AUG 26, 2026 · JOURNAL ARTICLE
Engineered hypoimmunogenic iPSC-derived mesenchymal stromal cells secreting high levels of functional FVIII ameliorate the hemophilia A phenotype
Cellular and Molecular Life Sciences
Preclinical proof-of-concept in animal models using engineered iPSC-derived cells; lacks human efficacy data, safety follow-up, and dose-response characterization needed to support clinical translation.
Study details
DesignPreclinical in vitro and in vivo efficacy s…
InterventionEngineered iMSCs expressing codon-optimized…