Genetic Treatment of Osteoporosis

Robert M. Nerem, Georgia Institute of Technology, EEC-9731643

About 25 million American women and men have osteoporosis and they suffer about 2 million fractures/year. While doctors can prescribe medications and recommend lifestyle changes to slow bone loss, there is no treatment to add significant mass or strength to bones. At the Georgia Tech/Emory University Center for the Engineering of Living Tissues (GTEC), an NSF-supported Engineering Research Center, engineers from Georgia Tech and orthopedic surgeons from Emory University have teamed up to develop a promising technology for localized gene therapy using LIM Mineralization Protein (LMP-1), a gene that stimulates surrounding cells to make bone.

GTEC Undergraduate Research Scholar Kimberly Huynh is working with Georgia Tech Assistant Professor Robert Guldberg to analyze tissue-engineered bone and determine not only the amount of new bone formation, but also how it is oriented and organized.

They are using an ex vivo approach in which the patient's own blood cells serve as the delivery mechanism containing the LMP-1 gene. The expectation is that gene therapy for tissue engineering will prove to be more effective and less expensive than existing protein-based therapies because the patient serves as his or her own "bioreactor" for bone growth-inducing factors. The center has licensed parts of the technology to industry for further research and development and expects that improved therapies to benefit patients with osteoporosis, osteoarthritis, and bone injuries will result within 5-10 years.