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Article

Development and Characterization of a Factor V-Deficient CRISPR Cell Model for the Correction of Mutations.

International journal of molecular sciences - 22 May 2022

Serrano Luis Javier, Garcia-Arranz Mariano, De Pablo-Moreno Juan A, Segovia José Carlos, Olivera-Salazar Rocío, Garcia-Olmo Damián, Liras Antonio

Abstract excerpt

Factor V deficiency, an ultra-rare congenital coagulopathy, is characterized by bleeding episodes that may be more or less intense as a function of the levels of coagulation factor activity present in plasma. Fresh-frozen plasma, often used to treat patients with factor V deficiency, is a scarcely effective palliative therapy with no specificity to the disease. CRISPR/Cas9-mediated gene editing, following precise...

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PMID: 35628611
DOI: 10.3390/ijms23105802
PMCID: PMC9148015

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