Introduction: Genetic diseases are diseases that a person is born with and its symptoms appear at any age depending on the type of disease, and one of the most efficient and safe ways to treat it is using stem cells. Genetic diseases such as; Hereditary diseases focused on dry skin, childhood neurological disorders, Duchenne muscular dystrophy and myotonic muscular dystrophy, coronary artery disease, dilated cardiomyopathy, congenital defects in the production or function of blood cells, metabolic diseases, beta thalassemia ,Genetic immunodeficiency, cancer, hemoglobinopathy, genetic bone problems, cartilage repair, autoimmune disorders, and heart and nerve diseases can be treated using stem cells. Stem cells exist in most parts of the body and have the ability to self-replicate and differentiate into multiple lineages, and treatment by these methods is the result of the advancement of regenerative medicine.
Methods: This is a research and review with PubMed, Google, Springer, Science Direct, Disease Information Search and Sciencehub that focuses on the treatment of genetic diseases using stem cells.
Results: Somatic stem cells help in tissue regeneration, including in burns and severe injuries in patients suffering from life -threatening immunodeficiencies, in which stem cells are isolated and genetically manipulated outside the body and re-infused. For example, the efficacy of SCAD gene therapy in severe combined immunodeficiency of adult epidermal stem cells in vivo. They have the capacity to renew the epidermis, covering, differentiating and protecting our body, which can be a replacement for damaged skin.
Human nerve cells cannot be studied due to their unavailability, but with cell programming techniques, a new source of human cells can be prepared for laboratory research. Induced pluripotent stem cells are a method that can differentiate into specific neuronal subgroups such as: dopaminergic, motor, inhibitory GABAergic and cortical neurons.
Duchenne muscular dystrophy is a neurological disorder that causes progressive muscle weakness and atrophy, and the most common and severe type is DMD, in which 1 in 3,500 to 5,000 boys have a mutation in the X chromosome gene responsible for dystrophin. , which is a coding generator. It provides both management support and investment. The use of therapeutic cells is based on stem cells that use myoblasts, satellite cells, marrow and bone cells, mesoangioblasts and finally HPSC cells from CD 133.
The main nature of mesenchyme and non-mesenchyme is different and they differ in terms of lineage, culture conditions, important factors and surface markers.
Correction of a genetic disease by gene editing mediated by CRA SPR-CAS9 in spermatogonial stem cells of SCT stem cell transplantation and for the treatment or improvement of a wide range of genetic diseases from inherent defects in blood cell production or function to metabolic diseases that They are mainly used to affect solid organs.
Spermatogonial stem cells can produce multiple male gametes after transplantation into the recipient's testes, and it is a valuable approach for gene therapy and continuous production of genetically modified cells.
Conclusion: All methods found to treat diseases and genetic defects using stem cells are under research, but many of them have already been successfully registered.
During the last few years, the rapid development of isolation and differentiation techniques has been noted in order to achieve effective transplantation results of myogenic HPSC cells.
Mesenchymal and non-mesenchymal stem cells have immunoregulatory capacity, elicit immunosuppressive effects, and are immune privileged due to low expression of MHCII stimulatory molecules on the cell surface.
To use stem cells in regeneration and repair, one must know information about their origin, fate and functional ability.
So far, 7718 clinical trials based on stem cells have been registered in the databases of the National Institutes of Health of the United States of America until January 25, 2020, of which 3015 trials have been conducted and had favorable results, and it should be noted that these trials are similar to studies based on HSC and MSC is in progress.