Validation of Clinical-Grade Electroporation Systems for CRISPR-Cas9-Mediated Gene Therapy in Primary Hepatocytes for the Correction of Inherited Metabolic Liver Disease

Validation of Clinical-Grade Electroporation Systems for CRISPR-Cas9-Mediated Gene Therapy in Primary Hepatocytes for the Correction of Inherited Metabolic Liver Disease

Hepatocyte transplantation (HTx) combined with ex vivo gene therapy has garnered significant interest due to its potential for treating many inherited metabolic liver diseases. The biggest obstacle for HTx is achieving sufficient engraftment levels to rescue diseased phenotypes, which becomes more c...

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Bibliographic Details
Main Authors: Justin Gibson, Abishek Dhungana, Menam Pokhrel, Benjamin Arthur, Pramita Suresh, Olumide Adebayo, Renee N. Cottle
Format: Article
Language:English
Published: MDPI AG 2025-05-01
Series:Cells
Subjects:
CRISPR-Cas9
clinical translation
gene therapy
therapeutic liver repopulation
electroporation
Online Access:https://www.mdpi.com/2073-4409/14/10/711
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