Induced Pluripotent Stem Cell Meets Severe Combined Immunodeficiency

(Pages: 1-10)
Reza Kouchaki, B.Sc, 1Bahareh Abd-Nikfarjam, Ph.D, 2Amirhosein Maali, M.Sc, 3Saeid Abroun, Ph.D, 4Farshad Foroughi, Ph.D., 2Sasan Ghaffari, M.Sc., 5Mehdi Azad, Ph.D., 1,*
Faculty of Allied Medicine, Qazvin University of Medical Sciences, Qazvin, Iran
Department of Immunology, School of Medicine, Qazvin University of Medical Sciences, Qazvin, Iran
Student Research Committee, Pasteur Institute of Iran, Tehran, Iran
Department of Hematology and Blood Banking, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, Iran
Hematology Department, School of Allied Medicine, Tehran University of Medical Sciences, Tehran, Iran
Faculty of Allied Medicine, Qazvin University of Medical Sciences, Qazvin, Iran
Department of Immunology, School of Medicine, Qazvin University of Medical Sciences, Qazvin, Iran
Student Research Committee, Pasteur Institute of Iran, Tehran, Iran
Department of Hematology and Blood Banking, Faculty of Medical Sciences, Tarbiat Modares University, Tehran, Iran
Hematology Department, School of Allied Medicine, Tehran University of Medical Sciences, Tehran, Iran
*Corresponding Address: P.O.Box: 34197-59811 Faculty of Allied Medicine Qazvin University of Medical Sciences Qazvin Iran Email:haematologicca@gmail.com
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Kouchaki Reza, Abd-Nikfarjam Bahareh, Maali Amirhosein, Abroun Saeid, Foroughi Farshad, Ghaffari Sasan, Azad Mehdi. Induced Pluripotent Stem Cell Meets Severe Combined Immunodeficiency . Cell J. 2020; 22(): 1-10.

Abstract

Severe combined immunodeficiency (SCID) is classified as a primary immunodeficiency, which is characterized by impaired T-lymphocytes differentiation. IL2RG, IL7Ralpha, JAK3, ADA, RAG1/RAG2, and DCLE1C (Artemis) are the most defective genes in SCID. The most recent SCID therapies are based on gene therapy (GT) of hematopoietic stem cells (HSC), which are faced with many challenges. The new studies in the field of stem cells have made great progress in overcoming the challenges ahead. In 2006, Yamanaka et al. achieved "reprogramming" technology by introducing four transcription factors known as Yamanaka factors, which generate induced pluripotent stem cells (iPSC) from somatic cells. It is possible to apply iPSC-derived HSC for transplantation in patients with abnormality or loss of function in specific cells or damaged tissue, such as T-cells and NK-cells in the context of SCID. The iPSC-based HSC transplantation in SCID and other hereditary disorders needs gene correction before transplantation. Furthermore, iPSC technology has been introduced as a promising tool in cellular-molecular disease modeling and drug discovery. In this article, we review iPSC-based GT and modeling for SCID disease and novel approaches of iPSC application in SCID.