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

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

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. Recent...

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Bibliographic Details
Main Authors: Irving Jair Lara-Navarro, Luis Felipe Jave-Suárez, Juan Antonio Marchal, Ana Rebeca Jaloma-Cruz
Format: Article
Language:English
Published: MDPI AG 2024-12-01
Series:Life
Subjects:
hemophilia B
lentiviral vector
CRISPR/Cas9 system
mesenchymal stem cells
gene therapy
Online Access:https://www.mdpi.com/2075-1729/14/12/1640
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