This study defines the most comprehensive CYP17A1 variant spectrum for complete 17-OHD in China, identifies 6 novel pathogenic variants, and uncovers splicing disruption as an under-recognized mechanism in missense mutations. These findings expand the molecular and clinical understanding of 17-OHD, highlight the founder effect of c.985_987delinsAA in the Chinese population, and provide critical insights for genetic diagnosis and counseling.
Novel CYP17A1 variants and functional validation in a large Chinese cohort of complete 17α-hydroxylase deficiency
Yaqing Cao·Min Nie·M. LI·Ran Zhang·Wen Zhang·C. Hu Guo·Xueyan Wu·Anli Tong·Lin Lu·Shi Chen·Chuang Wang·Baolei Guo·Xi Wang·jiangfeng mao
