Журнал диабета и клинической практики

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The Liver can Benefit from Gene Therapy and Therapeutic Genome Editing

Jerry Pitter

Gene therapy and therapeutic genome editing have emerged as promising approaches for the treatment of various diseases, including liver disorders. The liver, being a vital organ involved in numerous metabolic processes, presents a unique opportunity for these innovative techniques. This abstract explores the potential benefits of gene therapy and therapeutic genome editing in addressing liver disorders and highlights recent advancements in the field. Liver disorders encompass a wide range of conditions, such as genetic liver diseases, viral hepatitis, alcoholic liver disease, non-alcoholic fatty liver disease (NAFLD), and liver cancer. Traditional treatment options for these disorders often focus on symptom management and disease progression mitigation. However, gene therapy and therapeutic genome editing offer new possibilities for more targeted and curative interventions. Gene therapy in liver disorders involves delivering therapeutic genes to replace or supplement defective genes responsible for genetic liver diseases. By introducing functional genes into liver cells, disease symptoms can be alleviated, and potential cures may be achieved. Additionally, gene therapy can modulate gene expression in the liver, potentially halting or reversing the progression of liver disorders by targeting specific genes involved in fibrosis or inflammation.