- Sharma, Swarkar;
- Londono, Douglas;
- Eckalbar, Walter L;
- Gao, Xiaochong;
- Zhang, Dongping;
- Mauldin, Kristen;
- Kou, Ikuyo;
- Takahashi, Atsushi;
- Matsumoto, Morio;
- Kamiya, Nobuhiro;
- Murphy, Karl K;
- Cornelia, Reuel;
- Herring, John A;
- Burns, Dennis;
- Ahituv, Nadav;
- Ikegawa, Shiro;
- Gordon, Derek;
- Wise, Carol A
Idiopathic scoliosis (IS) is a common paediatric musculoskeletal disease that displays a strong female bias. By performing a genome-wide association study (GWAS) of 3,102 individuals, we identify significant associations with 20p11.22 SNPs for females (P=6.89 × 10(-9)) but not males (P=0.71). This association with IS is also found in independent female cohorts from the United States of America and Japan (overall P=2.15 × 10(-10), OR=1.30 (rs6137473)). Unexpectedly, the 20p11.22 IS risk alleles were previously associated with protection from early-onset alopecia, another sexually dimorphic condition. The 174-kb associated locus is distal to PAX1, which encodes paired box 1, a transcription factor involved in spine development. We identify a sequence in the associated locus with enhancer activity in zebrafish somitic muscle and spinal cord, an activity that is abolished by IS-associated SNPs. We thus identify a sexually dimorphic IS susceptibility locus, and propose the first functionally defined candidate mutations in an enhancer that may regulate expression in specific spinal cells.