Gene therapy is an experimental technique that uses genes to treat or prevent disease. Gene therapy is designed to introduce genetic material into cells to compensate for abnormal genes or to make a beneficial protein. Although gene therapy is a promising treatment option for a number of diseases, the technique remains risky and is still under study to make sure that it will be safe and effective.There are two types of gene therapy, somatic and germ line. Genetic manipulation of germ line cells will affect the future generation so will have the serious ethical and health implications. These issues will be of effectiveness on the using and how to use of this therapy.

Recent years, allogeneic bone marrow transplantation (BMT) has proved to be the successful cure for patients with thalassemia major, however this is restricted due to limited matched-related donor. Its complications include chronic graft-versus-host disease in 5-8% of patients. So, a molecular approach, such as gene therapy for direct normal beta globin gene transmission, seems quite promising to cure thalassemia. The goal of beta thalassemia gene therapy is to restore normal RBC production capacity in patients by suitable vector and correct inherited anemia. Virus therapy has recently been recognized as a promising new therapeutic approach in medical research. In this regard, the use of viruses as non-replicating gene therapy vectors or as replication viruses (oncolytic viruses) has provided attractive opportunities for viral therapy applications. Currently viral vectors have been used in nearly 70% of the clinical trials. In recent years, many researchers have designed erythroid-specific lentiviral vectors carrying the beta-globin gene that have obtained significant results. Their findings are presented in this study.

Background & Aims: Gene therapy is used in various diseases such as cancer. Breast cancer is the most common malignancy in women worldwide, which shows the necessity of using innovative approaches in treatment methods. The ability of artificial intelligence algorithms to process large data, complex patterns, and classify them can be used to improve the process of gene therapy in breast cancer. The aim of this article is to review the available information and emphasize the applications of artificial intelligence in targeted gene therapy for breast cancer. Materials & Methods: To carry out this study we used the articles on PubMed databases by searching for related keywords to collect information. Results: By designing artificial intelligence algorithms and analyzing very complex molecular pathways in the human body and sampling the experiences of scientists and doctors in clinical studies and simulating biological processes related to the regulation of gene expression in the human body, the effectiveness of gene carriers, control of gene delivery parameters/medicine and modeling of cells minimized the rate of medical errors and with early diagnosis of the disease and predicting the effectiveness of the medicine, it provided patient-centered treatments of the effectiveness of new treatments such as gene therapy with the least complications at the highest level. Conclusion: In recent decade, many efforts have been made to use all types of gene therapy for breast cancer patients with the least complications and the most effectiveness. Therefore, artificial intelligence is a powerful tool for optimizing early diagnosis and treatment for breast cancer. It’s combination with interdisciplinary sciences in improving the health of the society is a very interesting topic for scientists, but due to the limitations that exist for its use, such as ethical cases and high costs, it should be done with high precision and sufficient studies.

Sjögren syndrome is one of the autoimmune diseases which is characterized by lymphocytic infiltration to exocrine glands and causes keratoconjunctivitis sicca and xerostomia. Today, a large population, with a majority of women over 40, suffers from this disease and has several complications regarding oral health and reduced life quality such as severe dental caries, painful eyes, olfactory and gustatory deficiency, speech, mastication and swallowing discomforts. Unfortunately, these patients do not respond to the conventional therapies. Nowadays in medical world, which its target is basic therapy and not symptomatic one, several gene therapy approaches, have gained importance in treatment of this apparently incurable diseases. Due to the facts that this disease is the second prevelant autoimmune disease, after rheumatoid arthritis, and the conventional therapies of the disease are all relative and symptomatic, researchers have insisted on the basic and causative therapy through gene transfer more than before. In the Present article, through reviewing 58 references containing recent scientific and investigatory findings it has been tried, to consider the pathogenesis and conventional therapies of this syndrome. Another purpose of this study was to investigate several and potentially very effective gene transfer systems and different theraputic genes (mainly membrane water channels, ione transporter molecules, transcription factors, antifungal proteins and free radical scavengers).

2Thalassemia is an autosomal recessive hereditary disease that occurs due to a decrease in the synthesis of Please recheck. In beta thalassemia, defects in β-globin synthesis lead to an imbalance of β- and α-globin chains and the accumulation of α4 chains in the erythroid precursor which leads to ineffective erythropoiesis, shortened red blood cell survival, and finally clinical symptoms such as delayed sexual and physical maturation, endocrine dysfunction, cardiomyopathy, liver disease, bone deformities and hepatosplenomegaly. Current treatments such as transfusion, iron chelating agents and allogeneic stem cell hematopoietic transplantation have limitations in their use, including iron overload, lack of a human leukocyte antigen (HLA) matched compatible donor, and graft versus host disease (GVHD). Gene therapy is a new therapeutic option for beta thalassemia patients that induces the continuous expression of beta globin chains in the patient’s hematopoietic stem cells. The idea of gene therapy was first proposed in the early 1970s, and the ultimate goal of this treatment method is to express the defective gene in the target cell in a way that can reduce the symptoms of the disease or eliminate them (symptoms) altogether. There are two general methods for gene therapy: the integrating vector, in which the desired gene is inserted into the genome of the target cell and its lifelong expression follows, is the non-integrating method, in which the vector doesn’t integrate into the genome of the target cell and the cytoplasmic form enables gene expression. The first beta thalassemia gene therapy was performed in France in 2006, and in this clinical trial, the first patient with the E/β0 thalassemia was treated at the age of 18. Gene therapy for beta-thalassemia has been approved by the food and drug administration in 2022 for patients aged 12 years and older who have a non β0/β0 phenotype. It seems that this therapeutic option is the definitive treatment method for blood transfusion-dependent beta-thalassemia patients.     However, this treatment method still has limitations: high cost, sensitivity of lentiviral vector production, and the possibility of integration of the vector near the proto-oncogene and its activation are some of them.

Gene therapy is a new arena in which the treatment of many human diseases like cancer and cardiovascular disease can be achieved by using knowledge of genetic engineering. This kind of treatment is very young and its use goes back to about two decades before. It is in close communication and interaction with other fields like medicine, biology, ethics, law, philosophy, religion, politics and social issues and is still in its early stages of progress. Along with therapeutic effectiveness and the significant developments in this field of science, there are some problems in using this method like as developing viral infections, the financial costs, the need for repeating procedures, possible misuse of gene therapy as a weapon, allowing it to exploit a phenomenon called eugenics and custom children. Gene Therapy as a treatment is introduced (as described in the article) and close observation of gene therapy from Medical, ethical and legal view points are necessary and require continuous monitoring and legitimating it. The beneficial effects of this therapy and bitter experience of improper use of these methods in medical history shows how much we need ethical and legal attention in this field.