Molecular Updates in Gene Therapy of Liver Diseases


  • Abousree T. M. Ellethy Assistant Professor, Department of Basic Oral Sciences and Dental Education, College of Dentistry, Qassim University, Saudi Arabia
  • Gamal M. A. Hassan Professor, Department of Anatomy, College of Medicine, Suez Canal University, Ismailia, Egypt



gene therapy, monogenic and polygenic disorders, CRISPR-Cas9, genome editing delivery system, recombinant adeno-associated virus vectors, exosome, genetic disorders, liver diseases, hemophilia, FDA


Gene therapy is an advanced treatment approaches which are involved in insertion of genetic materials to the target cells for diseases management. It involves correcting defective genes that are appropriate for treating diseases caused by single gene failure in particularly suitable rare diseases e.g., cystic fibrosis, adrenoleukodystrophy beside liver diseases and cancers. It is looking forward to transfigure modern medicine for cure of numerous inherited metabolic liver disorders. Urgently, effective and continuous advanced biotechnology is required in developing nanoparticles vehicles, deliver mRNA biomolecules and target host cell genome. This is to reinsert the missed gene expressions, functional proteins within the target cells and disabling many obstacles. Gene editing aims to change the microorganism genetic material of DNA. It permits insertion of genetic materials, deletion, or alterations within a particular location within the gene. Many procedures have been investigated for getting gene editing. Currently, CRISPR-Cas9 is well designed DNA editing system and identified as regularly interspaced short palindromic repeats and associated with protein-9. The CRISPR-Cas9 system is cheap and fast, with high accuracy and efficiency comparing to other genetic editing tools. Molecular progress will avoid stimulating innate immunological responses. Many research studies started clinical trials based on the allowed advance in nanotechnology and molecular biology tools. They highlighted therapies to treat such liver disorders with safe and effective approaches. This study summarizes and discusses the progress in liver diseases gene therapy with a hope for success of clinical trials applications through advanced therapeutic potentials and gene editing.


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How to Cite

A. T. M. Ellethy and G. M. A. Hassan, “Molecular Updates in Gene Therapy of Liver Diseases”, IJRAMT, vol. 5, no. 4, pp. 65–72, Apr. 2024, doi: 10.5281/zenodo.11067723.