnimalsHumans

Sustainable Development Goals

SDG 3: 3. Good healthSDG 0: Good health and well-being

Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency

FAIR Checklist

Authors (34)

Funding (4 grants)

Impact

Open Access

Topics

Annotations (Europe PMC)