AAV9-Cyp21a1-Por

**AAV9-Cyp21a1-Por** is an investigational adeno-associated virus serotype 9 gene-therapy vector designed for congenital adrenal hyperplasia caused by 21-hydroxylase deficiency. The vector uses a muscle-specific promoter to express mouse **Cyp21a1** and **Por** cDNAs. Cyp21a1 encodes steroid 21-hydroxylase, while POR supplies the electron-transfer activity required for microsomal cytochrome P450 21-hydroxylase function. In a CRISPR/Cas9 21-hydroxylase-deficient mouse model, intramuscular administration lowered the progesterone-to-deoxycorticosterone ratio by approximately 90% and maintained biochemical improvement for up to seven months.

Other names

AAV9-Cyp21a1-PorAAV-9-Cyp21a1-PorAAV 9-Cyp21a1-Por