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Article

In vivo enrichment of genetically manipulated platelets for murine hemophilia B gene therapy.

Journal of cellular physiology - 1 Jan 2021

Chen Yingyu, Schroeder Jocelyn A, Gao Chunyan, Li Jing, Hu Jianda, Shi Qizhen

Abstract excerpt

Our previous studies have demonstrated that platelet-targeted factor IX (FIX) gene therapy can introduce sustained platelet-FIX expression in hemophilia B (FIXnull ) mice. In this study, we aimed to enhance platelet-FIX expression in FIXnull mice with O6 -methylguanine-DNA-methyltransferase (MGMT)-mediated in vivo drug selection of transduced cells under nonmyeloablative preconditioning. We constructed a novel...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 32510630
DOI: 10.1002/jcp.29861
PMCID: PMC7722216

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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