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  • A review context on the current concepts of gene therapy

  • Hossein Aftabi,1,*
    1. Department of Biology, Faculty of Sciences, Shahid Bahonar University of Kerman, Iran


  • Introduction: Introduction: Gene therapy is 21 century medical technique that treats diseases by introducing the therapeutic genes into target cells. The human body includes about 4 × 10¹³ cells having 30,000 genes in each cell. Any mutations in the genes may cause several genetic disorders and diseases. Gene therapy techniques treat monogenic diseases and cancer through the ex vivo or in vivo gene inserting. The objective of this review is to present the current concepts on the human gene therapy in treating cancer, cardiac, Parkinson, Alzheimer and immunodeficiency diseases the limitations of the gene therapy techniques.
  • Methods: Methods: This review is based on the available gene therapy data in PubMed, Science Direct, Scopus, Web of Science and Google Scholar from 2012 to 2026.The most relevant keywords were related to gene therapy, DNA, monogenic diseases, viral vectors, non-viral vectors, in vivo and ex vivo and the most relevant papers were included in this contribution.
  • Results: Results: Gene therapy involves as germline and somatic gene therapy. In germline therapy, the target cells are inserted by germ-line, eggs or sperm cells, thus genetic codes will be transferred to future generations and gives significant results in preventing diseases. Somatic cell gene therapy is the most preferable technique, where target therapeutic gene is inserted into the physical cells. This results in curing the patient without healing or inheriting the offspring, but the children of the patient may be affected by the same diseases. Somatic gene therapy may be involved by either Ex-vivo or In-vivo. In EX-vivo technique, the cells are taken from the physical cells, amended in the laboratory and then used in targeted patients, whereas in In-vivo technique, the genes are directly introduced into the targeted cells of the patients. The delivery techniques are viral vectors and non-viral vectors. The viral vectors are considered as the safer delivery methods and use adenoviruses (double-stranded DNA genome), lentiviruses and adeno-associated viruses (AAV) to transfer the therapeutic gens into target cells. The non-viral methods do not use viruses, but use liposomes, electroporation and microinjection to transfer the genes into the target cells. Gene therapy has treated the following diseases: Cancer: The clinical results on malignancy and cancer indicate that about 67% data on gene therapy are tested on cancer by an anti-cancer drug of Gendicine, yet drinkable gene therapy has also been used in treating esophageal carcinoma, yet suicide gene therapy has been proposed for future cancer treatment. Immunodeficiency: Clinical data indicate considerable therapeutic benefits that may cure X-linked severe combined immunodeficiency (X-SCID) related to the deficiency of adenosine deaminase (ADA), although Human Immunodeficiency Virus (HIV) may be treated for future gene therapy. Cardiovascular: Gene therapy data do not show significant progress in stress-induced myocardial perfusion, but some anti-ischemic effects are reported to treat the cardiovascular diseases. Single gene disorder: Gene therapy records some treatments in fibrosis, muscular dystrophies, junctional epidermolysis, haemophilia, and other single gene disorder diseases. Parkinson: little data are available on the gene therapy for Parkinson disease, yet transferring glutamic acid carboxylase (GAD) gene into the basal ganglia cells of cerebral areas in brain shows a decrease in the level of aminobutyric acid (GABA) in patients having Parkinson syndrome Alzheimer: By transferring Adeno Associated Virus (AAVs) into targeted cells no convincing effects are reported in preventing Alzheimer’s disease, thus healthy lifestyle is suggested for treating the Alzheimer. Diabetes: Injecting the vascular endothelial growth factor (VEGF) gene into the intramuscular connective tissues showed a convincing treatment for diabetic patients. Eye diseases: Gene therapy gives convincing results regarding the treatment of Leber Congenital Amaurosis (LCA), glaucoma, red-green color blindness and other eye diseases.
  • Conclusion: Conclusion: This review highlights that gene therapy is a promising medical technique and market-approved medicinal products for treating different diseases. Cancer is the most common disease (>60%) to be treated by gene therapy, followed by monogenic and cardiovascular diseases. The most advantages of gene therapy rest on the genuine germ-line gene therapy for positive treating of some genetic diseases. The main disadvantages include some medical risks, costly-technique and moral rights of the offspring generations.
  • Keywords: Keywords: Gene therapy, Monogenetic diseases, Cancer, Cardiac

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