Guanshan Zhu

Engineered hypoimmunogenic iPSC-derived mesenchymal stromal cells secreting high levels of functional FVIII ameliorate the hemophilia A phenotype

Background: Cell-based gene therapy represents a promising therapeutic avenue for genetic diseases such as hemophilia A (HA). However, clinical translation is limited by low transgene expression and host immune responses. Methods: Using CRISPR/Cas9, we targeted a codon-optimized...