Molecular biology and gene therapy in gastroenterology and hepatology

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, human diseases can be classified as monogenetic, complex genetic and acquired genetic diseases. Based on this classification, gene therapy is based on four concepts: gene substitution, gene augmentation, block of gene expression or function as well as DNA vaccination. 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 gene therapy of gastrointestinal and liver diseases will be part of our patient management and complement existing diagnostic, therapeutic and preventive strategies.