NELL-1 in the treatment of osteoporotic bone loss

AW James, J Shen, X Zhang, G Asatrian… - Nature …, 2015 - nature.com
AW James, J Shen, X Zhang, G Asatrian, R Goyal, JH Kwak, L Jiang, B Bengs, CT Culiat…
Nature communications, 2015nature.com
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 …
Abstract
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.
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