A CRISPR-engineered swine model of COL2A1 deficiency recapitulates
altered early skeletal developmental defects in humans
Abstract
Loss-of-function mutations in the COL2A1 gene were recently described as
a cause of type II collagenopathy, a major subgroup of genetic skeletal
diseases. However, the pathogenic mechanisms associated with COL2A1
mutations remain unclear, and there are few large-mammal models of these
diseases. In this study, we established a swine model carrying COL2A1
mutations using CRISPR/Cas9 and somatic cell nuclear transfer
technologies. Animals mutant for COL2A1 exhibited severe skeletal
dysplasia characterized by shortened long bones, abnormal vertebrae,
depressed nasal bridge, and cleft palate. Importantly, COL2A1 mutant
piglets suffered tracheal collapse, which was almost certainly the cause
of their death shortly after birth. In conclusion, we have demonstrated
for the first time that overt and striking skeletal dysplasia occurring
in human patients can be recapitulated in large transgenic mammals. This
model underscores the importance of employing large animals as models to
investigate the pathogenesis and potential therapeutics of skeletal
diseases.