[HTML][HTML] Functional variants of POC5 identified in patients with idiopathic scoliosis

SA Patten, P Margaritte-Jeannin… - The Journal of …, 2015 - Am Soc Clin Investig
SA Patten, P Margaritte-Jeannin, JC Bernard, E Alix, A Labalme, A Besson, SL Girard
The Journal of clinical investigation, 2015Am Soc Clin Investig
Idiopathic scoliosis (IS) is a spine deformity that affects approximately 3% of the population.
The underlying causes of IS are not well understood, although there is clear evidence that
there is a genetic component to the disease. Genetic mapping studies suggest high genetic
heterogeneity, but no IS disease-causing gene has yet been identified. Here, genetic
linkage analyses combined with exome sequencing identified a rare missense variant (p.
A446T) in the centriolar protein gene POC5 that cosegregated with the disease in a large …
Idiopathic scoliosis (IS) is a spine deformity that affects approximately 3% of the population. The underlying causes of IS are not well understood, although there is clear evidence that there is a genetic component to the disease. Genetic mapping studies suggest high genetic heterogeneity, but no IS disease-causing gene has yet been identified. Here, genetic linkage analyses combined with exome sequencing identified a rare missense variant (p.A446T) in the centriolar protein gene POC5 that cosegregated with the disease in a large family with multiple members affected with IS. Subsequently, the p.A446T variant was found in an additional set of families with IS and in an additional 3 cases of IS. Moreover, POC5 variant p.A455P was present and linked to IS in one family and another rare POC5 variant (p.A429V) was identified in an additional 5 cases of IS. In a zebrafish model, expression of any of the 3 human IS-associated POC5 variant mRNAs resulted in spine deformity, without affecting other skeletal structures. Together, these findings indicate that mutations in the POC5 gene contribute to the occurrence of IS.
The Journal of Clinical Investigation