Results: Representative Lentiviral Vector-mediated Hepatocyte Liver Function Rescue
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Conclusion
Trascrizione
Ex vivo gene therapy and autologous cell transplantation for the treatment of inborn errors of metabolism in the liver is an important alternative to the therapy for these disorders. And allows us to not only treat these disorders, but figure out
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This protocol is intended to describe porcine hepatocyte isolation and ex vivo gene delivery to cure models of metabolic diseases via autologous cell transplantation. Although this particular model enjoys unique advantages that favor successful therapy, the application is a relevant foundation to address additional diseases and indications.