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Updated: Jul 8, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Molecular therapy and prevention of liver diseases
1Department of Medicine II, University Hospital, D-79106 Freiburg, Germany. hubert.blum@uniklinik-freiburg.de
Abstract:
Molecular analyses have become an integral part of biomedical research as well as clinical medicine. The definition of the molecular and genetic basis of many human diseases has led to a better understanding of their pathogenesis and has in addition offered new perspectives for their diagnosis, therapy and prevention. Genetically, liver diseases can be classified as hereditary monogenic, acquired monogenic, complex genetic and diseases. Based on this classification, gene therapy is based on six concepts: gene repair, gene substitution, cell therapy, block of gene expression or function, DNA vaccination as well as gene augmentation. While recent developments are promising, various delivery, targeting and safety issues need to be addressed before gene therapy will enter clinical practice. In the future, molecular diagnosis and therapy liver diseases will be part of our patient management and complement existing diagnostic, therapeutic and preventive strategies.
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