الفهرس 
IPSCsOnly 14 pages are availabe for public view
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Abstract
Induced pluripotent stem cells are somatic cells that have been genetically reprogrammed, in such a way that they lose their tissue-specific qualities and become pluripotent. IPSCs resemble ESCs in many features, but are considered morally superior as they do not require destruction of embryos and being patient specific as they circumvent concerns over immune rejection with stem cell therapy.The therapeutic promise of iPSCs is considerable, with potential for drug development and testing, novel assisted reproductive techniques and disease in a dish modeling. The applications for iPSCs in medicine are extremely exciting, but the technology needs significant safety checks before it can become a reality. Cells may be cultured from a patient and reprogrammed to form iPSCs. The patient’s own iPSCs could then be repaired using gene therapy, or re-differentiated into cell types of interest and studied for disease pathophysiology and drug development and screening. The outcome of both of options should benefit the patient, either repaired healthy tissue is transplanted back into the patient, or patient and disease specific drugs are given to cure or control the disease.There are many problems with iPSC technology, which will need to be closely monitored if iPSCs are ever to be used in a clinical setting, the first obstacles is that reprogramming using OKSM is a slow and inefficient process, with only a small proportion (0.01-0.2%) of the culture progressing to pluripotency. This problem may be due to heterogeneous transgene expression in the starting cells, but even with improving the universality of transgene expression, such as polycistronic cassettes and the secondary reprogramming systems, the number of fully reprogrammed colonies remains low compared to the number of cells in culture.The second obstacles is that integrative methods of reprogramming can result in insertional mutagenesis, the integration of retroviruses into the host genome causing deregulation of nearby genes. This problem is exacerbated because cells can be infected repeatedly, acquiring more and more mutations. The more mutations the integration causes, the more likely the insertion will lead to the development of a tumour. This problem lead to the development of non integrational methods of reprogramming, but even without viral integration cancer is still a real concern. Thalassemia is an inherited disorder of autosomal recessive gene disorder caused by impaired synthesis of one or more globin chains. The impairment alters production of hemoglobin (Hb). Thalassemia causes varying degrees of anemia, which can range from asymptomatic to life threatening anemia. People of Mediterranean, Middle Eastern, African, and Southeast Asian descent are at higher risk of carrying the genes for thalassemia.
Thalassemia occurs when there is decreased or absent production of one of the types of globin chains (most commonly either α or β), that cause insufficeient amount of normal structure globin chains. This results in an imbalance between α and β chains and causes the clinical features of thalassemia, it can be separated into two major types such as α and β-thalassemia.