Advances in Gene Therapy Clinical Trials for Hemophilia Care

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Mini-review synthesizes data on AAV-based gene therapies for hemophilia, highlighting efficacy and challenges.

Gene therapy shows promising long-term production of coagulation factors in hemophilia, aiming for curative outcomes.
Recent trials report on efficacy, durability, and immunogenicity of AAV-based treatments in hemophilia patients.
Advancements in vector design have enhanced transgene expression but challenges like immune responses remain.
Emerging techniques like CRISPR-Cas9 offer potential improvements in gene therapy applications for hemophilia.

Rana et al. (2025) studied this question.

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