Potential Use of Patient-Specific Induced Pluripotent Stem Cell for Liver Fibrosis Thalassemia Treatment Management

Susi Susanah; Wahyu Widowati; Nur Melani Sari; Revika Revika; Hanna Kusuma; Rizal Rizal; Ahmad Faried

doi:10.3889/oamjms.2022.8326

Authors

Susi Susanah Department of Child Health, Faculty of Medicine, Universitas Padjadjaran, Dr. Hasan Sadikin Hospital, Bandung, Indonesia
Wahyu Widowati Department of Pharmacology, Faculty of Medicine, Maranatha Christian University, Bandung, Indonesia
Nur Melani Sari Department of Child Health, Faculty of Medicine, Universitas Padjadjaran, Dr. Hasan Sadikin Hospital, Bandung, Indonesia; Oncology and Stem Cell Working Group, Universitas Padjadjaran, Dr. Hasan Sadikin Hospital, Bandung, Indonesia https://orcid.org/0000-0002-9062-9624
Revika Revika Department of Biotechnology, Faculty of Technobiology, Atma Jaya Catholic University of Indonesia, South Tangerang, Banten, Indonesia
Hanna Kusuma Biomolecular and Biomedical Research Center, Aretha Medika Utama, Bandung, Indonesia
Rizal Rizal Biomolecular and Biomedical Research Center, Aretha Medika Utama, Bandung, Indonesia; Department of Electrical Engineering, Biomedical Engineering, Faculty of Engineering, Universitas Indonesia, Depok, Indonesia
Ahmad Faried Oncology and Stem Cell Working Group, Universitas Padjadjaran, Dr. Hasan Sadikin Hospital, Bandung, Indonesia https://orcid.org/0000-0003-2967-8125

DOI:

https://doi.org/10.3889/oamjms.2022.8326

Keywords:

Thalassemia, Iron overload, Liver fibrosis, Induced pluripotent stem cells-hepatic stellate cells, Management

Abstract

Thalassemia is the most common inherited single gene blood disease worldwide and present a significant health problem in the world. Approximately, 1.5% of the global populations (An estimated 80–90 million people) are carriers of β-thalassemia. Around 5% of Indonesia population is thought to carry the thalassemia gene. The globin imbalance in β-thalassemia major causes hemolysis and ineffective erythropoiesis which results in anemia leading to increases of iron absorption. Furthermore, repeated blood transfusion and long-term increased iron absorption will lead to excessive accumulation of iron in vital organs, especially in the liver, causes liver fibrosis then leading to liver disease. Iron overload can be controlled by iron chelating drugs with the risk of side effects; therefore, a breakthrough is needed. Stem cell technology has a potential to provide novel insight in thalassemia major, through induced pluripotent stem cells (iPSCs) who has the ability to differentiate into hepatic stellate cells (HSCs)-like cells. iPSCs derived HSC-like cells (iPSC-HSCs) present the phenotypic and functional characteristics of HSCs. The utilization of iPSCs is a new option in personalized thalassemia management.

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