REVIEW article
Front. Cell Dev. Biol.
Sec. Stem Cell Research
Volume 13 - 2025 | doi: 10.3389/fcell.2025.1595897
This article is part of the Research TopicAdvancements in Stem Cell Differentiation and Disease ApplicationsView all 5 articles
CRISPR/Cas-Edited iPSCs and Mesenchymal Stem Cells: A Concise Review of Their Potential in Thalassemia Therapy
Provisionally accepted
- 1Zunyi Medical University, Zunyi, China
- 2Affiliated Hospital of Zunyi Medical University, Zunyi, Guizhou Province, China
- 3Guizhou Medical University, Guiyang, Guizhou Province, China
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Thalassemia, a prevalent single-gene inherited disorder, relies on hematopoietic stem cell or bone marrow transplantation as its definitive treatment. However, the scarcity of suitable donors and the severe complications from anemia and iron overload pose significant challenges. An immediate necessity exists for a therapeutic method that tackles both the illness and its associated complications. Advancements in stem cell technology and gene editing methods such as CRISPR/Cas offer encouraging prospects for a therapy that could liberate patients from the necessity of ongoing blood transfusions and iron chelation treatments. The potential of genetic reprogramming using induced pluripotent stem cells (iPSCs) to address thalassemia is highly promising. Furthermore, mesenchymal stem cells (MSCs), recognized for their capacity to self-renew and differentiate into multiple lineages that include bone, cartilage, adipose tissue, and liver, demonstrate potential in alleviating several complications faced by thalassemia patients, including osteoporosis, cirrhosis, heart conditions, respiratory issues, and immune-related disorders. This review synthesizes and summarizes relevant studies to assess the therapeutic potential and predict the curative effects of these cellular approaches.
Keywords: Thalassemia, Gene Therapy, IPSC, MSc, complication
Received: 18 Mar 2025; Accepted: 24 Jun 2025.
Copyright: © 2025 Shu, Xie, Wang, Du, Huang, Chen and He. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence:
Jiaojiao Shu, Zunyi Medical University, Zunyi, China
Yan Chen, Affiliated Hospital of Zunyi Medical University, Zunyi, 563000, Guizhou Province, China
Zhixu He, Affiliated Hospital of Zunyi Medical University, Zunyi, 563000, Guizhou Province, China
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