ReviewFrontiers in bioengineering and biotechnology2025
Transformative approaches in hemophilia management: from traditional therapies to prenatal stem cell treatment.
Review in Frontiers in bioengineering and biotechnology, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
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
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