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

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

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 e...

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Bibliographic Details
Main Authors: Luis Javier Serrano, Mariano Garcia-Arranz, Juan A. De Pablo-Moreno, José Carlos Segovia, Rocío Olivera-Salazar, Damián Garcia-Olmo, Antonio Liras
Format: Article
Language:English
Published: MDPI AG 2022-05-01
Series:International Journal of Molecular Sciences
Subjects:
gene therapy
gene editing
CRISPR
factor V deficiency
coagulopathies
rare diseases
Online Access:https://www.mdpi.com/1422-0067/23/10/5802

Internet

https://www.mdpi.com/1422-0067/23/10/5802

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