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CRISPR/Cas9 Edition of the F9 Gene in Human Mesenchymal Stem Cells for Hemophilia B Therapy

11 Dec 2024

Abstract excerpt

Hemophilia B is a genetic disorder characterized by clotting factor IX deficiency and bleeding in joints and muscles. Current treatments involve intravenous infusion of plasma-derived products or recombinant proteins, which have limited efficacy due to the short half-life of infused proteins. Recently, gene therapy for bleeding disorders has offered a potential solution. This study aimed to develop an in vitro...

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CAR-T cell therapy research
CRISPR and Genetic Engineering
Virus-based gene therapy research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 39768347
DOI: 10.3390/life14121640

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 1 Sept 2026.

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CRISPR/Cas9 Edition of the F9 Gene in Human Mesenchymal Stem Cells for Hemophilia B TherapyPMID 39768347Read ↗

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Hemophilia B: Analysis of Mutations in the F9 Gene and Perspectives for the Development of Gene Therapy (A Review)Semantic proximity Read ↗

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Targeted genome engineering in human induced pluripotent stem cells from patients with hemophilia B using the CRISPR-Cas9 systemSemantic proximity Read ↗

ssODN-Mediated In-Frame Deletion with CRISPR/Cas9 Restores FVIII Function in Hemophilia A-Patient-Derived iPSCs and ECsSemantic proximity Read ↗

CRISPR-Mediated In Situ Introduction or Integration of F9-Padua in Human iPSCs for Gene Therapy of Hemophilia B.Semantic proximity Read ↗

Long-term correction of hemophilia A via integration of a functionally enhanced FVIII gene into the AAVS1 locus by nickase in patient-derived iPSCsSemantic proximity Read ↗

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AAV mediated genome engineering with a bypass coagulation factor alleviates the bleeding phenotype in a murine model of hemophilia B.Semantic proximity Read ↗

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The most common disease-causing mutation of factor XIII deficiency is corrected by CRISPR/CAS9 gene editing system.Semantic proximity Read ↗

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