Gene therapy: Reversing bone loss in mice

Tweet Nature China - Lingqiang Zhang at the Beijing Institute of Radiation Medicine, Ge Zhang and Ling Qin at the Chinese University of Hong Kong and co-workers have now developed a gene therapy for osteoporosis. The treatment uses small interfering RNAs (siRNAs) that are designed to target the gene Plekho1 in bone-forming cells.

In China, osteoporosis is a disease that has reached epidemic proportions. Treatment using bone-growth stimulating hormones is limited to a two-year period because longer usage may lead to increased bone resorption. This prevalence places a dire need for more effective treatment.

Previous studies have shown that Plekho1 inhibits bone formation, so knocking down Plekho1 should increase bone formation. The RNA is delivered only to bone-forming cells through a specific amino acid sequence embedded in the delivery system.

The unique delivery system successfully inhibited Plekho1 in bone-forming cells for both normal mice and mice suffering from osteoporosis. Within nine weeks, the treatment helped build bone mass and increase bone density, thus effectively reversing osteoporosis.

Nature Medicine - A delivery system targeting bone formation surfaces to facilitate RNAi-based anabolic therapy

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