OR25-01 Durable CYP21A2 Gene Therapy in Non-Human Primates for Treatment of Congenital Adrenal Hyperplasia

Eclov, Rachel J; Lewis, Terra E W; Kapadia, Mayank; Scott, David W; McCoy, Daniel D; Rouse, Jeremy L; Romero, Kirsten B; Beard, Clayton W; Mansfield, Gary S

doi:10.1210/jendso/bvaa046.899

OR25-01 Durable CYP21A2 Gene Therapy in Non-Human Primates for Treatment of Congenital Adrenal Hyperplasia

Severe Congenital Adrenal Hyperplasia (CAH) is most commonly caused by genetic defects in the CYP21A2 gene, which leads to a deficiency of 21-hydroxylase enzyme and disruption in the biosynthesis of Adrenal corticosteriods. Despite treatment with corticosteroids, patients remain at significant ri…