REVIEW article
Front. Bioeng. Biotechnol.
Sec. Cell and Gene Therapy
Transformative Approaches in Hemophilia Management: From Traditional Therapies to Prenatal Stem Cell Treatment
Provisionally accepted
YUNG-TSUNG KAO1
Hueng-chuen Fan2
Jen-Kun Chen3
Chih-Ching Yen4
Muhammad Sufian1
Shang-Hsun Yang5
Chuan-Mu Chen1*- 1National Chung Hsing University, Taichung, Taiwan
- 2Tungs' Taichung MetroHarbor Hospital, Wuqi, Taiwan
- 3National Health Research Institute, Miaoli, Taiwan
- 4China Medical University Hospital, Taichung, Taiwan
- 5National Cheng Kung University Hospital, Tainan City, Taiwan
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The increased global incidence of hemophilia, along with its concomitant consequences arising from prolonged hemorrhage after an injury and a heightened vulnerability to internal bleeding in joints or the brain, brings to the forefront the development of innovative therapeutic strategies to alleviate this hereditary genetic disorder. Current treatments for hemophilia primarily depend on plasma concentrates; however, their widespread use is constrained by the reliance on blood donations and the associated risk of infections. Alternative protein-based therapeutics, such as recombinant coagulation factors, bypassing agents, and non-factor-based drugs, have been approved and utilized for the prophylaxis and treatment of hemophilia with promising outcomes; yet, their shortcomings consist of the necessity for repeated administration and the likelihood of inhibitor formation. With the emergence of the cutting-edge gene editing (CRISPR/Cas9) and gene delivery (viral and non-viral vectors) techniques, the recent progression in gene therapy, stem cell transplantation, and prenatal interventions via in utero stem cell therapy inspires optimism for individuals afflicted with hemophilia and their families, even though these innovative techniques remain in the preclinical stage with a lot of technical and ethical issues needing to be resolved. This review article provides a comprehensive overview of hemophilia management, from traditional therapies to advanced prenatal stem cell treatments, highlighting the evolution and future directions in addressing this genetic bleeding disorder.
Keywords: Hemophilia, Gene Therapy, Bypassing therapy, in utero stem cell therapy, Prenatal treatment
Received: 12 Aug 2025; Accepted: 29 Oct 2025.
Copyright: © 2025 KAO, Fan, Chen, Yen, Sufian, Yang and Chen. 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: Chuan-Mu Chen, chchen1@dragon.nchu.edu.tw
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.