- James, Aaron W;
- Shen, Jia;
- Zhang, Xinli;
- Asatrian, Greg;
- Goyal, Raghav;
- Kwak, Jin H;
- Jiang, Lin;
- Bengs, Benjamin;
- Culiat, Cymbeline T;
- Turner, A Simon;
- Seim III, Howard B;
- Wu, Benjamin M;
- Lyons, Karen;
- Adams, John S;
- Ting, Kang;
- Soo, Chia
NELL-1 is a secreted, osteoinductive protein whose expression rheostatically controls skeletal ossification. Overexpression of NELL-1 results in craniosynostosis in humans and mice, whereas lack of Nell-1 expression is associated with skeletal undermineralization. Here we show that Nell-1-haploinsufficient mice have normal skeletal development but undergo age-related osteoporosis, characterized by a reduction in osteoblast:osteoclast (OB:OC) ratio and increased bone fragility. Recombinant NELL-1 binds to integrin β1 and consequently induces Wnt/β-catenin signalling, associated with increased OB differentiation and inhibition of OC-directed bone resorption. Systemic delivery of NELL-1 to mice with gonadectomy-induced osteoporosis results in improved bone mineral density. When extended to a large animal model, local delivery of NELL-1 to osteoporotic sheep spine leads to significant increase in bone formation. Altogether, these findings suggest that NELL-1 deficiency plays a role in osteoporosis and demonstrate the potential utility of NELL-1 as a combination anabolic/antiosteoclastic therapeutic for bone loss.